Wellington Management

Atsena Therapeutics is a clinical-stage company focused on developing gene therapies for inherited retinal diseases. Utilizing an adeno-associated virus (AAV) platform, including a novel spreading capsid, Atsena's clinical programs aim to prevent blindness caused by genetic mutations.

MedinCell S.A. is a pharmaceutical company focused on developing innovative therapeutic solutions through its proprietary BEPO technology, which facilitates long-acting injectable products utilizing biodegradable polymers and a biocompatible solvent. Based in Jacou, France, the company is engaged in ongoing research and development to optimize the efficiency and accessibility of medical treatments across various therapeutic areas. MedinCell's product pipeline includes several candidates, such as mdc-IRM, a subcutaneous injection for schizophrenia currently in Phase III trials, and mdc-CWM, an intra-articular injection aimed at treating pain and inflammation. Additionally, the company is exploring formulations like mdc-WWM for contraception and mdc-ANG for central nervous system disorders, alongside other candidates targeting pain management and organ transplant applications. Through its advancements in controlled, long-acting injectables, MedinCell aims to enhance treatment outcomes for patients.

Helicore Biopharma is a biopharmaceutical company dedicated to developing innovative treatments for obesity and related metabolic disorders. Its portfolio includes novel therapeutics based on glucose-dependent insulinotropic peptide (GIP) antagonism and monoclonal antibodies that bind circulating GIP ligands, forming a modular platform for anti-obesity medicines. The company aims to help patients with obesity and related conditions achieve significant weight loss.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Metsera is a clinical-stage biopharmaceutical company specializing in developing innovative therapies for obesity and metabolic conditions. It focuses on advancing oral and injectable treatments, integrating proprietary health technology tools for personalized care.

Day One Biopharmaceuticals is a clinical-stage biotechnology company focused on developing targeted therapies for genetically defined cancers across all ages, with an initial emphasis on pediatric patients. The company aims to bring effective cancer treatments to families by advancing therapies that can benefit both children and adults, prioritizing programs that can progress to the market rapidly. Day One pursues licensing or acquisition of promising products from research institutions and industry partners to expand its pipeline. Its lead candidate, DAY101, is an oral, brain-penetrant, highly selective type II pan-RAF kinase inhibitor designed to target cancers driven by the RAF signaling pathway. By applying insights from childhood cancer biology, the company seeks to address unmet needs in oncology and broaden access to precision therapies.

Savara is an orphan lung disease company. Savara’s pipeline comprises Molgradex, an inhaled granulocyte-macrophage colony-stimulating factor, or GM-CSF, in Phase 3 development for autoimmune pulmonary alveolar proteinosis, or aPAP, in Phase 2a development for nontuberculous mycobacteria, or NTM, lung infection, and in preparation for Phase 2a development in cystic fibrosis, or CF, affected individuals with chronic NTM lung infection; and AeroVanc, a Phase 3 stage inhaled vancomycin for treatment of persistent methicillin resistant staphylococcus aureus, or MRSA, lung infection in CF. Savara’s strategy involves expanding its pipeline of potentially best-in-class products through indication expansion, strategic development partnerships and product acquisitions, with the goal of becoming a leading company in its field. The most recent acquisition is aerosolized amikacin/fosfomycin, a Phase 2 ready, proprietary combination antibiotic, which has demonstrated potent and broad-spectrum antibacterial activity against highly drug resistant pathogens. Savara’s management team has significant experience in orphan drug development and pulmonary medicine, identifying unmet needs, developing and acquiring new product candidates, and effectively advancing them to approvals and commercialization.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Bluejay Therapeutics is a private biopharmaceutical company focused on developing curative treatments for infectious and liver diseases. Its initial program targets chronic hepatitis B, a widespread infection with unmet medical need, using two approaches: fully human IgG1 monoclonal antibodies against hepatitis B surface antigen and oral small-molecule inhibitors of HBsAg. The aim is to reduce viral surface antigen and restore adaptive immunity, potentially achieving a functional cure for HBV and enabling patients to regain immune control of infection. The company’s work also contemplates extending its approach to other viral liver diseases, including chronic hepatitis D.

