Perceptive Advisors

Merida Biosciences is a biotechnology company specializing in the development of precision therapies for autoimmune diseases. It employs a protein engineering platform to create novel Fc therapeutics, which selectively target and neutralize specific antibodies, aiming to treat the root cause of antibody-driven diseases with high precision and durability.

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

Zucara Therapeutics Inc. is a biotechnology company based in Toronto, Canada, with an additional office in Vancouver. Founded in 2014, the company focuses on developing innovative therapeutics aimed at preventing hypoglycemia, or low blood sugar, in patients with diabetes. Its lead pre-clinical technology targets somatostatin type 2 receptors in the pancreas, which are inadequately regulated in individuals with Type 1 diabetes. By blocking these receptors, Zucara's treatment aims to prevent hypoglycemia and restore natural glucose levels in the bloodstream. This approach represents a significant advancement over existing therapies, which typically only address hypoglycemia after it has occurred, rather than preventing it from happening in the first place.

Axonis Therapeutics is a biotechnology company focused on developing therapies for neurological disorders by targeting KCC2, a key mediator of inhibition in the brain. The company utilizes a proprietary discovery engine centered on the neuron-specific K-Cl cotransporter (KCC2) to address various conditions, including epilepsy and pain, as well as other central nervous system pathologies. By harnessing breakthrough research from institutions such as Boston Children’s Hospital, Harvard, and Laval University, Axonis aims to enhance the lives of individuals affected by these disorders through improved modulation of neuronal inhibition.

Crescent Biopharma is a biotechnology firm focused on developing novel precision-engineered medicines targeting verified biology to improve care for patients with solid tumors.

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.

Inhibikase Therapeutics has developed the first small molecule platform capable of treating both bacterial and viral infectious diseases through a common mechanism of action in the host. The company's host targeting strategy creates multi-pathogen anti-infectives without stimulating resistance.

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.

Oruka Therapeutics is advancing innovative biologics to redefine patient care standards in chronic skin 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.

scPharmaceuticals Inc. is a pharmaceutical company focused on developing and commercializing innovative therapeutic options for patients with serious medical conditions. The company’s lead product candidate, Furoscix, features a proprietary buffered formulation of furosemide delivered via the SmartDose drug delivery system, targeting congestion in patients with decompensated heart failure. In addition to Furoscix, scPharmaceuticals is advancing its product pipeline with scCeftriaxone, an antibiotic for infections from gram-positive and gram-negative organisms, and its scCarbapenem program, aimed at treating infections caused by gram-negative organisms. Founded in 2013 and headquartered in Burlington, Massachusetts, scPharmaceuticals aims to provide a convenient, safe, and effective alternative to intravenous drug administration through subcutaneous delivery, addressing the associated costs and risks. The company collaborates with West Pharmaceutical Services, Inc. to enhance the SmartDose system for Furoscix.

Confo Therapeutics is a drug discovery company based in Brussels, Belgium, founded in 2015. The company focuses on developing internal drug discovery programs centered on G-protein coupled receptors (GPCRs), which are critical targets for therapeutic intervention. Utilizing its proprietary CONFO technology, Confo Therapeutics stabilizes active-state conformations of GPCRs, enabling researchers to access previously unreachable structural features. This innovation facilitates the discovery of novel agonists, addressing unmet medical needs and potentially leading to improved treatments in the medical field. The company aims to build a robust portfolio of drug discovery programs, leveraging its unique approach to enhance therapeutic options.

InSightec Ltd. specializes in developing and distributing the Exablate platform, which employs MR-guided focused ultrasound technology for non-invasive medical treatments in oncology and gynecology. The company offers solutions such as Exablate Neuro, designed for personalized thalamotomy to address movement disorders and pain without invasive surgery, and a thermal ablation technique for the treatment of uterine fibroids, which alleviates symptoms while minimizing damage to surrounding tissues. Founded in 1999 and headquartered in Tirat Carmel, Israel, InSightec has expanded its presence globally with operations in Europe, Asia, and the Americas. The Exablate system has received various regulatory approvals, including CE marks and FDA approval, and has been recognized with multiple innovation awards for its contributions to healthcare. In addition to its medical devices, InSightec provides clinical training, business support, maintenance, and customer services to healthcare facilities using its technology.

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.

