Janus Henderson Investors

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.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Ottimo Pharma specializes in developing innovative cancer therapies for solid tumors. Its primary focus is Jankistomig, a bi-functional antibody that targets both immune checkpoint inhibition and angiogenesis, two key pathways involved in cancer growth. By simultaneously addressing these pathways, Ottimo Pharma's approach offers healthcare professionals a dual-pronged strategy to potentially enhance treatment outcomes and reduce the burden of cancer on patients and the healthcare system.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

35Pharma is a biopharmaceutical company focused on designing and developing innovative biologics to address diseases with significant unmet medical needs, particularly pulmonary hypertension and musculoskeletal disorders. The company leverages its expertise in protein engineering and a comprehensive understanding of transforming growth factor (TGF)-beta structure-function relationships to create highly effective ligand traps. These advancements aim to facilitate the development of targeted biologics for treating patients suffering from cardio-pulmonary and metabolic diseases.

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.

Metsera is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for obesity and metabolic conditions. The company focuses on advancing a variety of oral and injectable treatment options, including incretin, non-incretin, and combination therapies. These treatments are designed to address multiple therapeutic targets, positioning Metsera to meet the evolving demands of the weight loss treatment landscape. Additionally, Metsera integrates proprietary health technology tools to provide personalized care, enhancing the effectiveness of its therapeutic offerings.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Alpha9 Theranostics is a biopharma company that focuses on radiotherapeutics for the treatment of solid and hematologic malignancies. The company is creating a strong pipeline of radiopharmaceuticals to target solid and hematologic malignancies. Its systematic approach to radiotherapeutic design is fueled by a toolbox of technologies and chemistries, which offers broad potential for expansion into a multitude of oncology targets. Alpha9 Theranostics was founded in 2019 and is headquartered in Vancouver, Canada.

Aktis Oncology is a biotechnology company focused on developing targeted radiopharmaceuticals for the treatment of various solid tumor cancers. The company has created proprietary platforms that generate tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered to achieve high penetration into tumors and prolonged residence time, allowing for effective tumor elimination while reducing side effects associated with treatment. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing the precision of cancer treatment.

Tenax Therapeutics, Inc. is a specialty pharmaceutical company dedicated to the identification, development, and commercialization of products aimed at addressing cardiovascular and pulmonary diseases with significant unmet medical needs. Headquartered in Morrisville, North Carolina, Tenax focuses on levosimendan, a pharmaceutical product designed to reduce morbidity and mortality in cardiac surgery patients at risk for low cardiac output syndrome. The company is also actively conducting a Phase 2 clinical trial to explore the efficacy of levosimendan in treating pulmonary hypertension associated with heart failure with preserved ejection fraction. Additionally, Tenax is involved in developing novel formulations of imatinib mesylate, a kinase inhibitor with the potential to serve as a disease-modifying therapy for pulmonary arterial hypertension. Founded in 1967, Tenax Therapeutics underscores its commitment to advancing treatments within these critical therapeutic areas.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

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.

Lycia Therapeutics is a biotechnology firm focused on discovering and developing first-in-class therapies. The company utilizes its innovative lysosomal targeting chimeras (LYTACs) platform to create therapeutics that target and degrade extracellular and membrane-bound proteins associated with challenging diseases, including cancer and autoimmune disorders. By addressing these difficult-to-treat conditions, Lycia Therapeutics aims to provide medical practitioners with effective treatment options to improve patient outcomes.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops therapies for Batten disease, cystic fibrosis, and genetic eye disorders. Additionally, Abeona Therapeutics is advancing the AIM Vector Platform, which includes over 100 next-generation AAV capsids for delivering gene therapies to various organs. The company operates a manufacturing facility producing therapies and vectors for preclinical and clinical studies.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, that specializes in developing therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating innovative treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact millions of individuals. Eliem utilizes its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, including its lead candidate, ETX-155, aimed at addressing major depressive disorder and focal onset seizures. The company's mission centers on meeting unmet medical needs and improving patients' quality of life through its novel therapies.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cells for the treatment of cancer and autoimmune diseases. The company aims to enhance the efficacy of cell therapy by leveraging the inherent ability of NK cells to identify and eliminate abnormal cells without the need for genetic alteration. This approach allows for a broader application across various hematologic and solid tumor malignancies while potentially minimizing side effects associated with traditional T-cell therapies. Nkarta combines its NK expansion platform technology with proprietary cell engineering methods to produce a substantial supply of NK cells, enhancing their ability to recognize therapeutic targets and improving their persistence in the body for sustained activity. Through this innovative strategy, Nkarta seeks to provide more potent, well-tolerated, and rapidly available therapies for patients facing diverse cancer types.