Zenas BioPharma is a clinical-stage biopharmaceutical company focused on developing immune-based therapies for autoimmune and immune-mediated diseases. Based in Florida, United States, the company advances biologic programs and aims to modulate B cell–driven pathology. Its lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb on B cells, thereby inhibiting harmful immune activity without depleting B cells.

BridgeBio Oncology Therapeutics is a biopharmaceutical company specializing in the development of precision oncology therapeutics. It focuses on targeting RAS-dependent cancers, leveraging a profound understanding of RAS signaling biology to create drugs that selectively inhibit RAS-driven PI3K activation and a wide spectrum of KRAS mutants. The company aims to provide patients with tailored treatments that address both active and inactive cancer-driving states.

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.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Avalyn Pharma is a biopharmaceutical company focused on developing therapies for severe respiratory diseases, notably idiopathic pulmonary fibrosis (IPF). Established in 2011, the company is headquartered in Seattle, Washington.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

Upstream Bio is a clinical-stage biotechnology company focused on developing antibody therapies for severe respiratory disorders. Its lead product, Verekitug, targets the receptor for Thymic Stromal Lymphopoietin (TSLP), a cytokine involved in inflammatory responses.

Alkeus Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston that specializes in developing treatments for serious ophthalmic conditions. The company's primary focus is on addressing untreatable diseases of the eye, with its lead drug candidate being an orally delivered compound aimed at treating Stargardt disease and age-related macular degeneration. Through its innovative approach, Alkeus aims to empower medical professionals to manage symptoms of irreversible vision loss at an earlier stage in affected patients.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Zenas BioPharma is a clinical-stage biopharmaceutical company focused on developing immune-based therapies for autoimmune and immune-mediated diseases. Based in Florida, United States, the company advances biologic programs and aims to modulate B cell–driven pathology. Its lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb on B cells, thereby inhibiting harmful immune activity without depleting B cells.

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

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. The company specializes in developing tumor-targeted small molecule medicines that leverage radioisotopes to improve cancer treatment outcomes. RayzeBio focuses on creating innovative radiopharmaceuticals, particularly utilizing alpha-emitting radioisotopes like Actinium-225, to target solid tumors effectively. With a robust pipeline of drug candidates, RayzeBio is committed to addressing significant market opportunities in oncology through late-stage clinical programs, development initiatives, and discovery efforts. The company's mission is to provide effective therapeutic solutions aimed at defeating cancer.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral, selective TYK2 inhibitor in Phase 1 trials for various autoimmune diseases.

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.

Strata Oncology, Inc. is a precision oncology company focused on enhancing cancer care by facilitating access to precision medicine clinical trials and expediting drug approval processes. Based in Ann Arbor, Michigan, the company develops a comprehensive precision oncology platform that supports routine testing and efficient use of molecular data. Strata's offerings include The Strata Trial, a genomic testing program for systematic precision oncology implementation, and StrataNGS, a targeted assay for sequencing DNA and RNA from biopsies. Additionally, StrataPOINT integrates electronic medical record histories with molecular profiling to ensure eligible patients are tested and considered for trial enrollment. The company features a portfolio of pharma-sponsored therapeutic protocols, known as Strata Partnered Trials, aligned to specific biomarkers. Strata also operates The Strata Lab, a cancer sequencing facility, and maintains the Strata Precision Oncology Network, a collaborative entity aimed at promoting precision medicine initiatives. Formerly known as Strata Oncology Research, Inc., the company rebranded in 2016 and has been incorporated since 2015.

Nuvalent is a biotechnology company focused on developing targeted therapies for clinically proven kinase targets in cancer. Leveraging expertise in structure-based design, Nuvalent creates innovative small molecules with exquisite target selectivity to overcome resistance and minimize adverse events.