CinDome Pharma is a Cincinnati-based pharmaceutical company focused on developing treatments for gastrointestinal diseases, particularly chronic gastroparesis, which affects millions of individuals. The company is engaged in the creation of innovative drug therapies, including a novel chemical entity known as CIN-102, which utilizes deuteration to enhance safety, effectiveness, and tolerability. Through its commitment to addressing the challenges posed by chronic gastroparesis, CinDome Pharma aims to improve the quality of life for patients suffering from this condition.

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.

Zevra Therapeutics is a development-stage biopharmaceutical company focused on creating new therapies for pain, ADHD, and other central nervous system (CNS) diseases, particularly in the realm of rare diseases. The company is dedicated to developing safer, abuse-resistant opioid pain relievers and employs a proprietary Ligand Activated Therapy (LAT) approach to drug discovery and development. This innovative platform allows Zevra to enhance existing medications and streamline the development process while preserving strong intellectual property rights. By integrating scientific research with patient needs and data-driven strategies, Zevra Therapeutics aims to address the challenges of drug development, delivering transformational therapies for conditions with limited or no treatment options.

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.

Neurosterix is a biopharmaceutical company focused on developing innovative medicines for neurological disorders through a unique approach known as allosteric modulation. This method aims to address the limitations of traditional treatments, enhancing efficacy, safety, and tolerability for conditions such as schizophrenia and other neuropsychiatric disorders. By leveraging a specialized drug discovery and development platform, Neurosterix aspires to provide improved therapeutic options for underserved patient populations, ultimately aiming to enhance the quality of life for individuals affected by these challenging conditions.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

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.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

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.

Ocular Therapeutix, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for eye diseases using its proprietary bioresorbable hydrogel platform technology. The company offers several products, including ReSure Sealant, a hydrogel ophthalmic wound sealant designed to protect corneal incisions after cataract surgery, and DEXTENZA, an ophthalmic insert for the treatment of post-surgical ocular pain and inflammation, as well as allergic conjunctivitis and dry eye diseases. Ocular Therapeutix is also developing OTX-TP, an insert aimed at reducing intraocular pressure in glaucoma patients, and OTX-TIC, an intracameral implant for similar indications, along with OTX-TKI, an intravitreal implant targeting wet age-related macular degeneration. In addition, the company has preclinical programs focused on various ocular conditions. Ocular Therapeutix collaborates with Regeneron Pharmaceuticals to integrate its hydrogel technology with Regeneron's VEGF-targeting compounds for retinal disease treatments. Founded in 2006, the company is headquartered in Bedford, Massachusetts.

enGene, Inc. is a biotechnology company specializing in mucosal immunotherapy platforms aimed at treating inflammatory bowel disease and diabetes. The company has developed a non-integrating biopolymer-based nucleotide delivery technology that enables localized delivery of immune-modulating proteins to mucosal tissues, including the gastrointestinal tract, lung, and bladder. This innovative approach allows for the treatment of various immune disorders and supports systemic release of proteins from the gut to address conditions such as diabetes, anemia, and hemophilia. Founded in 1999 and based in Vancouver, Canada, enGene has formed a strategic alliance with Takeda Pharmaceutical Company Ltd. The company's platform facilitates the induction or suppression of protein expression levels, which can help regenerate physiologic, meal-regulated insulin secretion for diabetes patients.

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.

Apyx Medical Corp is a medical device company focused on energy technology, dedicated to developing, manufacturing, and marketing a variety of surgical and cosmetic products. The company is known for its innovative Renuvion cosmetic technology, which enables plastic and cosmetic surgeons to apply controlled heat to tissues, achieving desired aesthetic results with minimal invasiveness. Apyx Medical's flagship J-Plasma system utilizes a patented helium ionization process to generate a stable, focused beam of plasma, allowing for precise cutting, coagulation, and ablation of soft tissue while minimizing unintended damage to surrounding areas. Apyx Medical’s products are utilized in a range of settings, including doctor's offices, surgery centers, and hospitals, highlighting the company's commitment to enhancing surgical outcomes through advanced medical technologies.

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.

Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing therapies for idiopathic pulmonary fibrosis (IPF) and other severe respiratory diseases. The company is advancing inhaled formulations of pirfenidone, specifically AP01, which aims to enhance drug delivery to the lungs while potentially reducing side effects associated with oral treatments. This innovative approach seeks to improve the care and outcomes for patients suffering from these conditions. Established in 2011, Avalyn Pharma operates from its headquarters in Seattle, Washington, and maintains an additional office in San Diego, California. The company was previously known as Genoa Pharmaceuticals, Inc. before rebranding in July 2017.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

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.