MindMed provides psychedelic-inspired medicines and therapies for substance abuse disorders and other mental illnesses. The company is assembling a compelling drug development pipeline of innovative treatments based on psychedelic substances including Psilocybin, LSD, MDMA, DMT, and an Ibogaine derivative, 18-MC.

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.

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.

Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for severe autoimmune diseases through targeted therapies. The company's lead candidate, DNTH103, is a monoclonal antibody designed to selectively inhibit a specific component of the immune system, offering potential advantages over existing complement therapies. This treatment aims to improve safety and efficacy for patients suffering from serious autoimmune and inflammatory conditions. Dianthus is committed to advancing its pipeline of next-generation complement therapeutics, supported by a team of experienced professionals in the biotech and pharmaceutical sectors. The company envisions that DNTH103 could be administered as a self-injection for certain patients, enhancing convenience and accessibility in treatment.

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.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies to treat chronic inflammatory and autoimmune diseases. The company's pipeline includes three small molecule products. The lead program, IMU-838, is a selective immune modulator that blocks the enzyme DHODH, inhibiting the metabolism of activated immune cells and exhibiting antiviral effects. It is being developed for multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. IMU-935, a selective inverse agonist of the transcription factor RORγt, targets psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Additionally, IMU-856 aims to restore intestinal barrier function for diseases involving bowel barrier dysfunction.

AN2 Therapeutics, Inc. is a biopharmaceutical company based in Menlo Park, California, founded in 2017. The company is dedicated to the research, development, and commercialization of innovative medicines aimed at addressing infectious diseases. AN2 Therapeutics is focused on developing epetraborole, a once-daily oral treatment for patients suffering from chronic non-tuberculous mycobacterial lung disease, which represents a significant unmet medical need. Additionally, the company has established a strategic partnership with Brii Biosciences to enhance its development efforts.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company based in Shanghai, China, focused on discovering and developing cell therapies for cancer treatment. The company’s lead product candidates include GC012F, a dual-target autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia and is also being investigated for relapsed or refractory B cell non-Hodgkin’s lymphoma. Additionally, Gracell is developing GC007F for B cell non-Hodgkin's lymphoma and GC027, an allogeneic CAR-T product candidate for adult T cell acute lymphoblastic leukemia. The company also has GC007g, a donor-derived therapy for relapsed or refractory B cell acute lymphoblastic leukemia, and a pipeline of earlier stage candidates targeting other cancer types, including ovarian cancer and breast cancer. Founded in 2017, Gracell aims to provide innovative cellular therapeutics that enhance treatment options for patients suffering from various hematological malignancies and solid tumors.

CG Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative immunotherapies for cancer treatment, particularly focusing on bladder cancer. The company specializes in oncolytic immunotherapies, with its lead candidate, cretostimogene grenadenorepvec, designed as a targeted, intravesical immunotherapy. Currently, CG Oncology is conducting multiple clinical trials, including two phase three trials aimed at high-risk patients with non-muscle invasive bladder cancer (NMIBC) who are unresponsive to BCG therapy, as well as an additional phase two study in combination with a checkpoint inhibitor. The company aims to provide effective bladder-sparing treatment options for patients while advancing the development of its proprietary therapies.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.

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.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Compass Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative antibody therapeutics for the treatment of solid tumors and hematological malignancies. Founded in 2014 and headquartered in Cambridge, Massachusetts, the company utilizes its proprietary StitchMabs and common light-chain platforms to discover and create multispecific antibody combinations. Its lead product candidate, CTX-471, is an agonistic monoclonal antibody targeting CD137, currently undergoing Phase 1 clinical trials in patients with prior PD-1/PD-L1 checkpoint inhibitor treatment. Another significant candidate, CTX-009, is a bispecific antibody that inhibits DLL4-mediated Notch signaling and VEGF-A signaling, having completed a Phase 1 dose escalation study. Additionally, CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, is in the IND-enabling stage. Through its innovative approaches, Compass Therapeutics aims to enhance immune engagement and improve therapeutic outcomes for cancer patients.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Enliven Therapeutics is a clinical-stage precision oncology company based in Boulder, Colorado, focused on developing innovative small molecule therapies to enhance patient outcomes. Established in July 2019 by Sam Kintz, Joe Lyssikatos, and Anish Patel, the company employs a unique discovery process that leverages clinically validated biological targets alongside advanced chemistry. This methodology aims to overcome the limitations of current therapies by creating potentially first-in-class treatments that address critical challenges such as tolerability, combinability, resistance, and disease progression, particularly in cases involving brain metastases. Enliven's product pipeline includes ELVN-001, a selective small molecule kinase inhibitor targeting the BCR-ABL gene fusion in chronic myeloid leukemia, and ELVN-002, an irreversible HER2 inhibitor designed to penetrate the central nervous system and act against various HER2 variants.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