Oxford Nanopore Technologies Limited is a biotechnology company specializing in the development and commercialization of nanopore-based sequencing technology for the analysis of DNA, RNA, proteins, and small molecules. Its innovative product lineup includes the portable MinION device, the benchtop PromethION, and the GridION system, all of which enable real-time data streaming and scalable sequencing for a variety of applications in scientific research, personalized medicine, environmental monitoring, and agriculture. The Flongle offers smaller, on-demand sequencing solutions, catering to users requiring rapid insights. Operating in over 80 countries, Oxford Nanopore's technology is designed for accessibility and ease of use, featuring straightforward library preparation and automated solutions. The company maintains a robust patent portfolio, supporting its ongoing research and development in both biological and solid-state nanopore technologies. Founded in 2005 and based in Oxford, United Kingdom, Oxford Nanopore Technologies also has offices in multiple countries, including the United States, China, and Germany.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Entrada Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, focused on treating diseases through the intracellular delivery of biologics. Founded in 2016, the company has developed innovative technologies that facilitate the efficient delivery of proteins, peptides, and nucleic acids, targeting intracellular sites that have traditionally been difficult to access. Entrada's approach includes Intracellular Enzyme Replacement Therapy (IC-ERT) and Endosomal Escape Vehicle (EEV) therapeutics, which aim to improve the therapeutic index by enabling the delivery of a wide range of therapeutics across various organs and tissues. The company's pipeline addresses conditions related to neuromuscular diseases and expands into additional therapeutic areas, positioning Entrada at the forefront of advancing treatment options for patients with devastating illnesses.

Founded in 2018, Scribe Therapeutics specializes in engineering and developing in vivo therapies based on CRISPR technology to rewrite and repair genetic disorders. Its proprietary platform, X-Editing (XE), enables greater efficacy, specificity, and deliverability compared to existing CRISPR technologies.

Zenas BioPharma is a clinical-stage biopharmaceutical company focused on developing immune-based therapies for autoimmune and immune-mediated diseases. Based in Florida, United States, the company advances biologic programs and aims to modulate B cell–driven pathology. Its lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb on B cells, thereby inhibiting harmful immune activity without depleting B cells.

Ventyx Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral, selective TYK2 inhibitor in Phase 1 trials for various autoimmune diseases.

Centessa Pharmaceuticals is a biopharmaceutical company that focuses on advancing validated drug programs through an asset-centric R&D model. Each program is developed by dedicated subsidiaries, supported by centralized infrastructure and expert management.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

LianBio is a biopharmaceutical company founded in 2019, headquartered in Shanghai, China, with an additional office in Princeton, New Jersey. The company is dedicated to the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as addressing other unmet medical needs in Greater China and major Asian markets. LianBio focuses on in-licensing assets and collaborates with leading partners to enhance its pipeline, which includes clinically validated product candidates across various therapeutic areas such as cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory indications. The company aims to accelerate the availability of innovative medicines to improve patient outcomes through a science-driven approach.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Nuvation Bio is a global biopharmaceutical company dedicated to developing innovative therapies for oncology, focusing on patients with difficult-to-treat cancers where conventional treatments have proven ineffective.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Dyne Therapeutics is a clinical-stage biotechnology company developing therapies for genetically driven neuromuscular diseases. It aims to deliver disease-modifying therapeutics to skeletal, cardiac, and smooth muscle, addressing conditions such as muscular dystrophies and other muscle disorders. The company uses the FORCE platform to create targeted therapeutics with payloads designed to address the disease gene, connected via a linker to an antigen-binding fragment, enabling delivery to muscle tissue and the central nervous system. Its pipeline includes DYNE-101, DYNE-251, DYNE-302, and DYNE-401. Headquartered in Waltham, Massachusetts.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on the discovery and development of small molecule therapies targeting p53 mutations in cancer. Its lead candidate, PC14586, aims to correct and restore the function of the p53 protein, which is critical in regulating cell growth and preventing tumor formation. In addition to PC14586, the company is developing therapies for various p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, PMV Pharmaceuticals leverages extensive expertise in p53 biology to advance its innovative treatment options for cancer patients.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various hematological cancers and solid tumors. Established in 2017, the company specializes in therapies that target receptor tyrosine kinase-like orphan receptor 1 (ROR1), a protein present on many cancerous cells but absent in normal tissues, making it a promising target for cancer treatment. VelosBio's lead product, VLS-101, is an ROR1-directed ADC designed for patients with both hematologic and solid tumor malignancies. The company's approach aims to provide novel targeted therapies that can be utilized as monotherapy or in combination with other treatments across a wide array of cancer types.

