Perceptive Advisors

Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, that specializes in developing small molecule therapies for severe, rare musculoskeletal diseases. Founded in 2017, the company employs a precision medicine approach to create innovative treatments targeting various muscle disorders, including Duchenne and Becker muscular dystrophies, as well as limb girdle muscular dystrophies. Utilizing its proprietary muscle-focused drug discovery platform, Edgewise integrates expertise in muscle biology and small molecule engineering to identify and develop therapies that address genetically defined muscle disorders. The company emphasizes a holistic drug discovery method that assesses integrated muscle function, enabling it to create orally bioavailable therapies aimed at improving outcomes for patients with significant unmet medical needs in neuromuscular and cardiac diseases.

Ocuphire Pharma, Inc. is a clinical-stage biopharmaceutical company based in Farmington Hills, Michigan, dedicated to developing and commercializing therapies for various ophthalmic disorders. The company's primary focus is on innovative treatment options for front and back of the eye conditions, with its lead product candidate, Nyxol eye drops, currently in Phase 3 clinical development. Nyxol is a once-daily, preservative-free formulation aimed at addressing issues such as dim light or night vision disturbances, pharmacologically-induced mydriasis, and presbyopia. Additionally, Ocuphire is advancing another candidate, APX3330, a twice-daily oral tablet, which is entering Phase 2 clinical trials to target diabetic retinopathy and diabetic macular edema by inhibiting angiogenesis and inflammation. The company also seeks to explore opportunities for acquiring additional ophthalmic assets and forming strategic partnerships for global commercialization.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Kardigan is a pioneering heart health company dedicated to transforming cardiovascular drug development. It focuses on identifying and matching critical disease drivers with responsive patients from clinical trials, aiming to deliver curative medicines that go beyond symptom management, ultimately providing functional cures for cardiovascular diseases.

RAPT Therapeutics, Inc., established in 2015 and headquartered in South San Francisco, California, is a clinical-stage biopharmaceutical company specializing in immunology-based therapies. The company focuses on developing oral small molecule treatments for patients with unmet needs in oncology and inflammatory diseases. Its lead drug candidates are FLX475, an oral C-C motif chemokine receptor 4 antagonist in Phase 2 clinical trials for various cancer types, and RPT193, which selectively inhibits type 2 T helper cell migration into inflamed tissues. Additionally, RAPT is exploring other targets like general control nonderepressible 2 and hematopoietic progenitor kinase 1 in the discovery phase.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Pheon Therapeutics is a London-based company established in 2022, specializing in the development of Antibody-Drug Conjugates (ADCs) aimed at addressing a variety of difficult-to-treat cancers. The company focuses on creating next-generation ADCs, with its lead program targeting a novel antigen that is significantly expressed in solid tumors across multiple hard-to-treat cancer types. By enhancing the therapeutic options available to healthcare providers, Pheon Therapeutics aims to provide effective treatment alternatives for patients whose cancers do not respond to existing therapies.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, that focuses on developing innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Founded in 2018, the company is working on a small-molecule inhibitor designed to target the underlying causes of this progressive lung disease. The treatment aims to not only halt the progression of IPF but also has the potential to reverse its effects, offering healthcare professionals a new avenue for precision treatments in managing terminal conditions. Endeavor BioMedicines strives to improve patient outcomes and enhance quality of life for those affected by this challenging illness.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). This chronic condition, which encompasses disorders such as ulcerative colitis and Crohn's disease, involves inflammation in the gastrointestinal tract. Spyre Therapeutics combines advanced antibody engineering with rational therapeutic combinations and precision medicine strategies to enhance patient selection. The company's goal is to create next-generation products that improve upon existing treatment options, which include anti-inflammatory drugs, immunosuppressants, and biologics. Through its robust pipeline, Spyre Therapeutics seeks to address the unmet needs of patients suffering from IBD.

Precision BioSciences, Inc. is a biotechnology company focused on genome editing, headquartered in Durham, North Carolina. The company utilizes its proprietary ARCUS technology to develop therapeutic products aimed at treating human diseases and improving food and agricultural solutions. Precision BioSciences operates through two segments: Therapeutic and Food. The Therapeutic segment is particularly dedicated to advancing allogeneic CAR T immunotherapies for cancer treatment, including candidates like PBCAR0191 and PBCAR20A, which target specific tumor antigens. The company is also involved in in vivo gene correction and is exploring treatments for conditions such as acute lymphoblastic leukemia and chronic lymphocytic leukemia. Additionally, the Food segment develops innovative nutrition products. Precision BioSciences has formed strategic collaborations, including agreements with Shire Plc and Gilead Sciences, to enhance its research and development capabilities. Founded in 2006, the company aims to harness its next-generation gene editing technology to address pressing health challenges.