Quell's vision is to become a leading engineered Treg cell therapy company. The company will seek to develop next generation engineered T-regulatory cell (Treg) therapies for a range of solid organ transplant and autoimmune conditions. Quell has been founded by Syncona in partnership with six leading experts in the Treg field, cell engineering, solid organ transplantation and autoimmune diseases: Giovanna Lombardi, Professor of Human Transplant Immunology at Kings College London (KCL), Alberto Sanchez-Fueyo, Professor of Hepatology in the Institute of Liver Studies at KCL, Hans Stauss, Professor of Tumour Immunology and Director of the Institute of Immunity and Transplantation at University College London (UCL), Emma Morris, Professor of Clinical Cell & Gene Therapy and Inflammation, Immunity and Immunotherapeutics Theme Director, National Institute for Health Research University College London Hospitals Biomedical Research Centre, Marc Martinez-Llordella, Senior Lecturer in the Institute of Liver Studies at KCL and Elmar Jaeckel, Co-Leader Liver Transplant program MHH, Group Leader "Immune tolerance" in the Department of Gastroenterology, Hepatology and Endocrinology at Hannover Medical School.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

Asher Biotherapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2019. The company specializes in the development of immunotherapy drugs aimed at effectively treating cancer. Its innovative therapies are designed to enhance the immune system's ability to target and eliminate cancer cells while minimizing side effects. This focus allows healthcare providers to deliver effective treatments to patients in a shorter timeframe, aiming to improve overall outcomes in cancer care.

Synthekine Inc. is a biotechnology company focused on developing innovative cytokine therapies and immunotherapies for cancer and autoimmune disorders. Established in 2018 and located in Menlo Park, California, the company employs advanced platforms such as engineered partial agonists and orthogonal cell therapies. Its Synthekine Platform utilizes surrogate agonists instead of traditional mutant cytokines, allowing for the creation of therapeutics that enhance efficacy while minimizing side effects. The product pipeline includes STK-009, an orthogonal ligand, and SYNCAR-001, a CAR-T therapy targeting CD-19, along with STK-012, which consists of partial agonists of IL-2. Synthekine aims to revolutionize treatment paradigms by harnessing the principles of cytokine partial agonism and immunological specificity, leading to novel immunotherapies that improve patient outcomes.

BioTheryX, Inc. is a clinical-stage biopharmaceutical company based in Chappaqua, New York, focused on developing innovative therapies that restore protein homeostasis through targeted protein degradation. The company specializes in Protein Homeostatic Modulators and small molecule targeted protein degraders (TPDs), which utilize a unique mechanism involving molecular glues to regulate protein levels within the body. Their proprietary PRODEGY platform is built on a library of Cereblon binders, enabling the design of bifunctional and hybrid degraders aimed at treating various diseases, particularly cancer. BioTheryX's lead candidate, BTX-A51, is an oral small molecule and multi-kinase inhibitor targeting specific leukemic stem cells and oncogenic transcription factors. Established in 2007, BioTheryX aims to address significant unmet medical needs by leveraging its expertise in protein modulation and degradation.

Magenta Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapies to enhance stem cell transplantation for patients with blood cancers, genetic diseases, and autoimmune disorders. The company aims to improve the safety and efficacy of stem cell transplants, which are currently high-risk procedures often considered only as a last resort. Its pipeline includes several key product candidates, such as targeted antibody-drug conjugates for transplant conditioning, a novel stem cell mobilization agent, and allogeneic stem cell therapies. Magenta is also exploring collaborations to assess the effectiveness of its therapies in combination with existing treatments, such as gene therapies for sickle cell disease. Founded in 2015 and headquartered in Cambridge, Massachusetts, Magenta Therapeutics seeks to expand the curative potential of stem cell therapies to a broader patient population by addressing the toxicity and long-term side effects associated with current transplant methods.