Freeline Therapeutics is a clinical-stage biotechnology company specializing in systemic adeno-associated virus (AAV)-based gene therapies. Its pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A, all in various stages of clinical trials.

Goldfinch Bio, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, dedicated to discovering and developing precision therapies for patients with kidney diseases. The company focuses on conditions such as focal segmental glomerulosclerosis, diabetic nephropathy, and polycystic kidney disease. Key product candidates include GFB-887, a selective inhibitor of the TRPC5 channel, and GFB-024, a cannabinoid receptor 1 inverse agonist. Goldfinch Bio integrates advancements in kidney genetics and biology through its Kidney Genome Atlas, a genomic registry that aids in identifying new disease targets and biomarkers. Additionally, the company employs stem cell science to create human organoid models, which facilitate the validation of novel therapeutic targets. Goldfinch Bio has established a strategic collaboration with Gilead Sciences to further enhance its research and development efforts. Founded in 2016, Goldfinch Bio aims to transform the treatment landscape for progressive kidney disease.

Nurix Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing oral small-molecule therapies that modulate cellular protein levels to treat cancer and immune disorders. It operates DELigase, an integrated discovery platform that targets E3 ligases to alter protein abundance within cells. The company's product pipeline includes targeted protein degraders such as NX-2127, an oral Bruton's tyrosine kinase degrader for relapsed or refractory B-cell malignancies, and NX-1607, an oral Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology indications; these are in preclinical stages. Nurix also explores inhibitors of CBL-B, an E3 ligase that regulates T cell activation. The company collaborates with major industry partners, including Gilead Sciences and Sanofi, and was founded in 2009 and is headquartered in San Francisco.

Kymera Therapeutics is a biotechnology company focused on targeted protein degradation. It employs a proprietary predictive modeling platform to discover novel small molecule therapeutics that selectively degrade disease-causing proteins.

Generation Bio is a genetic medicines company developing durable, redosable treatments for patients with rare and prevalent diseases. Its non-viral platform combines closed-ended DNA, cell-targeted lipid nanoparticles, and scalable manufacturing processes to deliver large and multiple genes across various tissues.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

Impulse Dynamics N.V. is a medical device company that specializes in developing innovative electrical therapies for chronic heart failure. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, with product development centers located in New Jersey and Israel. Its primary offering, the CCM® therapy, utilizes cardiac contractility modulation technology to deliver non-excitatory electrical impulses to the heart, enhancing its contraction and improving blood flow to the body. This minimally invasive treatment option addresses the needs of heart failure patients who have limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for ongoing research and development.

Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that incorporates a proprietary dry-powder formulation of dihydroergotamine mesylate. Designed for self-administration, STS101 utilizes a pre-filled, single-use nasal delivery device, enhancing patient convenience and accessibility. The product is currently undergoing Phase III clinical trials, marking a significant step in its development. Founded in 2016, Satsuma is headquartered in South San Francisco, California.

Atreca is a biopharmaceutical company that discovers and develops antibody-based immunotherapeutics for various solid tumor types. Its lead product candidate, ATRC-101, is a monoclonal antibody with a novel mechanism of action and target derived from its proprietary discovery platform.

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at revolutionizing cancer treatment. By harnessing the immune system's ability to recognize and attack neoantigens, the company seeks to develop innovative therapies that improve patient outcomes. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to enhance the immune response against tumors. Through its focused research and development efforts, Neon Therapeutics is committed to advancing the field of immuno-oncology and providing new options for cancer patients.