Crinetics Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company headquartered in San Diego, California, dedicated to the discovery, development, and commercialization of innovative therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist currently undergoing Phase I clinical trials aimed at treating acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is developing several other therapeutics, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, and an oral somatostatin type 5 receptor agonist for congenital hyperinsulinism. The company is also working on an oral adrenocorticotrophic hormone antagonist for conditions such as Cushing’s syndrome and congenital adrenal hyperplasia. Founded in 2008, Crinetics Pharmaceuticals is focused on leveraging its internal discovery efforts to create effective treatments while utilizing hormonal biomarker endpoints to enhance the efficiency and cost-effectiveness of its clinical studies.

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New York, focused on developing innovative therapies for serious medical conditions with significant unmet needs. The company's lead product candidate, AT-007, is in phase I/II clinical trials for the treatment of galactosemia, while AT-001 is undergoing phase II trials for diabetic cardiomyopathy and phase I trials for diabetic peripheral neuropathy. Additionally, AT-003 is in phase I trials for diabetic retinopathy. The company also has preclinical candidates, such as AT-104, aimed at treating orphan hematological oncology conditions. Founded in 2016, Applied Therapeutics leverages advancements in technology and regulatory processes to create drugs targeting well-established molecular mechanisms.

Mainstay Medical, based in Dublin, Ireland, with operations in the U.S. and Australia, is a medical device company specializing in the development and commercialization of innovative treatments for chronic low back pain. Its flagship product, ReActiv8, is an implantable neurostimulation system designed to restore muscle control and improve function in the lumbar spine, thereby enhancing patients' quality of life. Founded in 2008, the company collaborates with scientists and clinical experts to address the significant unmet need in this large, underserved patient population.

Impulse Dynamics N.V. is a medical device company focused on developing electrical therapies for chronic heart failure, operating primarily in the United States and Europe. The company's key innovation is its cardiac contractility modulation (CCM) therapy, which utilizes non-excitatory electrical impulses delivered to the heart muscles. This therapy aims to enhance cardiac strength and improve blood flow, offering a minimally invasive treatment option for patients with limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for research initiatives. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, alongside product development centers in New Jersey and Israel.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for patients affected by rare allergic and immunological diseases. The company focuses on creating life-changing treatments, with its lead program, STAR-0215, being a monoclonal antibody designed to inhibit plasma kallikrein, currently in preclinical development for hereditary angioedema. Additionally, Astria is advancing STAR-0310, another monoclonal antibody, which targets the OX40 receptor and is also in preclinical development for atopic dermatitis, a condition characterized by impaired skin barrier function and significant itching. Through its targeted approach, Astria Therapeutics aims to address unmet medical needs in the field of immunology.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). This chronic condition, which encompasses disorders such as ulcerative colitis and Crohn's disease, involves inflammation in the gastrointestinal tract. Spyre Therapeutics combines advanced antibody engineering with rational therapeutic combinations and precision medicine strategies to enhance patient selection. The company's goal is to create next-generation products that improve upon existing treatment options, which include anti-inflammatory drugs, immunosuppressants, and biologics. Through its robust pipeline, Spyre Therapeutics seeks to address the unmet needs of patients suffering from IBD.

Soleno Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapeutics for rare diseases. The company's primary focus is on its lead candidate, Diazoxide Choline Controlled Release (DCCR), a once-daily oral tablet designed for the treatment of Prader-Willi Syndrome (PWS). DCCR contains diazoxide choline, a choline salt of diazoxide, which is a benzothiadiazine. Currently, the product is being evaluated in a Phase III clinical development program. Established in 1999 and headquartered in Redwood City, California, Soleno Therapeutics was formerly known as Capnia, Inc. before rebranding in May 2017.