Nuvalent is a clinical-stage biotechnology company focused on developing targeted therapies for cancer, specifically aimed at clinically validated kinase targets. With a strong foundation in structure-based design, Nuvalent creates small molecules that exhibit high selectivity, aiming to address issues of resistance and minimize side effects associated with conventional treatments. The company's pipeline includes lead programs such as NVL-520 for ROS1-positive non-small cell lung cancer (NSCLC) and NVL-655 for ALK-positive NSCLC, as well as several discovery-stage initiatives. All operations are conducted within the United States, where Nuvalent seeks to enhance treatment options for patients with significant unmet medical needs.

TwinStrand Biosciences, established in 2015 and based in Bellevue, Washington, specializes in advancing DNA sequencing technology. The company's core innovation, Duplex Sequencing, enhances sequencing accuracy by over 10,000 times, enabling the detection of previously invisible mutations. This breakthrough technology has potential applications across various sectors, including oncology, infectious disease diagnosis, organ transplantation, reproductive health, genetic testing, and forensics.

NeoGenomics Laboratories operates a network of cancer-focused testing facilities in the United States, Switzerland, and Singapore. The company is divided into two main segments: Clinical Services and Pharma Services. It provides a range of genetic and molecular testing services, including cytogenetics, fluorescence in-situ hybridization, flow cytometry, immunohistochemistry, and molecular testing, aimed at helping clinicians and researchers understand cancer at a genetic level. Additionally, NeoGenomics offers morphologic analysis for diagnosis and supports pharmaceutical companies in their oncology programs through testing services that facilitate drug development and clinical trials. The company's collaboration with Inivata Limited for the commercialization of the InVisionFirst-Lung liquid biopsy test exemplifies its commitment to advancing cancer diagnostics. Founded in 2001 and headquartered in Fort Myers, Florida, NeoGenomics serves hospitals, pathologists, oncologists, academic centers, and other clinical laboratories, enhancing cancer care through innovative testing solutions.

Janux Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative immunotherapies aimed at generating immune responses to combat tumors while preserving healthy tissue. Utilizing its proprietary tumor-activated T Cell Engager platform technology, Janux targets all three stages of the anti-tumor immune response. The company's approach is designed to enhance the efficacy of cancer treatments by specifically engaging the immune system to identify and eliminate cancer cells.

ValenzaBio is focused on developing safe and effective treatment options for patients suffering from autoimmune and inflammatory diseases. The company utilizes biomarker-driven approaches to inform its research and clinical development. Its pipeline features several monoclonal antibodies designed to target surface antigens, aiming to reduce harmful subpopulations of autoreactive cells. ValenzaBio's most advanced program has successfully reached Phase 1 proof-of-concept in patients, while other preclinical programs are underway, exploring differentiated mechanisms of action. The company's mission is to address the needs of patients who lack effective treatment alternatives.

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.

Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, that specializes in developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in cancer therapies through its proprietary SNÅP platform, which facilitates rapid and precise drug design by generating iterative molecular snapshots. Tyra is particularly focused on creating selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. The company's lead product candidate, TYRA-300, specifically targets FGFR3 and is initially aimed at patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through its innovative approach, Tyra Biosciences seeks to improve treatment outcomes for cancer patients by providing alternatives to existing therapies.

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.

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.

Amunix Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to discovering and developing innovative protein and peptide therapeutics aimed at treating cancer. The company employs several proprietary platforms, including XTEN for extending drug half-life, XPAT for analyzing protease activity in the tumor microenvironment, and ProTIA, which focuses on immuno-oncology therapeutics. Amunix's product pipeline includes prodrugs and transformative T-cell engagers designed to activate the immune system against solid tumors. The company collaborates with various academic, biotechnology, and pharmaceutical sectors, maintaining strategic partnerships with organizations such as Janssen and Biogen-Idec. Founded in 2006 and headquartered in Mountain View, California, Amunix strives to deliver breakthroughs that can harness the immune system to combat cancer more effectively.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Viracta Therapeutics, Inc. is a clinical-stage drug development company based in Cardiff, California, that specializes in precision oncology focused on virus-associated malignancies. The company utilizes a viral activation therapy platform to develop treatments for cancers linked to the Epstein-Barr Virus (EBV) and other virus-related diseases. Its lead investigational drug, nanatinostat, is being evaluated in combination with the antiviral valganciclovir as a potential oral therapy for EBV-positive lymphoma, including post-transplant lymphoproliferative disorder, plasmablastic lymphoma, and angioimmunoblastic T cell lymphoma. Viracta is also exploring the application of this approach to other EBV-associated conditions, such as nasopharyngeal carcinoma and gastric carcinoma. Founded in 2007 and rebranded from HemaQuest Pharmaceuticals in 2015, Viracta Therapeutics is actively conducting multiple clinical trials to advance its therapeutic candidates.

