Boundless Bio, Inc., biopharma company, develops therapies and medicines for the treatment of cancers. It offers therapeutics based on extrachromosomal DNA (ecDNA) one of the drivers of the aggressive cancers, such as cancers characterized by high copy number amplification of oncogenes. The company was formerly known as Pretzel Therapeutics, Inc. Boundless Bio, Inc. was founded in 2018 and is based in LA Jolla, California.
Saluda Medical Pty Ltd. is a medical device company focused on developing innovative solutions for the neuromodulation industry. The firm is known for its investigational device, Evoke, which features a closed-loop spinal cord stimulation system. This technology measures the spinal cord's response to stimulation and adjusts the stimulation parameters in real-time to optimize therapeutic outcomes for patients. Founded in 2010 and headquartered in Artarmon, Australia, with additional offices in Minnesota and the United Kingdom, Saluda Medical aims to provide effective treatment for chronic neuropathic pain. The company's unique approach involves monitoring each patient's neural fingerprint to automatically tailor electrical stimulation, ensuring that therapy is customized to meet individual needs. Saluda Medical's commitment to advancing medical technology is underpinned by a strong foundation of scientific research and a team of experienced professionals.
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 utilize radioisotopes, particularly focusing on alpha-emitting isotopes like Actinium-225 for the treatment of solid tumors. RayzeBio aims to improve cancer treatment outcomes by creating a robust pipeline of radiopharmaceuticals targeting validated oncology drug targets. Its portfolio includes multiple drug candidates at various stages of development, ranging from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.
Chroma Medicine is a genomic medicine company focused on epigenetic editing to transform the treatment of genetically driven diseases. By leveraging epigenetics, which regulates gene expression naturally, Chroma Medicine aims to develop innovative therapies that provide precise control over gene activity. The company utilizes programmable epigenetic editors that combine DNA binding domains with epigenetic effector domains, allowing for targeted manipulation of genes and chromatin structure. This approach promises to create a new class of therapeutics that offers unparalleled control over gene expression, potentially leading to more effective treatments for a range of genetic disorders.
Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing oncology medicines tailored to individual patients. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments, thereby enhancing the effectiveness of drug development. Acrivon's lead program is ACR-368, also known as prexasertib, which is currently in Phase 2 trials. Additionally, the pipeline includes preclinical programs that target critical components of the DNA Damage Response and cell cycle regulation pathways, specifically focusing on proteins such as WEE1 and PKMYT1. By leveraging these innovative approaches, Acrivon aims to improve therapeutic outcomes for cancer patients.
Ventyx Biosciences, Inc. is a biotechnology company based in Encinitas, California, focused on developing selective inhibitors of TYK2 for the treatment of autoimmune diseases. Incorporated in 2018, the company boasts a diverse portfolio of innovative clinical and pre-clinical programs aimed at high-value therapeutic targets. Through its research and development efforts, Ventyx is committed to advancing treatments that address unmet medical needs in the autoimmune disease space.
Asher Biotherapeutics, Inc. is a biotechnology company focused on developing immunotherapy drugs for cancer treatment. Founded in 2019 and headquartered in South San Francisco, California, the company aims to create innovative therapeutic solutions to improve patient outcomes in oncology. Asher Bio leverages advanced technologies and research to enhance the effectiveness of immune-based therapies, positioning itself as a key player in the evolving field of cancer treatment.
Strata Oncology is a precision oncology company focused on enhancing cancer care through increased access to clinical trials and expediting drug approval processes. Based in Ann Arbor, Michigan, Strata develops a comprehensive oncology platform that facilitates routine genomic testing and the efficient use of molecular data. Its offerings include The Strata Trial, a genomic testing protocol aimed at implementing enterprise-wide precision oncology; StrataNGS, a targeted assay for sequencing DNA and RNA from biopsy samples; and StrataPOINT, which integrates electronic medical record data with molecular profiling to ensure eligible patients are tested and considered for precision trials. Additionally, the company manages Strata Partnered Trials, a collection of pharmaceutical-sponsored protocols linked to specific biomarkers, and operates The Strata Lab, a cancer sequencing facility. Through its collaborative network of cancer centers, Strata is dedicated to advancing precision medicine for patients with cancer, ultimately providing actionable insights for healthcare providers.
NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.
Nuvalent is a biotechnology company that develops targeted therapies for clinically proven kinase targets in cancer. Leveraging deep expertise in structure-based design, Nuvalent develops innovative small molecules with exquisite target selectivity to overcome resistance, minimize adverse events, and drive more durable responses. It is advancing a robust pipeline with parallel lead programs in ROS1-positive and ALK-positive NSCLC, along with multiple discovery-stage research programs. It was founded in 2017 and is based in Cambridge, Massachusetts.
Boundless Bio, Inc., biopharma company, develops therapies and medicines for the treatment of cancers. It offers therapeutics based on extrachromosomal DNA (ecDNA) one of the drivers of the aggressive cancers, such as cancers characterized by high copy number amplification of oncogenes. The company was formerly known as Pretzel Therapeutics, Inc. Boundless Bio, Inc. was founded in 2018 and is based in LA Jolla, California.
Entrada Therapeutics, Inc., a biotechnology company, engages in the treatment of diseases through the intracellular delivery of biologic. The company enables intracellular delivery of proteins, peptides, and nucleic acids and allows development of programs across intracellular target classes. Its Intracellular enzyme replacement therapy (IC-ERT) is a medical treatment that corrects an enzyme deficiency in the cell, Protein-Protein Interaction Inhibitors. The company was founded in 2016 and is based in Boston, Massachusetts.
Ventyx Biosciences, Inc. is a biotechnology company based in Encinitas, California, focused on developing selective inhibitors of TYK2 for the treatment of autoimmune diseases. Incorporated in 2018, the company boasts a diverse portfolio of innovative clinical and pre-clinical programs aimed at high-value therapeutic targets. Through its research and development efforts, Ventyx is committed to advancing treatments that address unmet medical needs in the autoimmune disease space.
Artiva Biotherapeutics, Inc., a biotech company, develops and manufactures cellular immunotherapies for cancer patients. The company offers a pipeline of off-the-shelf, allogeneic natural killer (NK) cell therapies for the treatment of hematologic cancers or solid tumors. The company’s products target CD20 and CD19 in B-cell lymphomas and HER2 in various solid tumors. The company was founded in 2019 and is headquartered in San Diego, California. Artiva Biotherapeutics, Inc. operates as a subsidiary of Green Cross Holdings Corporation.
Centessa Pharmaceuticals is a clinical-stage biopharmaceutical company focused on transforming the drug development process through an asset-centric research and development model. The company advances a diverse portfolio of validated programs, each managed by a dedicated subsidiary and backed by a centralized infrastructure and experienced management team. Among its pipeline assets are SerpinPC, targeting Hemophilia A and B, LB101 for solid tumors, ORX750 for narcolepsy type 1 and other sleep disorders, and MGX292 for pulmonary arterial hypertension. Additionally, Centessa is developing an undisclosed asset for solid tumors.
Gemini Therapeutics, Inc. is a precision medicine company established in 2015 and based in Cambridge, Massachusetts. The company specializes in developing therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini's approach focuses on addressing genetically-defined conditions by creating therapeutic candidates that target specific molecular abnormalities prevalent in patients with significant clinical needs. Their diverse pipeline features a range of innovative treatment modalities, including monoclonal antibodies, recombinant proteins, and gene therapies. The company has secured funding from prominent life science investors and collaborates with academic institutions globally to advance its mission of delivering potentially first-in-class therapeutics.