Arcus Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Hayward, California, focused on developing innovative immunotherapies for cancer treatment. Founded in 2015, the company leverages insights from immunology to create novel therapies targeting the ATP-adenosine pathway, a critical driver of immunosuppression in the tumor microenvironment. Arcus has a diverse product pipeline featuring several candidates, including Etrumadenant (AB928), a dual A2a/A2b adenosine receptor antagonist in Phase 1b/2 trials; Zimberelimab, an anti-PD-1 monoclonal antibody in Phase Ib trials; and AB154, an anti-TIGIT monoclonal antibody in Phase 2 development for first-line metastatic non-small cell lung cancer. Additionally, AB680, a small-molecule CD73 inhibitor, is undergoing Phase 1/1b studies for the treatment of first-line metastatic pancreatic cancer. The company collaborates with other organizations, including AstraZeneca and Strata Oncology, to advance its clinical programs.

Tricida, Inc. is a late-stage pharmaceutical company based in South San Francisco, California, established in 2013. The company specializes in the development and commercialization of TRC101, a non-absorbed, orally-administered polymer designed to treat metabolic acidosis in patients with chronic kidney disease (CKD). Metabolic acidosis, a common complication of CKD, can accelerate kidney deterioration and is associated with serious health risks, including muscle wasting and loss of bone density. Tricida has successfully completed a Phase 3, double-blind, placebo-controlled trial of TRC101, positioning the company to address a significant unmet medical need in CKD management through the innovative approach of binding and removing acid from the gastrointestinal tract.

Forty Seven, Inc. is a clinical-stage immuno-oncology company headquartered in Menlo Park, California, focused on developing innovative therapies to activate macrophages for cancer treatment. The company’s lead product, magrolimab, is a humanized monoclonal antibody targeting the CD47 receptor, which tumors exploit to evade immune detection. Magrolimab is currently undergoing Phase 1b/2 clinical trials for various cancers, including solid tumors and hematologic malignancies such as acute myeloid leukemia and non-Hodgkin's lymphoma. Additionally, Forty Seven is advancing other antibody-based therapies, including FSI-189, which targets SIRPa, and FSI-174, an anti-cKIT antibody. The company collaborates with Genentech and Acerta Pharma to explore further applications of its therapies in combination with other cancer treatments. Founded in 2014 and previously known as CD47 Sciences, Inc., Forty Seven operates as a subsidiary of Gilead Sciences, Inc.

Atreca is a biopharmaceutical company that discovers and develops antibody-based immunotherapeutics for various solid tumor types. Its lead product candidate, ATRC-101, is a monoclonal antibody with a novel mechanism of action and target derived from its proprietary discovery platform.

Nightstar Therapeutics is a clinical-stage gene therapy company based in London, United Kingdom, specializing in the development of innovative one-time treatments for rare inherited retinal diseases. The company is advancing several product candidates, including its lead candidate NSR-REP1, which is in phase 3 clinical development for choroideremia. Additionally, Nightstar is working on NSR-RPGR, currently in phase 2/3 trials for X-linked retinitis pigmentosa, as well as NSR-BEST1. The company is also developing NSR-ABCA4, which is in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address significant unmet medical needs in the field of retinal disorders.

Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at revolutionizing cancer treatment. By harnessing the immune system's ability to recognize and attack neoantigens, the company seeks to develop innovative therapies that improve patient outcomes. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to enhance the immune response against tumors. Through its focused research and development efforts, Neon Therapeutics is committed to advancing the field of immuno-oncology and providing new options for cancer patients.