Inhibrx, Inc. is a clinical-stage biotechnology company based in La Jolla, California, dedicated to developing a diverse pipeline of novel biologic therapeutic candidates. The company employs advanced protein engineering techniques, including its proprietary sdAb platform, to target complex disease biology, with a focus on oncology, orphan diseases, and infectious diseases. Inhibrx's lead candidates include INBRX-109, a multivalent agonist in Phase 1 trials for solid tumors; INBRX-105, an antagonist of PD-L1 also in Phase 1 trials; INBRX-101, an Fc-fusion protein for alpha-1 antitrypsin deficiency; and INBRX-103, a monoclonal antibody targeting CD47. Additionally, the company is developing preclinical programs such as INBRX-106, an agonist of OX40, and INBRX-111, an antibody targeting Pseudomonas aeruginosa. Inhibrx has established collaborations with organizations like Celgene and Bluebird Bio and has received funding from various granting agencies, including the NIH and CARB-X.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing innovative small-molecule therapies for neurological disorders. The company focuses on understanding the intricate biology of neuronal receptor complexes, which are essential for proper neuronal function and signaling. By mapping and targeting these receptor systems, Rapport Therapeutics aims to create precision medicines that can effectively address the unmet needs of patients suffering from central nervous system disorders. Through its foundational research, the company seeks to transform treatment options and improve outcomes for individuals with these challenging conditions.

Forte Biosciences is a biotechnology company specializing in dermatology and biopharmaceuticals. The company's lead product, FB-401, is a topically applied live biotherapeutic designed to treat inflammatory skin diseases, developed in collaboration with the National Institutes of Health and the National Institute of Allergy and Infectious Diseases. Additionally, Forte Biosciences is advancing another product candidate, FB-102, which is a proprietary therapeutic molecule aimed at addressing a range of autoimmune conditions, including graft-versus-host disease, vitiligo, and alopecia areata. The company is currently in the preclinical trial stage for FB-102, reflecting its commitment to developing innovative treatments for dermatological and autoimmune disorders.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

Fusion Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company headquartered in Hamilton, Canada, specializing in the development of radiopharmaceuticals as precision medicines for challenging cancers. The company employs its proprietary Targeted Alpha Therapies platform and Fast-Clear linker technology to connect alpha particle-emitting isotopes to antibodies, enabling the selective delivery of therapeutic agents to tumors. Fusion's lead product candidate, FPI-1434, is currently undergoing Phase 1 clinical trials as a monotherapy for solid tumors expressing insulin-like growth factor 1 receptor. Additionally, the company is exploring the use of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors in preclinical studies to enhance anti-tumor activity. Fusion is also advancing another product candidate, FPI-1966, into clinical development for head and neck and bladder cancers that express fibroblast growth factor receptor. Founded in 2014 as a spinout of the Centre for Probe Development and Commercialization, Fusion aims to transform how cancers are treated through innovative radiochemistry techniques.

Biodesix, Inc. is a diagnostic solutions company based in Boulder, Colorado, specializing in blood-based tests for lung cancer and related services. The company offers a range of diagnostic tests, including the Nodify XL2 and Nodify CDT tests, which assist physicians in assessing the risk of malignancy in patients with suspicious lung nodules. Additionally, Biodesix provides tumor profiling and immune profiling tests, such as GeneStrat and VeriStrat, to support timely treatment decisions for oncologists. These tests are processed in a CLIA-certified laboratory, with results typically available within 72 hours. Beyond its diagnostic offerings, Biodesix also engages in clinical research, development, and testing services for biopharmaceutical companies, focusing on the development and commercialization of companion diagnostics to enhance therapeutic efficacy. Established in 2005, Biodesix continues to advance its proprietary mass spectrometry-based discovery platform to improve patient outcomes in lung disease.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing medicines for underserved patient populations affected by rare diseases and cancer. The company employs a precision medicine approach and has a diverse portfolio of small molecule targeted oncology products. Its lead candidate, nirogacestat, an oral gamma secretase inhibitor, is currently in Phase III clinical trials for desmoid tumors. Additionally, SpringWorks is advancing several other product candidates, including mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for multiple myeloma. The company has established collaborations with organizations such as BeiGene, GlaxoSmithKline, and Allogene, aimed at enhancing the development of its therapies. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks is committed to unlocking the potential of its pipeline to provide innovative solutions for patients suffering from cancer and rare diseases.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing implanted drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company utilizes a refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of its formulation of the anti-epileptic drug valproic acid, known as CT-010. This innovative approach aims to bypass the blood-brain barrier, enhancing treatment outcomes for patients with chronic neurological conditions. Founded in 2010, Cerebral Therapeutics is dedicated to improving the lives of individuals affected by uncontrolled neurological diseases.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company that specializes in developing and commercializing innovative drug and delivery solutions for the localized treatment of ear, nose, and throat diseases. Utilizing its proprietary XTreo technology platform, the company aims to deliver medications directly to affected tissues for extended periods with a single administration. Its lead product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed for non-invasive in-office procedures, providing up to six months of continuous drug therapy specifically for chronic rhinosinusitis. The active ingredient in these formulations is mometasone furoate, which is recognized for its efficacy and safety in various FDA-approved therapies. Founded in 2005 and headquartered in Watertown, Massachusetts, Lyra Therapeutics was formerly known as 480 Biomedical, Inc. before rebranding in July 2018.