Centessa Pharmaceuticals is a clinical-stage biopharmaceutical company focused on innovating the drug development process through an asset-centric research and development model. The company manages a diverse portfolio of therapeutics across several therapeutic areas, including SerpinPC for Hemophilia A and B, LB101 for solid tumors, ORX750 for Narcolepsy Type 1 and other sleep disorders, and MGX292 for pulmonary arterial hypertension. Each therapeutic program is developed by a dedicated subsidiary, leveraging a centralized infrastructure and experienced management team to ensure efficient advancement of its pipeline assets.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing innovative therapies that leverage the biology of DNA repair for cancer treatment and synthetic lethality in autoimmune diseases. The company utilizes its expertise in DNA damage response biology to create a pipeline of small molecule therapeutics aimed at selectively targeting vulnerabilities in cancer cells and inducing their self-destruction through DNA damage overload. Cyteir's lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, which is crucial for repairing double-strand DNA breaks. The company is dedicated to addressing a variety of hematological malignancies, solid tumors, and chronic autoimmune disorders through its integrated drug development platform. Founded in 2012, Cyteir is committed to advancing next-generation therapies that can improve patient outcomes in challenging disease states.

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.

HemoShear Therapeutics, LLC is dedicated to discovering novel biological targets and drug treatments for rare metabolic disorders, particularly focusing on inherited conditions such as Organic Acidemias and Nonalcoholic Steatohepatitis, a chronic liver disease. The company employs its proprietary REVEAL-Tx platform to replicate human disease biology, enabling the development and validation of new therapies. By creating human-relevant systems, HemoShear generates significant human response data that aids pharmaceutical and biotechnology partners in making informed decisions and reducing the risk of costly failures in drug development. Founded in 2008 and based in Charlottesville, Virginia, HemoShear Therapeutics aims to improve the quality of life for patients suffering from metabolic defects that disrupt essential biochemical processes.

Inivata Limited is a clinical cancer genomics company based in Cambridge, United Kingdom, established in 2014. The company specializes in the analysis of circulating tumor DNA (ctDNA) to enhance cancer testing and treatment for oncologists and their patients. Inivata's innovative approach utilizes TAm-Seq technology to detect and analyze genomic material from a patient's cell-free ctDNA, collected through non-invasive blood samples, known as liquid biopsies. This method revolutionizes cancer detection and monitoring by providing a less invasive alternative to traditional biopsies. The company's focus is on enabling clinicians to make more informed treatment decisions by monitoring response to therapies and detecting potential relapses in cancer patients. Inivata has also formed a strategic collaboration with NeoGenomics, Inc.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

NewAmsterdam Pharma, established in 2019 and headquartered in Naarden, the Netherlands, is a late-stage biopharmaceutical company dedicated to improving patient care in cardio-metabolic disease populations. The company's primary focus is the development of transformative therapies, with its lead product, obicetrapib, being a next-generation, oral, low-dose CETP inhibitor designed to lower LDL-C levels more effectively than current treatments, particularly for patients with statin intolerance or Arteriosclerotic Cardiovascular Disease/Familial Hypercholesterolemia (ASCVD/FH) on maximally-tolerated statin therapy.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.

Biomea Fusion is a preclinical-stage biopharmaceutical company dedicated to advancing precision medicine for cancer treatment. The company's focus is on the discovery and development of irreversible small molecule drugs that target specific genetic alterations associated with tumor growth. Its lead product candidate, BMF-219, is designed as a potent and selective irreversible inhibitor of menin, a key transcriptional regulator involved in oncogenic signaling across various cancers. Biomea Fusion aims to expedite the development of these innovative therapies to provide highly effective treatment options for patients with genetically defined cancers.

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.