Mersana Therapeutics is a clinical-stage biopharmaceutical company that develops antibody-drug conjugates (ADCs) for cancer patients with unmet medical needs. It leverages proprietary ADC platforms, including Dolaflexin, Dolasynthen, and Immunosynthen, to create a diverse pipeline of product candidates designed to improve therapeutic benefit and address oncology indications underserved by traditional ADCs. The lead candidate XMT-1536 targets NaPi2b and is in Phase I trials for ovarian cancer, non-small cell lung cancer, and other indications. The company also advances additional programs such as Emi-Le (XMT-1660), XMT-2056, XMT-2068, and XMT-2175, reflecting its focus on next-generation ADCs. Mersana is headquartered in Cambridge, Massachusetts, and was founded in 2005.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Allena Pharmaceuticals, Inc. is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, focused on the development and commercialization of innovative oral enzyme therapeutics for rare and severe metabolic and kidney disorders. Founded in 2011, the company’s lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a condition characterized by elevated urinary oxalate levels that can lead to kidney stones and chronic kidney disease. Additionally, Allena is developing ALLN-346, a novel enzyme intended to address hyperuricemia in patients with moderate to severe chronic kidney diseases. By targeting metabolic disorders associated with the accumulation of harmful metabolites, Allena Pharmaceuticals aims to provide effective treatments for patients facing serious health challenges.

CytomX Therapeutics is a clinical-stage oncology-focused biopharmaceutical company that develops proteolytically-activated antibodies based on its Probody technology platform. Probody therapeutics are designed to be activated by the tumor microenvironment, enabling targeted cancer immunotherapies with reduced activity in healthy tissues. The company advances antibody and antibody-drug conjugate programs such as CX-072 (PD-L1), CX-2009 (CD166-targeted PDC), BMS-986249 (CTLA-4 Probody), CX-2029 (CD71-targeted PDC), and BMS-986288 (CTLA-4 Probody) in clinical research, and maintains collaborations with AbbVie, Amgen, Bristol-Myers Squibb, ImmunoGen, Pfizer, and Astellas to develop Probody therapeutics. Founded in 2008 and headquartered in South San Francisco, the company aims to improve the precision and safety of antibody-based therapies for cancer and inflammatory diseases.

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).

Jounce Therapeutics, Inc. is a clinical-stage immunotherapy company based in Cambridge, Massachusetts, focused on developing innovative therapies for cancer treatment. The company aims to harness the immune system to target and eliminate cancerous cells, thereby enhancing patient outcomes and quality of life. Among its key product candidates is vopratelimab, a monoclonal antibody currently undergoing Phase II clinical trials for patients with non-small cell lung cancer and urothelial cancer. Jounce is also advancing JTX-4014, an anti-PD-1 antibody for combination therapy, and JTX-1811, which is designed to deplete T regulatory cells in the tumor microenvironment. Additionally, JTX-8064 targets a specific receptor on macrophages to modulate the immune response. Founded in 2013 by experts in immunobiology and cancer biology, Jounce utilizes a proprietary Translational Science Platform to analyze the cellular and molecular characteristics of tumors, driving its commitment to developing first-in-class cancer immunotherapies.

Voyager Therapeutics, Inc. is a clinical-stage gene therapy company dedicated to developing innovative treatments for severe neurological diseases. The company’s lead clinical candidate, VY-AADC, is currently undergoing an open-label Phase 1b trial for Parkinson’s disease. Its diverse preclinical pipeline includes candidates for various conditions, such as VY-SOD102 for amyotrophic lateral sclerosis, VY-HTT01 for Huntington’s disease, VY-FXN01 for Friedreich’s ataxia, and programs targeting tauopathies and synucleinopathies, which encompass Alzheimer’s disease and related disorders. Voyager Therapeutics employs a gene therapy platform known as TRACER, which enables the engineering and optimization of adeno-associated virus (AAV) therapies aimed at producing lasting therapeutic effects from a single treatment. The company collaborates with several institutions, including the University of Massachusetts and ClearPoint Neuro, as well as industry partners like Brammer Bio and Fujifilm Diosynth Biotechnologies, to enhance its gene therapy initiatives. Founded in 2013 and headquartered in Cambridge, Massachusetts, Voyager Therapeutics is committed to addressing unmet medical needs in the central nervous system.

Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for underserved patient communities, particularly those affected by blood disorders. The company is advancing its lead product candidate, Oxbryta (voxelotor), which is an oral, once-daily therapy designed for the treatment of sickle cell disease (SCD). Oxbryta has completed Phase III clinical trials in adult and adolescent patients and is currently being evaluated for safety and pharmacokinetics in a Phase IIa trial involving pediatric patients. In addition to its efforts in SCD, Global Blood Therapeutics is engaged in research and development for other blood-related conditions, including hereditary angioedema, and holds licensed rights to its product candidates in multiple regions, including the United States and Europe.

Moderna is a biotechnology company focused on messenger RNA therapeutics and vaccines. It develops mRNA-based medicines that instruct cells to produce therapeutic proteins or antibodies in vivo, targeting infectious diseases, oncology, cardiovascular diseases, and rare genetic disorders. Founded in 2010 and headquartered in Cambridge, Massachusetts, the company has built a broad pipeline with multiple development candidates across several modalities and a track record highlighted by a COVID-19 vaccine authorized in the United States. Moderna collaborates with major pharmaceutical and research partners, including AstraZeneca, Merck, Vertex, and Alexion, as well as academic institutions such as Harvard University and Karolinska Institutet. It maintains manufacturing collaborations with Lonza for mRNA production and Catalent for fill-finish, and engages with public sector and philanthropic supporters such as BARDA, DARPA, and the Gates Foundation. As of 2025, the company continued expanding its mRNA development program across infectious disease, oncology, cardiovascular, and rare genetic diseases.

Nivalis Therapeutics is a biotechnology company focused on developing innovative disease-modifying therapies aimed at cystic fibrosis (CF). The company is particularly dedicated to preserving intracellular S-nitrosoglutathione (GSNO), a naturally occurring molecule that plays a crucial role in cell signaling and is linked to the pathophysiology of CF. Its lead therapeutic candidate, N91115, specifically targets patients with the F508del mutation, which is the most prevalent mutation responsible for cystic fibrosis. Founded in 2007 and based in Boulder, Colorado, Nivalis Therapeutics aims to significantly improve the quality of life for individuals affected by cystic fibrosis and their families.

Blueprint Medicines is a Cambridge, Massachusetts-based biopharmaceutical company that develops targeted, small-molecule therapies to treat genomically defined cancers, rare diseases, and related conditions. The company focuses on precision medicines that address molecular drivers and resistance mechanisms, advancing a pipeline that includes avapritinib for systemic mastocytosis and certain gastrointestinal stromal tumors, BLU-263, a KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders, fisogatinib for advanced hepatocellular carcinoma, and pralsetinib, a RET inhibitor for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. It is also pursuing BLU-782, an ALK2 inhibitor in early clinical trials for fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with partner companies such as Clementia Pharmaceuticals, CStone Pharmaceuticals, Genentech, and Hoffmann-La Roche. Founded in 2008, the company operates in the United States and Europe and combines its Insights-to-Validation platform with a proprietary chemical library to discover and develop new therapeutic compounds and combination therapies in oncology, hematology, and related areas.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Adaptimmune Therapeutics is a biopharmaceutical company focused on developing novel cell therapies to treat solid tumors. It employs its proprietary SPEAR T-cell platform to identify cancer targets and generate therapeutic candidates for clinical trials.

Civitas Therapeutics is a biopharmaceutical company dedicated to the development and commercialization of innovative pulmonary delivery therapies aimed at treating central nervous system and respiratory disorders. Utilizing its proprietary ARCUS technology, Civitas aims to transform the therapeutic landscape for patients across the United States. The company's focus is on creating effective treatments that enhance patient outcomes and address unmet medical needs in these critical areas.

Spark Therapeutics focuses on developing potentially curative, one-time gene therapy products to treat debilitating genetic diseases. Its pipeline includes LUXTURNA for inherited retinal disease, SPK-FIX for hemophilia B (in collaboration with Pfizer), and preclinical programs targeting neurodegenerative diseases and other retinal degenerative conditions.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.