VivoSense, also known as Vivonoetics, is a data analysis and software company focused on providing wearable sensor physiological monitoring solutions for research and clinical trials. The company develops digital clinical measures designed to enhance patient research and care by delivering digital biomarkers derived from wearable sensor data. This platform allows researchers to collect and analyze real-world data accurately, facilitating informed decision-making and risk evaluation associated with various medical treatments in regulated clinical environments. Through its innovative approach, VivoSense aims to improve the efficiency and effectiveness of clinical research.

DNAnexus, Inc. is a provider of cloud-based genome informatics and data management tools aimed at enterprises engaged in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company offers a platform that facilitates the analysis of raw sequencing data and integration with clinical and phenotypic data, promoting seamless collaboration among users. Key offerings include MOSAIC Microbiome, a platform for collaborative microbiome research; DNAnexus Apollo, which focuses on clinico-genomic data exploration; and DNAnexus Titan, a comprehensive data analysis solution. Additionally, DNAnexus Portals provide secure online workspaces for cross-disciplinary collaboration. The Clinico-Genomic Data Solution program addresses the need for disease-specific datasets by building networks with healthcare partners to enhance diagnosis and treatment pathways. DNAnexus serves a diverse client base, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, that focuses on developing innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Founded in 2018, the company is working on a small-molecule inhibitor designed to target the underlying causes of this progressive lung disease. The treatment aims to not only halt the progression of IPF but also has the potential to reverse its effects, offering healthcare professionals a new avenue for precision treatments in managing terminal conditions. Endeavor BioMedicines strives to improve patient outcomes and enhance quality of life for those affected by this challenging illness.

Orchestra BioMed is a biomedical innovation company based in New Hope, Pennsylvania, founded in 2017. The company specializes in developing high-impact therapeutic solutions for major medical conditions, with a primary focus on cardiovascular diseases, which are responsible for approximately 31% of global deaths annually. Orchestra BioMed's business model involves strategic partnerships with medical device companies to accelerate the commercialization of its products. Its key product candidates include BackBeat Cardiac Neuromodulation Therapy for hypertension and Virtue Sirolimus AngioInfusionBalloon for atherosclerotic artery disease.

AvengeBio is a biotechnology company based in Cambridge, Massachusetts, focused on developing cell-generated immunotherapies aimed at eradicating solid tumors. Founded in 2019, the company is advancing a drug delivery platform designed to enable the precision delivery of cytokines and other potent immunomodulatory molecules. This innovative approach allows for controlled release over time, fostering robust local and systemic anti-tumor immune responses while minimizing the toxicities typically associated with conventional immunotherapies. Through its research and development efforts, AvengeBio seeks to address the challenges posed by intractable solid tumors and improve treatment outcomes for cancer patients.

Mythic Therapeutics is a biotechnology company dedicated to advancing cancer treatment through innovative antibody-drug conjugates (ADCs). The company specializes in a protein engineering approach that enhances the potency of ADCs while maintaining safety, allowing for more effective targeted therapies across various tumor types. By improving the uptake of these therapeutics in cancerous cells and minimizing payload release in healthy cells, Mythic Therapeutics aims to increase the efficacy of treatments without causing significant side effects or toxicity. This technology positions the company to make a meaningful impact in cancer care, enabling healthcare practitioners to better utilize ADCs for a wide array of patients.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, established in 2018. The company is dedicated to developing clinical candidates targeting cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at treating resistant hypertension and primary aldosteronism. CinCor Pharma's mission is to advance its promising clinical candidates toward marketing approval, emphasizing the development of innovative therapies for patients suffering from cardio-renal conditions.

Science 37, Inc. is a technology-enabled clinical trial company that specializes in developing patient-centric models for clinical research, aimed at accelerating biomedical discovery. The company utilizes its innovative platform, NORA (Network Oriented Research Assistant), a cloud-based mobile research tool that facilitates communication between researchers, patients, and mobile nurses through videos, surveys, and photographs. By providing decentralized clinical trial services, Science 37 enables access to diverse patient populations that traditional site-based research may overlook. The company has established itself as a leader in conducting virtual trials, leveraging an extensive network of telemedicine investigators and home-health nurses to enhance patient enrollment and retention. Serving a wide range of clients, including pharmaceutical sponsors, biotech firms, and academic institutions, Science 37 aims to transform clinical research through its comprehensive, fully integrated trial platform. Founded in 2014 and headquartered in Los Angeles, California, Science 37 continues to innovate in the field of clinical research.