Design Therapeutics, Inc. is a clinical-stage biotechnology company based in Solana Beach, California, focused on developing innovative therapies for degenerative disorders resulting from nucleotide repeat expansions. Founded in 2017, the company is advancing its lead program aimed at treating Friedreich's ataxia, while also exploring treatment options for other conditions such as Fragile X syndrome and myotonic dystrophy. Design Therapeutics is known for its GeneTACTM molecules, a new class of small-molecule gene-targeted therapies designed to address the underlying causes of diseases linked to inherited nucleotide repeat expansion mutations. The company's ongoing research efforts seek to develop disease-modifying treatments for serious conditions driven by these genetic factors.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Scorpion Therapeutics, Inc. is a biotechnology company focused on developing next-generation precision oncology technologies aimed at improving cancer treatment. Founded in 2020 and based in Boston, Massachusetts, the company specializes in creating precision oncology drugs that target known oncogenes, address currently undruggable cancer targets, and explore new therapeutic targets. By integrating advanced technologies in target discovery, medicinal chemistry, and translational medicine, Scorpion Therapeutics seeks to expand the reach of precision medicine to a broader patient population. The company's approach is designed to unlock novel small-molecule drugs that can overcome the limitations of existing therapies, thereby enhancing treatment options for cancer patients.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, specializing in the development of small molecule therapies for severe musculoskeletal diseases. Founded in 2017, the company leverages its expertise in skeletal muscle physiology to create innovative treatments for genetically defined muscle disorders, including Duchenne, Becker, and limb girdle muscular dystrophies. Edgewise Therapeutics employs a precision medicine platform to identify and target key muscle proteins and modulators, aiming to address the underlying causes of these conditions. Additionally, the company has established a state-of-the-art research facility to enhance its capabilities in understanding muscle adaptation and injury related to disease.
Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company based in Shanghai, China, focused on discovering and developing innovative cell therapies for cancer treatment. The company's lead product candidates include GC012F, a dual-targeted CAR-T therapy for multiple myeloma currently in Phase I trials; GC019F, which targets adult B cell acute lymphoblastic leukemia and is also in Phase I trials; and GC007F, aimed at B cell non-Hodgkin’s lymphoma, also in Phase I. Additionally, Gracell is developing GC027, a CAR-T candidate targeting adult T cell acute lymphoblastic leukemia, and GC007g, an allogeneic CAR-T therapy for relapsed or refractory B-ALL, both in Phase I trials. Beyond these, Gracell has a pipeline of earlier-stage product candidates targeting various cancers, including ovarian and breast cancer. Founded in 2017, Gracell Biotechnologies is committed to advancing cellular therapeutics to improve patient outcomes in hematological malignancies and solid tumors.
Nuvation Bio Inc., a biopharmaceutical company, focuses on the development of therapies for oncology. Its portfolio includes various oncology programs with multiple drug development candidates. Nuvation Bio Inc.was formerly known as RePharmation Inc. and changed its name to Nuvation Bio Inc. in April 2019. The company was founded in 2018 and is based in New York, New York with an additional office in San Francisco, California.
Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing targeted therapies for women's cancers, particularly estrogen receptor (ER)-positive breast cancer. The company's lead candidate, OP-1250, is an ER antagonist and selective ER degrader currently undergoing Phase 1/2 clinical trials for the treatment of metastatic or locally advanced, ER-positive, and human epidermal growth factor receptor 2-negative breast cancer. Olema's research focuses on utilizing its expertise in endocrine-driven cancers and the molecular mechanisms of the ER to create more effective treatments that aim to improve patient outcomes. In addition to OP-1250, Olema is advancing another drug candidate, OP-3136, further enriching its pipeline. Established in 2006 and headquartered in San Francisco, California, Olema is committed to transforming the standard of care for women facing these challenging diagnoses.
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.
NovellusDx, Ltd. is a biotechnology company based in Jerusalem, Israel, established in 2011 by Haim Gil-ad and Yoram Altschuler. The company specializes in developing targeted therapies for cancer patients by focusing on tumor-specific driver mutations. NovellusDx utilizes innovative testing methods to evaluate the effectiveness of targeted therapy drugs on emulated tumors, allowing for the quantification of therapeutic effects. The company also monitors the oncogenic activity of relevant genes and proteins in patients, assessing changes before and after drug administration. Additionally, NovellusDx develops assays to detect the misregulated translocation of mutated signaling proteins to the nucleus, measuring the impact of drug candidates on specific tumors, particularly concerning their ability to inhibit the translocation of up-regulated signaling proteins.
