Wellington Management

MedinCell is an injectable slow-release drug specialist company.

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.

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.

Bluejay Therapeutics is a biopharmaceutical company dedicated to developing cures for infectious diseases, with a primary focus on chronic Hepatitis B. This condition poses a significant global health challenge, and the company aims to address the urgent need for effective treatments. Bluejay is advancing two innovative therapeutic approaches: best-in-class fully human IgG1 anti-HBs monoclonal antibodies and first-in-class oral small molecule inhibitors targeting HBsAg. These strategies are designed to reduce hepatitis B surface antigen levels and restore adaptive immunity, potentially leading to a functional cure for patients. Additionally, the company’s research encompasses therapeutic options for chronic Hepatitis D, further solidifying its commitment to improving immunity and recovery for individuals affected by serious viral and liver diseases.

Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

BridgeBio Oncology Therapeutics operates as a biopharmaceutical company.

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.

Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Avalyn Pharma is a biopharma company that advances therapies for the treatment of Idiopathic Pulmonary Fibrosis (IPF) and other severe respiratory diseases. Its therapies rapidly advance AP01 for the treatment of IPF, provides additional pipeline candidates for IPF, improve the care and outcomes of severe respiratory diseases, and develop novel inhaled therapeutics. Avalyn Pharma was established in 2011 and is headquartered in Seattle, Washington.

Upstream Bio is a clinical-stage biotechnology company dedicated to developing antibody therapies for inflammatory diseases, particularly severe asthma and other respiratory disorders. The company's lead candidate, verekitug, is a monoclonal antibody that acts as an antagonist to the Thymic Stromal Lymphopoietin (TSLP) receptor. TSLP is a key cytokine that drives inflammatory responses and is strategically positioned upstream of various signaling pathways influencing immune cells. Upstream Bio aims to address unmet medical needs in the treatment of severe respiratory conditions by leveraging this validated target to create effective therapies.

Alkeus Pharmaceuticals is a Boston-based clinical-stage biopharmaceutical company dedicated to developing treatments for serious and untreatable ophthalmic conditions. The company's primary focus is on its lead drug candidate, an orally delivered compound aimed at addressing Stargardt disease and age-related macular degeneration. This innovative treatment has the potential to help medical professionals manage symptoms of irreversible vision loss early in life, offering hope to patients affected by these debilitating eye diseases.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Chroma Medicine is a genomic medicine company based in Cambridge, Massachusetts, founded in 2021. The company focuses on epigenetic editing, aiming to revolutionize the treatment of genetically driven diseases. By utilizing epigenetics, which is nature's inherent mechanism for regulating genes, Chroma Medicine develops programmable epigenetic editors that target specific genes and control chromatin conformation. This innovative approach combines a DNA binding domain with epigenetic effector domains, providing medical practitioners with a new class of therapeutics that offers enhanced control over gene expression.

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.

Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

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.

Chroma Medicine is a genomic medicine company based in Cambridge, Massachusetts, founded in 2021. The company focuses on epigenetic editing, aiming to revolutionize the treatment of genetically driven diseases. By utilizing epigenetics, which is nature's inherent mechanism for regulating genes, Chroma Medicine develops programmable epigenetic editors that target specific genes and control chromatin conformation. This innovative approach combines a DNA binding domain with epigenetic effector domains, providing medical practitioners with a new class of therapeutics that offers enhanced control over gene expression.

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

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.

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.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Entrada Therapeutics, Inc. is a biotechnology company focused on treating diseases through the intracellular delivery of biologics, such as proteins, peptides, and nucleic acids. Founded in 2016 and based in Boston, Massachusetts, the company aims to transform patient care by developing a new class of medicines that target intracellular mechanisms previously deemed inaccessible. Entrada's technology addresses challenges associated with delivering both large and small molecules, significantly enhancing the efficiency of therapeutic delivery into the cytosol. Its pipeline includes innovative therapies for neuromuscular diseases and other conditions, utilizing proprietary Endosomal Escape Vehicle (EEV) therapeutics to improve treatment outcomes across various organs and tissues.

Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

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.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Centessa Pharmaceuticals is a next-generation biopharmaceutical company that aims to reshape the traditional drug development process. The company applies an asset-centric R&D model at scale to advance a portfolio of highly validated programs led by industry-leading teams. Each program is developed by a Centessa Subsidiary and supported by a centralized infrastructure and the Centessa management team.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

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

Scorpion Therapeutics, Inc. is a biotechnology company based in Boston, Massachusetts, focused on developing next-generation precision oncology technologies for cancer treatment. Founded in 2020, the company specializes in creating targeted small-molecule drugs that interact with validated cancer targets. Its research encompasses therapies aimed at known oncogenes, previously undruggable cancer targets, and novel targets. By integrating technologies across target discovery, medicinal chemistry, and translational medicine, Scorpion Therapeutics aims to advance a comprehensive pipeline of optimized compounds. The company's goal is to expand the reach of precision medicine, enabling healthcare professionals to offer innovative treatments to a broader range of cancer patients.

LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics.

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.

Nuvation Bio Inc. is a biopharmaceutical company dedicated to developing innovative therapies for oncology, addressing unmet medical needs in the treatment of challenging cancers that have not responded to conventional therapies. Founded in 2018 and based in New York, with an additional office in San Francisco, the company has a portfolio of drug development candidates aimed at improving patient outcomes. One of its key clinical-stage candidates is NUV-868, an oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. Additionally, Nuvation Bio is advancing its proprietary small molecule Drug-Drug Conjugate platform, further expanding its therapeutic arsenal in oncology.

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.

Escape Bio is a clinical-stage biopharmaceutical company that develops small molecule drugs intended to treat neurodegenerative diseases. The company is developing therapeutics for the novel, precisely therapies for genetic neurodegenerative diseases. Escape Bio's pipeline includes candidates targeting known genetic drivers of multiple diseases, including ESB1609, a small molecule S1P5 receptor agonist for the treatment of CNS lysosomal storage disorders and ESB5070, a small molecule kinase inhibitor for Parkinson’s disease patients who have an LRRK2 G2019S variant and an Alzheimer's disease program targeting ApoE4. E-scape Bio was founded in 2015 and is headquartered in San Francisco, California, United States.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on discovering and developing small molecule therapies targeting p53 mutations in cancer. The company's lead candidate, PC14586, aims to correct and restore the function of p53, a critical tumor suppressor protein. In addition to this product, PMV Pharmaceuticals is also working on therapies for specific p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, the company leverages over three decades of expertise in p53 biology, aiming to develop first-in-class modulators that could improve treatment outcomes for 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.

VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat various forms of cancer, including hematological cancers and solid tumors. The company's primary area of research centers on targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein found in numerous tumors but absent in normal tissues, which makes it a promising target for cancer therapy. VelosBio's lead product, VLS-101, is a ROR1-directed ADC that has shown positive preclinical results and aims to provide new treatment options for patients. Founded in 2017, VelosBio operates under the umbrella of Merck & Co., Inc. as of December 2020, focusing on developing targeted therapies that can be administered alone or in combination with other treatments across a wide range of cancer types.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Nurix Therapeutics is a biopharmaceutical company specializing in the discovery, development, and commercialization of small molecule therapies aimed at modulating cellular protein levels to treat cancer and immune disorders. The company's pipeline includes promising preclinical candidates such as NX-2127, an orally available Bruton’s tyrosine kinase degrader targeted at relapsed or refractory B-cell malignancies, and NX-1607, a Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology applications. Nurix's innovative drug discovery platform, DELigase, leverages its expertise in E3 ligases—enzymes that regulate protein modulation within cells—to identify and advance novel drug candidates. Founded in 2009 and based in San Francisco, Nurix Therapeutics aims to provide effective therapies for challenging diseases through its cutting-edge research and development efforts.

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.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Aligos Therapeutics, Inc. is a clinical stage biopharmaceutical company that was founded in 2018 with the mission to become a world leader in the treatment of viral infections and liver diseases. Aligos is focused on the development of targeted antiviral therapies for chronic hepatitis B (CHB) and coronaviruses as well as leveraging its expertise in liver diseases to create targeted therapeutics for nonalcoholic steatohepatitis (NASH). Aligos’ strategy is to harness the deep expertise and decades of drug development experience its workforce has in liver disease, particularly viral hepatitis, to rapidly advance its pipeline of potentially best-in-class molecules.

FORMA Therapeutics is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, specializing in the development and commercialization of innovative therapeutics for rare hematologic diseases and various cancers. The company's notable product candidates include FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, which targets metastatic castration-resistant prostate cancer. Additionally, FORMA is advancing FT-2102, an oral investigational agent designed to inhibit mutated IDH1 enzymes, now being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and exploratory Phase 1 trials for glioma. The company also develops FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. By integrating advanced drug discovery technologies and oncology expertise, FORMA aims to target challenging biological pathways that contribute to cancer, thereby enhancing the development of small molecule drug candidates with unique mechanisms of action.

Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

Satsuma Pharmaceuticals 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 features a proprietary dry-powder formulation of dihydroergotamine mesylate. This product is designed for self-administration through a pre-filled, single-use nasal delivery device and is currently undergoing Phase III clinical trials. Founded in 2016 and based in South San Francisco, California, Satsuma aims to provide an effective therapeutic option for individuals suffering from acute migraine episodes.

Neon Therapeutics is a clinical-stage biopharmaceutical company. It is focused in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. The company's pipeline products are NEO-PV-01, NEO-PTC-01, and NEO-SV-01.

Arcus Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Hayward, California, focused on developing innovative cancer therapies. The company’s product pipeline includes several key candidates, such as AB928, a dual A2a/A2b adenosine receptor antagonist in Phase 1b/2 trials, and Zimberelimab, an anti-PD-1 monoclonal antibody currently being evaluated in Phase Ib studies. Additionally, Arcus is advancing AB154, an anti-TIGIT monoclonal antibody in Phase 2 trials, and AB680, a small-molecule CD73 inhibitor in Phase 1/1b studies for the treatment of metastatic pancreatic cancer. The company collaborates with Strata Oncology and AstraZeneca to enhance the development of its therapies and has strategic partnerships with firms like WuXi Biologics. Founded in 2015, Arcus aims to leverage emerging insights in immunology to create effective cancer treatments across various indications, including non-small cell lung cancer and pancreatic cancer.

Tricida, Inc. is a late-stage pharmaceutical company based in South San Francisco, California, dedicated to the development and commercialization of its lead drug candidate, TRC101. This non-absorbed, orally-administered polymer is designed to treat metabolic acidosis, a condition often associated with chronic kidney disease (CKD). Metabolic acidosis can contribute to the progression of CKD and is linked to various health issues such as muscle wasting, loss of bone density, and increased mortality risk. Tricida has successfully completed a Phase 3, double-blind, placebo-controlled trial of TRC101, aiming to provide an effective treatment option for patients suffering from this condition. The company was founded in 2013 and is focused on addressing the unmet medical needs of individuals with CKD.

Vets First Choice provides veterinary practices with an online pharmacy and home delivery services. It partners with veterinarians to create insight into gaps in patient care and offer professionalized pro-active prescription management to drive client engagement and improve compliance. The company was founded by Ben Shaw in 2010 and is based in Portland, Maine.

Neon Therapeutics is a clinical-stage biopharmaceutical company. It is focused in the field of neoantigen-targeted therapies, dedicated to transforming the treatment of cancer by directing the immune system towards neoantigens. The company's pipeline products are NEO-PV-01, NEO-PTC-01, and NEO-SV-01.

Mersana Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of antibody-drug conjugates (ADCs) for cancer treatment. The company utilizes its proprietary biodegradable polymer platform, Dolaflexin, to create a pipeline of ADC product candidates aimed at addressing oncology indications with significant unmet needs. Its lead candidate, XMT-1536, is currently in Phase I clinical trials targeting NaPi2b for the treatment of ovarian cancer, non-small cell lung cancer, and other rare conditions. Mersana is also advancing additional candidates, including XMT-1660, targeting B7-H4, and XMT-2056, a STING agonist ADC, both designed to improve tolerability and therapeutic outcomes. The company has established strategic partnerships with Merck KGaA and Asana BioSciences to further develop its ADC candidates. Founded in 2005, Mersana Therapeutics aims to deliver innovative treatments to patients through its advanced research and development efforts.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company headquartered in Watertown, Massachusetts, that specializes in developing therapies for diseases affecting mucosal tissues using its proprietary nanoparticle-based Mucus Penetrating Particles (MPP) technology. This innovative approach enhances drug distribution and pharmacokinetics by delivering biocompatible, drug-loaded particles that uniformly coat mucosal surfaces. The company's lead product candidate, KPI-121 0.25%, has successfully completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS, another product, has also completed two Phase III trials for treating inflammation and pain following ocular surgery. Kala is advancing its pipeline, including KPI-285, a receptor tyrosine kinase inhibitor for retinal diseases, which is currently in preclinical studies. The company aims to address significant clinical needs while exploring research collaborations to enhance its product offerings.