Genome Medical, Inc. is a genomic medicine delivery company based in South San Francisco, California, established in 2016. It operates a technology platform known as Genome Care Delivery, which connects individuals and healthcare providers with genetic specialists to facilitate virtual genetic consultations. The company focuses on various areas of clinical care, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacogenomics. By providing expert genetic assessments and personalized clinical action plans, Genome Medical helps patients understand their genetic risks and make informed treatment decisions. Additionally, the company supports healthcare providers in navigating the growing field of genetics, enhancing disease diagnosis, and improving patient care while aiming to reduce overall healthcare costs.

GeneDx is a company dedicated to providing personalized and actionable health insights that enhance diagnosis and treatment, ultimately improving health outcomes. Positioned at the intersection of diagnostics and data science, GeneDx combines decades of genomic expertise with the capability to interpret clinical data at scale. The company focuses on accelerating the application of genomic information and large-scale clinical data to establish precision medicine as a standard practice in healthcare. GeneDx leads the transformation of healthcare through its comprehensive exome and genome testing services, leveraging one of the largest rare disease data sets in the world. The business operates through two segments: GeneDx, which generates the majority of its revenue, and Legacy Sema4 diagnostics.

Amylyx Pharmaceuticals develops innovative therapies for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The company's primary product, AMX0035, is an investigational therapy that combines two active compounds, sodium phenylbutyrate and Taurursodiol. This fixed-dose formulation aims to address mitochondrial energy crises and the accumulation of toxic proteins in the endoplasmic reticulum, which contribute to neurological decline. In addition to ALS, Amylyx's research pipeline includes potential treatments for Wolfram syndrome and Alzheimer's disease. Founded in 2013 and based in Cambridge, Massachusetts, Amylyx Pharmaceuticals is dedicated to advancing solutions for serious brain disorders.

Point Biopharma Global Inc., established in 2019 and headquartered in Toronto, Canada, specializes in the development and commercialization of radioligand therapies for cancer treatment. The company leverages a portfolio of advanced radiopharmaceutical assets, expertise in radioisotopes like Actinium-225 and Lutetium-177, and innovative manufacturing technology. Point Biopharma Global's pipeline includes several promising candidates, such as PNT2002, PNT2004, PNT2003, and PNT2001, aiming to revolutionize theragnostic drug development and radioligand commercialization.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

Nautilus Biotechnology is a development-stage life sciences company focused on advancing proteomics, the study of proteins and their functions, to enhance therapeutic development and medical diagnostics. The company has created a platform technology that aims to quantify and unravel the complexities of the human proteome. Its flagship product, the Proteomic Analysis System, is a prototype single-molecule instrument designed to facilitate these efforts. The team at Nautilus comprises experts from various disciplines, including protein chemistry, molecular biology, and data science, enabling a multifaceted approach to overcoming challenges in the field. By harnessing this diverse expertise, Nautilus aims to revolutionize drug development and improve healthcare management through personalized and predictive medicine.

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.

Juno Diagnostics, Inc. is a health technology company based in San Diego, California, that specializes in cell-free DNA-based prenatal testing. Founded in 2017, the company aims to democratize access to genetic information by developing simple, cost-effective devices for point-of-need genetic testing. Juno's technology allows for noninvasive prenatal tests that enhance sample collection and provide high-quality test results without the challenges associated with traditional testing methods, such as high costs, long lead times, and the need for phlebotomy. In addition to prenatal testing, Juno is also involved in developing genetic testing tools and applications in areas such as epigenetics, inherited diseases, and cancer diagnostics.

Renovia Inc. was a Boston-based healthcare company founded in 2016, focused on developing prescription digital therapeutics for pelvic floor disorders. The company aimed to enhance the lives of women suffering from conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. Renovia's innovative device provided a clinically proven, non-surgical, drug-free treatment option, allowing for real-time visualization of pelvic movement during muscle training, thereby facilitating effective therapy. However, in late 2022, Renovia ceased operations following a strategic review by its investors, which included the consideration of a potential sale. In January 2023, most of the company's assets, including its intellectual property, were acquired by Axena Health, Inc.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Crossover Health, established in 2010, specializes in providing comprehensive workplace healthcare services for employers and their employees. Headquartered in Aliso Viejo, California, with health centers across the United States, the company offers a wide range of services including primary care, urgent care, chronic disease management, health coaching, physical therapy, and more. Crossover Health aims to deliver exceptional member experiences and advanced health management services, ultimately promoting a healthy and motivated workforce for its clients.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