E-Scape Bio, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule therapies for neurodegenerative diseases. Established in 2015 and headquartered 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. E-Scape Bio is advancing a pipeline of therapeutics that target specific genetic drivers of these diseases, including ESB1609, a small molecule S1P5 receptor agonist for CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor designed for Parkinson’s disease patients with the LRRK2 G2019S variant. Additionally, the company is developing a program targeting ApoE4 in Alzheimer’s disease, emphasizing its commitment to precision medicine in treating genetically defined subpopulations.
PMV Pharma is developing first-in-class p53 and p53 pathway modulators for the treatment of cancer. Bringing together leaders in the field to utilize over three decades of p53 biology, PMV Pharma combines unique biological understanding with pharmaceutical development focus.
Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing targeted therapies for women's cancers, particularly estrogen receptor (ER)-positive breast cancer. The company's lead candidate, OP-1250, is an ER antagonist and selective ER degrader currently undergoing Phase 1/2 clinical trials for the treatment of metastatic or locally advanced, ER-positive, and human epidermal growth factor receptor 2-negative breast cancer. Olema's research focuses on utilizing its expertise in endocrine-driven cancers and the molecular mechanisms of the ER to create more effective treatments that aim to improve patient outcomes. In addition to OP-1250, Olema is advancing another drug candidate, OP-3136, further enriching its pipeline. Established in 2006 and headquartered in San Francisco, California, Olema is committed to transforming the standard of care for women facing these challenging diagnoses.
VelosBio, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-drug conjugates (ADCs) to treat hematological cancers and solid tumors. The company specializes in therapies targeting receptor tyrosine kinase-like orphan receptor 1 (ROR1), a cell-surface protein commonly found in various tumors but absent in normal tissues, making it a promising target for cancer treatment. Its lead product, VLS-101, is designed as a ROR1-directed ADC aimed at patients with both hematologic and solid tumor malignancies. Founded in 2017, VelosBio has demonstrated positive preclinical results for its ROR1-directed therapeutics, paving the way for potential use in monotherapy or in combination with other treatments across a wide range of cancers. As of late 2020, VelosBio operates as a subsidiary of Merck & Co., Inc.
Goldfinch Bio, Inc. is a clinical-stage biotechnology company dedicated to discovering and developing precision therapies for kidney diseases. The company focuses on researching and commercializing treatments for conditions such as focal segmental glomerulosclerosis, diabetic nephropathy, and polycystic kidney disease. Notable programs include GFB-887, a selective inhibitor of the TRPC5 channel, and GFB-024, a cannabinoid receptor 1 inverse agonist. Goldfinch Bio is also developing the Kidney Genome Atlas, a genomic registry aimed at identifying new disease targets and biomarkers, and utilizes a biology platform that leverages stem cell science to create human organoid models for target validation. Founded in 2016 and based in Cambridge, Massachusetts, Goldfinch Bio has established a strategic collaboration with Gilead Sciences to enhance its capabilities in kidney disease therapy development.
Freeline Therapeutics is a clinical-stage biotechnology company focused on developing liver-directed gene therapies for bleeding disorders and other diseases. Based in Stevenage, United Kingdom, Freeline's lead product candidate, FLT180a, is undergoing Phase 1/2 clinical trials aimed at treating moderate to severe hemophilia B. The company's pipeline also includes FLT190, which is in dose-escalating Phase 1/2 trials for Fabry disease, FLT201 for type 1 Gaucher disease, and FLT210 for hemophilia A. Freeline utilizes a next-generation proprietary adeno-associated virus (AAV) vector platform, enhancing its potential for systemic gene therapy applications. Since its founding in 2015, the company has been dedicated to advancing innovative treatments in the field of gene therapy.