Elevation Oncology, Inc. is a biotechnology company dedicated to developing precision medicines for patients with genomically defined cancers. The company focuses on creating targeted therapeutics that inhibit specific genetic alterations known to drive cancer progression. Its lead drug candidate, seribantumab, targets neuregulin-1 (NRG1) fusions, which are rare genomic alterations that can be treated through targeted HER3 inhibition. Currently, seribantumab is undergoing clinical testing in the Phase 2 CRESTONE study, which evaluates its efficacy in patients with solid tumors of any origin that harbor NRG1 fusions. Founded in 2019 and based in New York, Elevation Oncology aims to make genomic tests actionable by selectively developing drugs that address these specific alterations in cancer patients.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

Flame Biosciences, Inc. is a clinical-stage biotechnology company based in New York, dedicated to the research, development, and commercialization of innovative therapies targeting cancer and inflammatory diseases. The company is focused on creating transformative treatments, including FL-101, which addresses lung cancers associated with inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Established in 2017, Flame Biosciences seeks to improve the quality of life for individuals suffering from debilitating and life-threatening conditions through its advanced therapeutic solutions.

Galecto is a clinical-stage biotechnology company focused on developing small-molecule therapeutics to address severe diseases, particularly fibrosis and cancer. Established in 2011, Galecto builds on over a decade of research into the roles of galectin-3 and lysyl oxidase-like 2 (LOXL2) in fibrotic diseases. The company's lead product candidate, GB0139, is an inhaled galectin-3 inhibitor specifically designed for treating severe fibrotic lung diseases, such as idiopathic pulmonary fibrosis (IPF), where there is a significant unmet medical need. Additionally, Galecto is advancing GB1211, an oral selective galectin-3 inhibitor aimed at addressing fibrosis related to non-alcoholic steatohepatitis. With a robust patent portfolio, Galecto leverages its unique therapeutic platform to target the underlying biological processes associated with fibrosis and related diseases.

Eledon Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in Irvine, California, specializing in the development of innovative treatments for patients with disorders of the ear, nose, and throat, as well as for those undergoing organ or cellular transplantation and individuals with autoimmune and neurodegenerative diseases. The company’s lead product, OP0201, is a surfactant-based nasal aerosol designed for patients at risk for or suffering from otitis media. Additionally, Eledon has developed a foam-based drug delivery technology, OP0101 and OP0102, aimed at delivering medications to the ear, nose, and sinus cavities. The company also focuses on the CD40L pathway, with its main compound in development, tegoprubart, being an IgG1 anti-CD40L antibody that shows promise for treating autoimmune diseases and supporting organ transplants. Eledon Pharmaceuticals was formerly known as Novus Therapeutics, Inc. before its rebranding in January 2021.

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.

Kinnate Biopharma Inc. is a biopharmaceutical company based in San Diego, California, focused on developing small molecule kinase inhibitors for the treatment of genomically defined cancers. Founded in 2018, the company aims to create precision oncology therapeutics to address the needs of underserved patient populations. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is working on KIN003, which targets specific alterations in the FGFR2 and FGFR3 genes, and has other small molecule research programs, including a Cyclin-Dependent Kinase 12 inhibitor. Kinnate Biopharma employs expertise in structure-based drug discovery and translational research to advance its pipeline of candidates aimed at treating cancers associated with specific oncogenic alterations.

Chinook Therapeutics, Inc. is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently prepared for Phase III trials targeting IgA nephropathy and other primary glomerular diseases. In addition to atrasentan, Chinook is developing BION-1301, an investigational anti-APRIL monoclonal antibody evaluated in a Phase Ib trial for IgA nephropathy, and CHK-336, a preclinical candidate aimed at treating an undisclosed ultra-orphan kidney disease. Chinook Therapeutics is also engaged in research programs addressing other rare and severe chronic kidney conditions, including polycystic kidney disease.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Cybin is a biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for patients suffering from classical complement-mediated disorders affecting the body, brain, and eye. The company's research is centered around its platform technology that targets classical complement-mediated autoimmune and neurodegenerative diseases associated with the abnormal activation of C1q, a key molecule in the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody intended for intravenous use in treating autoimmune and neurodegenerative disorders. Another candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration aimed at neurodegenerative ophthalmic conditions. The company employs a disciplined, biomarker-driven development strategy to ensure its therapies effectively engage the target at tolerable doses within the intended patient tissues, while also exploring additional indications within both orphan and larger market segments.