Day One Biopharmaceuticals is a clinical-stage biotechnology company that specializes in developing targeted cancer therapies for patients of all ages, with a particular focus on pediatric patients. Recognizing the advancements in cancer research, the company aims to create treatments that can benefit both children and adults diagnosed with genetically defined cancers. Day One Biopharmaceuticals prioritizes the rapid development of therapies to meet the urgent needs of families facing cancer diagnoses. Their lead product candidate, DAY101, is an oral, highly-selective pan-RAF kinase inhibitor designed to penetrate the brain, addressing a significant treatment gap in pediatric oncology. By licensing and acquiring promising products from research institutions and other companies, Day One Biopharmaceuticals strives to bring effective medicines to market for those impacted by cancer.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, focused on developing innovative vaccines using its proprietary Multiple Antigen Presenting System (MAPS) technology. This approach allows for the combination of protective polysaccharides and proteins into a single vaccine, thereby enhancing immune responses against a variety of infectious diseases affecting both children and adults. Affinivax targets several significant pathogens, including Streptococcus pneumoniae, bacterial nosocomial infections, Salmonella typhi, and Staphylococcus aureus. The company aims to create vaccines that not only provide broader disease coverage compared to existing options but also address infectious diseases lacking effective immunization strategies. Founded in 2014, Affinivax benefits from the expertise of leading professionals in infectious diseases and vaccines, and it has received backing from the Bill & Melinda Gates Foundation, along with an exclusive license from Boston Children’s Hospital for its MAPS technology.

IsoPlexis is a life science technology company based in Branford, Connecticut, founded in 2013. It focuses on developing a single-cell detection system that identifies patient immune responses at the single-cell level. This innovative platform enables the assessment of therapeutic safety and efficacy, as well as the monitoring of disease progression. IsoPlexis specializes in cell-based proteomic systems and biomarkers, providing insights into functional protein biology and cellular signaling networks with high resolution. By offering these capabilities, the company aids in predicting patient responses to novel immunotherapies and cellular therapies, ultimately enhancing therapeutic development and informing clinical decisions.

IsoPlexis is a life science technology company based in Branford, Connecticut, founded in 2013. It focuses on developing a single-cell detection system that identifies patient immune responses at the single-cell level. This innovative platform enables the assessment of therapeutic safety and efficacy, as well as the monitoring of disease progression. IsoPlexis specializes in cell-based proteomic systems and biomarkers, providing insights into functional protein biology and cellular signaling networks with high resolution. By offering these capabilities, the company aids in predicting patient responses to novel immunotherapies and cellular therapies, ultimately enhancing therapeutic development and informing clinical decisions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Adagio Medical specializes in the design and manufacture of medical devices aimed at treating cardiac arrhythmias. Founded in 2011 and based in Laguna Hills, California, the company is dedicated to the research and development of innovative cryoablation catheters. These devices are engineered to create safe, continuous, transmural lesions that effectively address various cardiac conditions, including paroxysmal and persistent atrial fibrillation, atrial flutter, and ventricular tachycardia. Adagio Medical's product offerings include the continuous lesion ablation system (iCLAS), specialized catheters, esophageal balloons, and consoles, all developed to enhance the capabilities of electrophysiologists in the treatment of cardiovascular diseases.

Prometheus Biosciences, established in 1995 and headquartered in San Diego, California, specializes in developing and commercializing innovative pharmaceutical and diagnostic products. The company's portfolio includes tests that enable physicians to provide personalized patient care, such as monitoring drug and anti-drug antibody levels, diagnosing and prognosticating inflammatory bowel disease (IBD), and detecting celiac disease and lactose intolerance. Prometheus also offers therapeutic products like Entocort EC for treating Crohn's disease and has agreements to develop and market other drugs. The company's commitment to innovation positions it as a leader in precision healthcare, particularly in gastroenterology.

Impulse Dynamics N.V. is a medical device company focused on developing electrical therapies for chronic heart failure, operating primarily in the United States and Europe. The company's key innovation is its cardiac contractility modulation (CCM) therapy, which utilizes non-excitatory electrical impulses delivered to the heart muscles. This therapy aims to enhance cardiac strength and improve blood flow, offering a minimally invasive treatment option for patients with limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for research initiatives. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, alongside product development centers in New Jersey and Israel.