Allena Pharmaceuticals is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, that specializes in developing and commercializing oral enzyme therapeutics for rare and severe metabolic and kidney disorders. The company's primary focus is on conditions that involve the excessive accumulation of metabolites such as oxalate and urate, which can lead to complications like kidney stones and chronic kidney disease. Allena's lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a disorder linked to significantly high urinary oxalate levels. Additionally, the company is developing ALLN-346, a novel enzyme designed to address hyperuricemia in patients with moderate to severe chronic kidney diseases. Allena Pharmaceuticals aims to provide innovative treatments that address unmet medical needs in these patient populations.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

CytomX Therapeutics is an oncology-focused biopharmaceutical company based in South San Francisco, California. The company specializes in developing proteolytically-activated antibody therapeutics using its proprietary Probody technology platform. This innovative approach aims to create cancer immunotherapies that enhance targeting of tumor cells while minimizing effects on healthy tissues. CytomX's clinical-stage product candidates include CX-072, which targets programmed cell death ligand 1; CX-2009, a drug conjugate against CD166; and BMS-986249, a CTLA-4 Probody therapeutic for metastatic melanoma, among others. The company has established strategic collaborations with several major pharmaceutical companies, including AbbVie, Amgen, and Bristol-Myers Squibb, to further the development of its Probody therapeutics. Founded in 2008, CytomX is dedicated to providing patients with more effective and less toxic treatment options for cancer and inflammatory diseases.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in South San Francisco, California, specializing in the discovery, development, and delivery of innovative treatments for underserved patient populations. The company's lead product candidate is Oxbryta (voxelotor), an oral, once-daily therapy designed to treat sickle cell disease (SCD). Oxbryta has successfully completed Phase III clinical trials for adult and adolescent patients and is currently being evaluated in a Phase IIa trial for younger patients. Additionally, Global Blood Therapeutics has a collaboration agreement with Syros Pharmaceuticals to develop therapies targeting both SCD and beta thalassemia. Founded in 2011, the company is also engaged in research for other blood-based disorders, including hereditary angioedema.

Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing therapeutics and vaccines using messenger RNA technology. Founded in 2010, the company focuses on various therapeutic areas, including infectious diseases, oncology, rare genetic disorders, and cardiovascular diseases. As of September 2024, Moderna has 40 mRNA development candidates in clinical development, demonstrating its commitment to advancing medical science. The company's mRNA platform has gained significant recognition, particularly with the authorization of its COVID-19 vaccine in December 2020. Moderna has established strategic collaborations with notable organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation. Additionally, it collaborates with Lonza Ltd. and Catalent for the manufacturing of its COVID-19 vaccine. Through its innovative approach, Moderna aims to provide effective solutions for a range of health challenges.

Nivalis Therapeutics is developing a novel class of disease-modifying therapies that are designed to preserve intracellular GSNO (S-nitrosoglutathione), an endogenous molecule with cell signaling effects that are implicated in the pathophysiology of cystic fibrosis (CF).The Company’s lead candidate, N91115 initially targets patients with the F508del mutation, the most common disease-causing mutation in CF. Nivalis Therapeutics is dedicated to making a difference in the lives of patients with cystic fibrosis and their families. The company was founded in 2007 and is headquartered in Boulder, Colorado, United States.

Blueprint Medicines is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance its research and development efforts.

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 is a clinical-stage biopharmaceutical company based in Abingdon, United Kingdom, established in 2007. The company specializes in the development of T cell therapies aimed at treating cancer, human immunodeficiency virus, and infectious diseases. Adaptimmune employs a proprietary platform to identify cancer targets, genetically engineer T-cell receptors, and produce therapeutic candidates for patient administration. Its key programs include MAGE-A4 SPEAR T-cell therapy, NY-ESO SPEAR T-cell therapy, and CD70, among others, focusing primarily on solid tumors. By utilizing affinity T cell receptor proteins, Adaptimmune enhances the ability of patient T cells to target and destroy cancerous or infected cells.

Civitas Therapeutics is a biopharmaceutical company dedicated to developing and commercializing innovative pulmonary delivery therapies. Utilizing its proprietary ARCUS technology, the company creates therapeutics aimed at treating central nervous system and respiratory disorders. Civitas Therapeutics serves patients across the United States, focusing on delivering transformative treatment options to improve health outcomes.

Spark Therapeutics, Inc. is a biotechnology company focused on developing gene therapy products aimed at treating debilitating genetic diseases. Headquartered in Philadelphia, Pennsylvania, the company was founded in 2013 and has established itself as a leader in the field of gene therapy, with a robust pipeline that includes LUXTURNA for retinal dystrophy, as well as several candidates for hemophilia and choroideremia. Spark is also exploring therapies for neurodegenerative diseases and inherited retinal diseases. The company holds a collaboration agreement with Pfizer for the development of hemophilia B treatments and has partnered with Novartis for the commercialization of its lead product outside the United States. Spark Therapeutics leverages a proprietary manufacturing platform and a team with extensive expertise in research, clinical development, and regulatory affairs, building on a legacy of innovation in gene therapy from its origins at The Children’s Hospital of Philadelphia. As of December 2019, Spark operates as a subsidiary of Roche Holding AG.