Shattuck Labs, Inc. is a clinical-stage biotechnology company based in Austin, Texas, focused on developing innovative therapeutics for cancer and autoimmune diseases. The company utilizes its proprietary Agonist Redirected Checkpoint (ARC) platform, which involves dual-function fusion proteins designed to enhance immune responses against tumors. Its lead product candidate, SL-172154, is undergoing Phase 1 clinical trials for the treatment of ovarian cancer, while another candidate, SL-279252, is being developed in collaboration with Takeda Pharmaceuticals and is also in Phase 1 trials for advanced solid tumors and lymphoma. Shattuck Labs aims to address a variety of cancer types through its research in immuno-oncology, leveraging its technology to promote effective immune responses and improve patient outcomes. Founded in 2016, the company is committed to advancing the field of biologic medicine.

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.

Everest Medicines Limited is a clinical-stage biopharmaceutical company based in Shanghai, China, founded in 2017. The company focuses on licensing, developing, and commercializing innovative therapies to meet critical unmet medical needs in Greater China and other Asia Pacific markets. Its diverse portfolio includes eight clinical-stage drug candidates aimed at treating various conditions such as oncology, immunology, cardio-renal diseases, and infectious diseases. Everest Medicines aims to partner with innovative global companies to bring transformative pharmaceutical solutions to the Emerging Market, leveraging its expertise in clinical development, regulatory affairs, and business operations. The company's pipeline features several notable candidates, including Nefecon, EVER001, PTX-COVID19-B, and Eravacycline, among others.

iTeos Therapeutics is a biopharmaceutical company dedicated to developing immuno-oncology therapies for cancer patients. Founded in 2011, the company operates from headquarters in Cambridge, Massachusetts, and Gosselies, Belgium. iTeos focuses on discovering and advancing small molecule immunomodulators that target the tumor microenvironment, aiming to enhance the body's immune response against cancer. Its pipeline includes EOS-850, a small molecule antagonist of the adenosine A2a receptor, and EOS-448, an antagonist of TIGIT, both currently in Phase 1/2a clinical trials. iTeos' approach is built on the expertise of Ludwig Cancer Research, aiming to improve clinical outcomes by restoring the immune system's ability to fight cancer.

Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, that specializes in the discovery and development of novel small molecule therapeutics aimed at selectively degrading disease-causing proteins. The company utilizes a proprietary platform for targeted protein degradation, leveraging the body’s natural protein recycling system. Kymera is developing several programs, including the IRAK4 program for immunology-inflammation diseases such as hidradenitis suppurativa and rheumatoid arthritis, the IRAKIMiD program to address MYD88-mutated diffuse large B cell lymphoma, and the STAT3 program targeting hematologic malignancies, solid tumors, and autoimmune diseases. Founded in 2015, Kymera Therapeutics is pioneering new approaches to treat diseases that have previously been considered untreatable, focusing on previously inaccessible therapeutic targets.

Vaxcyte, Inc. is a biotechnology company focused on developing innovative vaccines to prevent and treat infectious diseases globally. The company’s lead candidate, VAX-24, is a 24-valent investigational pneumococcal conjugate vaccine. Additionally, Vaxcyte is advancing VAX-XP, which targets emerging strains that contribute to invasive pneumococcal disease and antibiotic resistance. Other notable candidates include VAX-A1, designed to protect against Group A Streptococcus infections, and VAX-PG, a therapeutic vaccine aimed at treating periodontal disease caused by Porphyromonas gingivalis. Vaxcyte utilizes a proprietary cell-free protein synthesis platform that enables the creation of protein carriers and antigens, enhancing vaccine development beyond conventional methods. Founded in 2013 and headquartered in Foster City, California, the company was previously known as SutroVax, Inc. before rebranding in May 2020.

ALX Oncology is a clinical-stage immuno-oncology company based in Burlingame, California, established in 2015. The company specializes in developing innovative therapies aimed at treating various forms of cancer, with a particular focus on its lead product candidate, ALX148. This therapeutic agent is designed to block the CD47 protein, which cancer cells exploit to evade the immune system. ALX148 is currently being investigated for its efficacy in treating myelodysplastic syndromes, acute myeloid leukemia, and several solid tumor types, including head and neck squamous cell carcinoma and certain gastric cancers. The company leverages advanced protein engineering technologies to enhance the affinity of its CD47 blockers, aiming to minimize hematologic toxicities often associated with existing treatments. ALX Oncology's commitment to improving cancer therapies is rooted in pioneering research conducted by its founders at Stanford University.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and headquartered in South San Francisco, California. The company specializes in developing innovative therapeutics to address unmet medical needs in viral and liver diseases, particularly targeting chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH). Its lead drug candidate, ALG-010133, is a synthetic oligonucleotide currently undergoing Phase I clinical trials for CHB. Aligos is also advancing several other candidates, including ALG-000184, a capsid assembly modulator for CHB; ALG-020572, an oligonucleotide for CHB; ALG-125097, an siRNA drug candidate for CHB; and ALG-055009, a small molecule THR-ß agonist for NASH. The company's strategy leverages the extensive expertise of its team in liver disease and viral hepatitis to develop targeted antiviral therapies and expedite its drug development pipeline.