A-Alpha Bio, Inc. is a biotechnology company based in Seattle, Washington, established in 2017. The company specializes in synthetic biology, offering a platform known as AlphaSeq, which assists pharmaceutical companies in the development of drugs that interact with multiple targets. A-Alpha Bio provides cell-based tools for high-throughput and quantitative measurements of protein interactions, facilitating target discovery, library screening, and preclinical drug characterization. Its proprietary technology enables the mapping of protein-protein interactions at a large scale, allowing for the identification and quantification of interactions among thousands of protein pairs simultaneously. By delivering detailed maps of these interactions, A-Alpha Bio helps researchers identify potential drug targets and understand disease mechanisms, thereby streamlining the therapeutic discovery process and improving the efficiency of drug development. The company comprises a team of experts in synthetic biology, structural biology, and next-generation sequencing, focused on overcoming challenges in the drug development pipeline.

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.

Satellos Bioscience is a private regenerative medicine company dedicated to developing innovative therapeutics aimed at stimulating or restoring muscle regeneration. The company is particularly focused on addressing severe disorders, with a key program centered on creating an oral therapeutic drug for Duchenne muscular dystrophy. This drug is designed to correct the dysregulation identified by Satellos as a fundamental cause of the disease's progressive nature. Through its research and development efforts, Satellos aims to provide effective treatments that enhance muscle regeneration and improve the quality of life for individuals affected by this debilitating condition.

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.

Apogee Therapeutics is a biotechnology company based in San Francisco, California, founded in 2022. The company focuses on developing differentiated biologics for the treatment of immunological and inflammatory disorders, specifically targeting atopic dermatitis and chronic obstructive pulmonary disease. Apogee Therapeutics aims to address high unmet medical needs by advancing novel therapies that utilize advanced antibody engineering to enhance therapeutic properties such as half-life. The company's key programs, APG777 and APG808, are designed to leverage established mechanisms of action to improve treatment outcomes for patients suffering from these conditions.

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.

Zenas BioPharma is a clinical-stage biopharmaceutical company based in Florida, dedicated to developing immune-based therapies for patients. The company's lead product candidate, obexelimab, is a bifunctional monoclonal antibody that targets both CD19 and FcγRIIb, which are found on B cells. This innovative approach aims to inhibit the activity of B cells involved in various autoimmune diseases while preserving their overall population, thus offering a potential therapeutic advantage over traditional treatments that deplete these cells.

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.

InSightec Ltd. specializes in developing and distributing the Exablate platform, which employs MR-guided focused ultrasound technology for non-invasive medical treatments in oncology and gynecology. The company offers solutions such as Exablate Neuro, designed for personalized thalamotomy to address movement disorders and pain without invasive surgery, and a thermal ablation technique for the treatment of uterine fibroids, which alleviates symptoms while minimizing damage to surrounding tissues. Founded in 1999 and headquartered in Tirat Carmel, Israel, InSightec has expanded its presence globally with operations in Europe, Asia, and the Americas. The Exablate system has received various regulatory approvals, including CE marks and FDA approval, and has been recognized with multiple innovation awards for its contributions to healthcare. In addition to its medical devices, InSightec provides clinical training, business support, maintenance, and customer services to healthcare facilities using its technology.

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.

Surf Bio is a preclinical biopharmaceutical company focused on developing innovative therapeutic solutions using a novel biotechnology platform. This platform centers around a next-generation surfactant designed to enhance drug effectiveness for a variety of medical conditions, including diabetes, oncology, infectious diseases, and gene therapy. By improving therapeutic applications, Surf Bio aims to provide healthcare organizations worldwide with access to new and more effective treatments for patients facing critical health issues.

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.

Neuron23, Inc. is a biotechnology company focused on developing therapies for genetic disorders, particularly neurological diseases such as Parkinson's and Alzheimer's. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence and genetic insights to enhance drug discovery. Neuron23's innovative platform integrates the latest advancements in human genetics with AI-driven methodologies to create targeted treatments for patients facing debilitating health challenges. By focusing on genetically defined conditions, Neuron23 aims to improve therapeutic options and outcomes for individuals affected by these disorders.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

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.

Ceptur Therapeutics is focused on developing innovative genetic medicines aimed at addressing diseases that currently lack effective treatments. The company utilizes its proprietary U1 Adaptor technology, which enables long-lasting and non-genotoxic silencing of disease-causing genes, particularly in challenging tissues beyond the liver and central nervous system. This technology addresses the limitations of existing gene silencing methods and holds promise for cancer treatment, allowing healthcare providers to offer new therapeutic options for patients with various types of cancers.