AavantiBio, Inc. is a biopharmaceutical company based in Gainesville, Florida, focused on developing gene transfer and gene editing therapies for rare genetic diseases. Established in 2019, the company primarily targets Friedreich’s Ataxia, a severe inherited condition that leads to significant cardiac and central nervous system dysfunction. In addition to addressing this specific disorder, AavantiBio is advancing a pipeline of innovative gene therapies aimed at treating various autoimmune diseases and other conditions with unmet medical needs. Through its commitment to research and development, the company seeks to improve the lives of patients affected by these debilitating genetic disorders.

Nuvation Bio Inc. is a biopharmaceutical company dedicated to addressing unmet needs in oncology through the development of novel therapies for challenging cancers. Founded in 2018 and headquartered in New York, with an additional office in San Francisco, Nuvation Bio focuses on treating patients whose conditions have not responded to conventional treatments. The company's leading clinical-stage candidate, NUV-868, is a BD2-selective oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. In addition to NUV-868, Nuvation Bio is advancing a proprietary small molecule Drug-Drug Conjugate platform aimed at enhancing therapeutic efficacy. The company’s comprehensive portfolio includes multiple drug development candidates designed to meet the specific needs of cancer patients.

Metabolon, Inc. is a health technology company specializing in metabolomics, focusing on the development of diagnostic tests and analytical methods for early disease detection and precision medicine. The company offers a range of clinical diagnostics, including QUANTOSE IR for measuring insulin resistance, QUANTOSE IGT for assessing glucose tolerance, and Meta IMD for identifying metabolites linked to inherited metabolic disorders. In addition to its diagnostic capabilities, Metabolon provides customizable research services and multiomics solutions to support academic and commercial investigations across various life science sectors. Its technologies are applicable in areas such as cardiovascular health, diabetes, oncology, and respiratory disorders. Founded in 2000 and headquartered in Morrisville, North Carolina, with additional offices in Europe and sales locations in the UK, Spain, and China, Metabolon collaborates with numerous strategic partners in the pharmaceutical and biotechnology industries to enhance research and development outcomes.

Escient Pharmaceuticals is a biotechnology company based in San Diego, California, that develops and manufactures drugs targeting G protein-coupled receptors (GPCRs) for the treatment of neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, the company specializes in harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs), to address various neurosensory-inflammatory disorders. Escient's innovative approach aims to provide first-in-class therapies for conditions associated with mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers and patients facing these challenging diseases.

I-Mab Biopharma (Shanghai) Co., Ltd. is a clinical-stage biopharmaceutical company based in Shanghai, China, with additional offices in Beijing and Rockville, Maryland. Founded in 2016, the company focuses on the discovery, development, and commercialization of innovative biologics aimed at addressing significant unmet medical needs, particularly in the fields of immuno-oncology and immuno-inflammation. I-Mab's diverse drug pipeline targets various therapeutic areas, including multiple myeloma, autoimmune diseases, pediatric growth hormone deficiency, and several cancer indications such as head and neck cancer. Key candidates in its portfolio include Uliledlimab, an antibody for solid tumors, along with Ragistomig and Givastomig, highlighting the company's commitment to developing transformative therapies for patients.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing therapies that target oncogenic drivers. The company employs a tumor-agnostic approach, selecting patients based on the underlying genetics of their tumors rather than their histological characteristics. Its leading product candidate, milademetan, is an oral small molecule inhibitor of MDM2, which plays a role in various cancers. In addition to milademetan, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells through the inhibition of RAD52. This targeted strategy aims to improve treatment outcomes for patients who are genetically predisposed to benefit from these therapies.

Metabolon, Inc. is a health technology company specializing in metabolomics, focusing on the development of diagnostic tests and analytical methods for early disease detection and precision medicine. The company offers a range of clinical diagnostics, including QUANTOSE IR for measuring insulin resistance, QUANTOSE IGT for assessing glucose tolerance, and Meta IMD for identifying metabolites linked to inherited metabolic disorders. In addition to its diagnostic capabilities, Metabolon provides customizable research services and multiomics solutions to support academic and commercial investigations across various life science sectors. Its technologies are applicable in areas such as cardiovascular health, diabetes, oncology, and respiratory disorders. Founded in 2000 and headquartered in Morrisville, North Carolina, with additional offices in Europe and sales locations in the UK, Spain, and China, Metabolon collaborates with numerous strategic partners in the pharmaceutical and biotechnology industries to enhance research and development outcomes.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Verona Pharma is a clinical-stage biopharmaceutical company based in London, established in 2005. The company specializes in developing and commercializing therapies for respiratory diseases that have significant unmet medical needs, including chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. Its lead product candidate, ensifentrine, is an inhaled dual inhibitor of the enzymes phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is being developed in various formulations, including nebulized, dry powder inhaler, and pressurized metered-dose inhaler, with the nebulized form currently in Phase 2b clinical development for the maintenance treatment of COPD. Verona Pharma's commitment is to enhance the health and quality of life for individuals suffering from chronic respiratory conditions through innovative therapeutic solutions.