Forma Therapeutics, a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, specializes in developing and commercializing novel therapeutics for rare hematologic diseases and cancers. Its pipeline includes FT-4202 in Phase 1 trials for sickle cell disease, FT-7051 for metastatic castration-resistant prostate cancer, FT-2102 in Phase 2 trials for relapsed/refractory AML and exploratory Phase 1 trials for glioma, and selective fatty acid synthase inhibitors FT-4101 and FT-8225. Forma's approach integrates innovative drug discovery technologies and oncology expertise to target high-value, challenging drug targets validated through genomic medicine.

IGM Biosciences is a clinical-stage biotechnology company focused on the research and development of engineered Immunoglobulin M (IgM) antibodies primarily for cancer treatment. The company’s lead product candidate, IGM-2323, is a bispecific IgM antibody targeting CD20 and CD3, currently undergoing Phase 1 clinical trials for relapsed or refractory B cell Non-Hodgkin's lymphoma. Additionally, IGM is developing IGM-8444, an IgM antibody that targets Death Receptor 5, and IGM-7354, a bispecific IgM antibody designed to deliver interleukin-15 cytokines to PD-L1 expressing cells, both aimed at treating various malignancies. The company’s proprietary IgM antibody technology platform is also applicable for developing therapies for infectious diseases and autoimmune conditions. IGM Biosciences has formed collaborations with other firms to explore new therapeutic options, including antibodies targeting SARS-CoV-2. Established in 1993 and headquartered in Mountain View, California, IGM Biosciences was previously known as Palingen, Inc. before rebranding in 2010.

Phathom Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Buffalo Grove, Illinois, focused on developing and commercializing innovative treatments for gastrointestinal diseases. Established in 2018 and launched in 2019, the company is advancing its lead drug candidate, vonoprazan, a potassium-competitive acid blocker (P-CAB) that effectively inhibits acid secretion in the stomach. Vonoprazan is currently undergoing Phase III clinical trials for the treatment of erosive gastroesophageal reflux disease and is also being studied in combination with antibiotics for the treatment of Helicobacter pylori infection. The company aims to address significant unmet medical needs in patients suffering from acid-related disorders and has assembled a team of experts in the gastrointestinal and pharmaceutical fields to drive its development efforts.

Aprea is a biotech company focusing on the discovery and development of novel anticancer compounds reactivating the tumor suppressor protein p53. The Company's lead program, APR-246, is a first-in-class small molecule drug candidate in clinical development. Aprea Therapeutics was established in 2003 in Stockholms Lan, Sweden by Klas Wiman and Galina Selivanova.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

Akero Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, that specializes in developing treatments for serious metabolic diseases, particularly nonalcoholic steatohepatitis (NASH). Founded in 2017 and formerly known as Pippin Pharmaceuticals, the company is dedicated to addressing the significant unmet medical needs associated with NASH, a condition characterized by liver inflammation and damage, which can lead to severe complications such as fibrosis and liver failure. Akero's lead product candidate, AKR-001, is a long-acting Fc fusion modified FGF21 protein designed to restore metabolic balance by targeting both liver and adipose tissues, thereby reducing liver fat and suppressing inflammation and fibrosis. This innovative approach builds upon two decades of research into FGF21 biology, positioning Akero as a frontrunner in the quest for effective NASH therapies.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

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.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

Lexeo Therapeutics is a biotechnology company specializing in adeno-associated virus (AAV)-mediated therapies for both rare and common diseases caused by single gene mutations. Headquartered in New York, the company maintains an integrated pipeline developed in collaboration with Weill Cornell Medicine's Department of Genetic Medicine. Lexeo focuses on advancing its clinical programs towards commercialization while continuing to research new therapies for various patient populations and unmet medical needs.