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.

Sermonix Pharmaceuticals is a biotechnology company based in Columbus, Ohio, founded in 2014. The company specializes in the development and commercialization of female-specific oncology products, focusing on late-stage women’s health treatments. Its primary product, lasofoxifene, is a selective estrogen receptor modulator designed for the treatment of vulvovaginal atrophy and the treatment and prevention of post-menopausal osteoporosis. Additionally, Sermonix is involved in developing pharmaceutical drugs aimed at treating ESR1-mutated metastatic breast and gynecological cancers, thereby addressing critical needs in women's health and 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.

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.

A-Alpha Bio, Inc. is a biotechnology company based in Seattle, Washington, established in 2017. The company specializes in synthetic biology, offering a platform known as AlphaSeq, which assists pharmaceutical companies in the development of drugs that interact with multiple targets. A-Alpha Bio provides cell-based tools for high-throughput and quantitative measurements of protein interactions, facilitating target discovery, library screening, and preclinical drug characterization. Its proprietary technology enables the mapping of protein-protein interactions at a large scale, allowing for the identification and quantification of interactions among thousands of protein pairs simultaneously. By delivering detailed maps of these interactions, A-Alpha Bio helps researchers identify potential drug targets and understand disease mechanisms, thereby streamlining the therapeutic discovery process and improving the efficiency of drug development. The company comprises a team of experts in synthetic biology, structural biology, and next-generation sequencing, focused on overcoming challenges in the drug development pipeline.

Zentera Therapeutics is a biopharmaceutical company.

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.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

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.

Absci is a drug and target discovery company that leverages deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. Its Integrated Drug Creation Platform streamlines the process of identifying novel drug targets, discovering optimal biotherapeutic candidates, and generating the necessary cell lines for manufacturing. By combining biologic drug discovery and cell line development into a single, efficient workflow, Absci collaborates with biotech and pharmaceutical innovators to develop next-generation protein-based drugs. These innovations include Bionic Proteins, which feature nonstandard amino acids, and other unique drug designs that may be unattainable through conventional methods. Absci's mission is to facilitate the development of improved medicines by effectively translating innovative ideas into viable drugs.

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.

Pipeline Therapeutics Inc. is a biotechnology company focused on developing and commercializing small molecules aimed at neuroregeneration, which includes promoting synaptogenesis, remyelination, and axonal repair. Based in San Diego, California, the company’s lead product candidate, PIPE-505, is a gamma secretase inhibitor designed to treat mild-to-moderate sensorineural hearing loss associated with cochlear synaptopathy. Additionally, Pipeline Therapeutics is advancing earlier-stage programs, such as PIPE-307, which target remyelination and axonal repair to address various neurological disorders, including multiple sclerosis. Founded in 2017, the company aims to harness the body's natural repair mechanisms to facilitate recovery from neurological damage.

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.

Earli is a company focused on developing innovative cancer treatment devices that utilize synthetic biomarkers for early cancer detection. The company's approach involves a novel gene therapy technique known as Synthetic Biopsy, which prompts cancer cells to produce synthetic, non-human biomarkers. These biomarkers can be detected through various means, including blood, urine, or breath, allowing for timely tracking and treatment of cancer. By facilitating the early identification of cancer when it is still homogeneous, Earli aims to significantly enhance survival rates. Their technology not only aids in detection but is also applicable for therapeutic interventions, thereby personalizing treatment and improving patient outcomes.

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.

Neuron23, Inc. is a biotechnology company focused on developing therapies for genetic disorders, particularly neurological diseases such as Parkinson's and Alzheimer's. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence and genetic insights to enhance drug discovery. Neuron23's innovative platform integrates the latest advancements in human genetics with AI-driven methodologies to create targeted treatments for patients facing debilitating health challenges. By focusing on genetically defined conditions, Neuron23 aims to improve therapeutic options and outcomes for individuals affected by these disorders.

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.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, focused on developing patient-specific immunotherapies to treat cancer. Founded in 2016, the company creates personalized vaccines and T cell therapies that harness the immune system to target and eliminate cancer cells. By identifying truncal tumor neo-antigens and other cancer-specific markers, Achilles Therapeutics aims to develop next-generation therapies that selectively attack tumors while sparing healthy tissues. Currently, the company is conducting two clinical trials: the CHIRON trial for patients with non-small-cell lung cancer and the THETIS trial for those with recurrent or metastatic melanoma.

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.