Pear Therapeutics, Inc. is a healthcare company specializing in the discovery, development, and delivery of software-based therapeutics aimed at treating behavioral health disorders, including addiction, schizophrenia, anxiety, depression, and chronic insomnia. Founded in 2013 and headquartered in Boston, with an additional office in San Francisco, Pear Therapeutics offers a range of digital therapies designed to enhance treatment outcomes. Notable products include reSET, a tool for substance abuse treatment used alongside traditional therapy; reSET-O, which addresses opioid use disorder; and Somryst, aimed at chronic insomnia. The company also provides PearConnect, a patient services center that connects users with advocates, clinicians, and insurance providers to facilitate support and resource access throughout treatment. By integrating digital interventions with pharmaceuticals, Pear Therapeutics aims to improve patient engagement, clinical outcomes, and cost-effectiveness in healthcare.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Genome Medical, Inc. is a genomic medicine delivery company based in South San Francisco, California, established in 2016. It operates a technology platform known as Genome Care Delivery, which connects individuals and healthcare providers with genetic specialists to facilitate virtual genetic consultations. The company focuses on various areas of clinical care, including cancer, cardiovascular health, reproductive health, pediatrics, and pharmacogenomics. By providing expert genetic assessments and personalized clinical action plans, Genome Medical helps patients understand their genetic risks and make informed treatment decisions. Additionally, the company supports healthcare providers in navigating the growing field of genetics, enhancing disease diagnosis, and improving patient care while aiming to reduce overall healthcare costs.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

DNAnexus, Inc. is a provider of cloud-based genome informatics and data management tools aimed at enterprises engaged in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company offers a platform that facilitates the analysis of raw sequencing data and integration with clinical and phenotypic data, promoting seamless collaboration among users. Key offerings include MOSAIC Microbiome, a platform for collaborative microbiome research; DNAnexus Apollo, which focuses on clinico-genomic data exploration; and DNAnexus Titan, a comprehensive data analysis solution. Additionally, DNAnexus Portals provide secure online workspaces for cross-disciplinary collaboration. The Clinico-Genomic Data Solution program addresses the need for disease-specific datasets by building networks with healthcare partners to enhance diagnosis and treatment pathways. DNAnexus serves a diverse client base, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

Nautilus Biotechnology is a development-stage life sciences company focused on advancing proteomics, the study of proteins and their functions, to enhance therapeutic development and medical diagnostics. The company has created a platform technology that aims to quantify and unravel the complexities of the human proteome. Its flagship product, the Proteomic Analysis System, is a prototype single-molecule instrument designed to facilitate these efforts. The team at Nautilus comprises experts from various disciplines, including protein chemistry, molecular biology, and data science, enabling a multifaceted approach to overcoming challenges in the field. By harnessing this diverse expertise, Nautilus aims to revolutionize drug development and improve healthcare management through personalized and predictive medicine.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that focuses on discovering and developing antiviral therapies for severe viral infections. The company's lead candidate, AT-527, is an antiviral agent currently undergoing phase 3 clinical trials for the treatment of COVID-19, caused by SARS-CoV-2. Additionally, Atea is developing several other product candidates, including AT-787, which is in phase 2 trials for hepatitis C, and AT-752 for dengue. Other candidates, such as AT-889 and AT-934, are also in phase 2 clinical trials targeting respiratory syncytial virus. Established in 2014, Atea Pharmaceuticals aims to improve treatment options for patients suffering from serious viral infections.

COMPASS Pathways is a mental health care company based in Cheshire, United Kingdom, with operations in the United States. The company is focused on developing innovative therapies for treatment-resistant depression (TRD) using its proprietary psilocybin formulation, COMP360. Currently undergoing Phase IIb clinical trials, COMP360 aims to provide a new treatment option for patients who have not responded to traditional depression therapies. COMPASS Pathways emphasizes the combination of psilocybin-assisted therapy with psychological support to enhance treatment efficacy. The company is committed to addressing the significant unmet needs in mental health care by advancing evidence-based solutions for those suffering from mental health challenges.