Verve Therapeutics is a biotechnology company focused on developing innovative therapies aimed at editing the adult human genome to provide lifelong protection against coronary artery disease, a leading global cause of mortality. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company employs gene-editing technology to create treatments that shift the management of cardiovascular diseases from chronic care to potentially curative interventions. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are recognized for their roles in regulating blood lipids, specifically low-density lipoprotein cholesterol. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance delivery technologies for its cardiovascular treatments.
Nurix Therapeutics is a biopharmaceutical company dedicated to the discovery and development of small molecule therapies aimed at treating cancer and immune disorders. The company focuses on innovative approaches to modulate cellular protein levels, utilizing its expertise in the ubiquitin-proteasome system. Its lead preclinical candidates include NX-2127, an orally available Bruton’s tyrosine kinase (BTK) degrader targeting relapsed or refractory B-cell malignancies, and NX-1607, a Casitas B-lineage lymphoma proto-oncogene-B (CBL-B) inhibitor for immuno-oncology applications. Nurix also offers DELigase, a discovery platform that identifies and advances novel drug candidates by targeting E3 ligases, which play a crucial role in protein regulation. Founded in 2009 and based in San Francisco, the company has established strategic collaborations with major pharmaceutical firms, enhancing its potential in the competitive biopharmaceutical landscape.
Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, established in 2015. It specializes in targeted protein degradation, developing innovative small molecule therapeutics that selectively degrade proteins associated with various diseases by utilizing the body's natural protein degradation mechanisms. The company is advancing several therapeutic programs, including the IRAK4 program aimed at treating immunology-inflammation diseases such as hidradenitis suppurativa, atopic dermatitis, and rheumatoid arthritis. Additionally, it is developing the IRAKIMiD program for MYD88-mutated diffuse large B cell lymphoma and the STAT3 program for hematologic malignancies and solid tumors, as well as autoimmune diseases. Through its proprietary predictive modeling platform, Kymera Therapeutics seeks to accelerate drug discovery and address previously untreatable conditions.
Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and headquartered in South San Francisco, California. The company specializes in developing innovative therapeutics to address unmet medical needs in viral and liver diseases, particularly targeting chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH). Its lead drug candidate, ALG-010133, is a synthetic oligonucleotide currently undergoing Phase I clinical trials for CHB. Aligos is also advancing several other candidates, including ALG-000184, a capsid assembly modulator for CHB; ALG-020572, an oligonucleotide for CHB; ALG-125097, an siRNA drug candidate for CHB; and ALG-055009, a small molecule THR-ß agonist for NASH. The company's strategy leverages the extensive expertise of its team in liver disease and viral hepatitis to develop targeted antiviral therapies and expedite its drug development pipeline.
Forma Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and cancers. The company's key product candidates include FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, aimed at treating metastatic castration-resistant prostate cancer. Additionally, Forma is advancing FT-2102, an oral investigational agent targeting mutated IDH1 enzymes, which is being assessed in a Phase 2 trial for relapsed/refractory acute myeloid leukemia and an exploratory Phase 1 trial for glioma. The pipeline also features FT-4101 and FT-8225, which are selective inhibitors of fatty acid synthase. Founded in 2007 and headquartered in Watertown, Massachusetts, Forma integrates advanced drug discovery technologies with oncology expertise to target significant challenges in cancer treatment and address unmet medical needs.
Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraines. The company's primary product candidate, STS101, is a drug-device combination featuring a proprietary dry-powder formulation of dihydroergotamine mesylate. This medication is designed for self-administration using a pre-filled, single-use nasal delivery device. Currently, STS101 is undergoing Phase III clinical trials, marking a significant step in its development process. Founded in 2016 and based in South San Francisco, California, Satsuma Pharmaceuticals aims to provide effective solutions for individuals suffering from acute migraine attacks.
Atreca, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on discovering and developing antibody-based immunotherapeutics for various solid tumors. Its lead product candidate, ATRC-101, is a monoclonal antibody currently in clinical development, which demonstrates a unique mechanism of action and targets derived from antibodies identified through Atreca's innovative discovery platform. ATRC-101 has shown in vitro reactivity with a significant majority of cancer samples, including those from ovarian, non-small cell lung, colorectal, and breast cancer patients. Additionally, the company has established a strategic research collaboration with Merck Sharp & Dohme Corp. to identify antigenic targets of select antibodies with potential applications in oncology. Founded in 2010, Atreca aims to advance its therapies to improve treatment options for cancer patients.
Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at advancing cancer treatment. By focusing on the immune system's response to neoantigens, the company seeks to develop innovative therapies that enhance the body's ability to fight cancer. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to leverage the unique characteristics of neoantigens to improve patient outcomes.
Arcus Biosciences is a clinical-stage biopharmaceutical company based in Hayward, California, focused on developing innovative cancer therapies through insights in immunology. Founded in 2015 by experienced researchers from the biotechnology sector, the company is advancing a pipeline that includes several promising candidates. Notably, it is developing AB928, a dual A2a/A2b adenosine receptor antagonist, and Zimberelimab, an anti-PD-1 monoclonal antibody, both currently in clinical trials. Additionally, Arcus is working on AB154, an anti-TIGIT monoclonal antibody, and AB680, a small-molecule CD73 inhibitor, targeting various cancer types such as non-small cell lung cancer and pancreatic cancer. The company has established partnerships with organizations like Strata Oncology and AstraZeneca to enhance its research efforts and expand its clinical trials. By focusing on the ATP-adenosine pathway, which plays a crucial role in tumor immunosuppression, Arcus aims to create and optimize differentiated immuno-oncology therapies for patients.
Tricida, Inc. is a pharmaceutical company based in South San Francisco, California, established in 2013. The company is dedicated to the development and commercialization of its lead product candidate, TRC101, a non-absorbed, orally-administered polymer designed to treat metabolic acidosis in patients with chronic kidney disease (CKD). Metabolic acidosis, a condition often associated with CKD, can hasten kidney deterioration and lead to serious complications such as muscle wasting and loss of bone density. Tricida has successfully completed a Phase 3, double-blind, placebo-controlled trial of TRC101, which aims to bind and remove acid from the gastrointestinal tract, thereby addressing the underlying metabolic issues and potentially slowing the progression of CKD.
Forty Seven, Inc. is a clinical-stage immuno-oncology company that is developing therapies targeting cancer immune evasion pathways based on technology licensed from Stanford University. Forty Seven's lead program, 5F9, is a monoclonal antibody against the CD47 receptor, a "don't eat me" signal that cancer cells commandeer to avoid being ingested by macrophages. This antibody is currently being evaluated in six clinical studies in patients with solid tumors, acute myeloid leukemia, non-Hodgkin's lymphoma and colorectal carcinoma.
Atreca, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on discovering and developing antibody-based immunotherapeutics for various solid tumors. Its lead product candidate, ATRC-101, is a monoclonal antibody currently in clinical development, which demonstrates a unique mechanism of action and targets derived from antibodies identified through Atreca's innovative discovery platform. ATRC-101 has shown in vitro reactivity with a significant majority of cancer samples, including those from ovarian, non-small cell lung, colorectal, and breast cancer patients. Additionally, the company has established a strategic research collaboration with Merck Sharp & Dohme Corp. to identify antigenic targets of select antibodies with potential applications in oncology. Founded in 2010, Atreca aims to advance its therapies to improve treatment options for cancer patients.
Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.
Neon Therapeutics is a clinical-stage biopharmaceutical company specializing in neoantigen-targeted therapies aimed at advancing cancer treatment. By focusing on the immune system's response to neoantigens, the company seeks to develop innovative therapies that enhance the body's ability to fight cancer. Its product pipeline includes NEO-PV-01, NEO-PTC-01, and NEO-SV-01, which are designed to leverage the unique characteristics of neoantigens to improve patient outcomes.
Allena Pharmaceuticals is developing and commercializing non-systemic protein therapeutics to treat metabolic and orphan diseases, with a particular focus on nephrology and urologic conditions. Its proprietary technological approach enables the design, formulation, and delivery of non-absorbed and stable enzymes orally and in sufficient doses for activity in the GI tract.
It was founded in 2011 and headquartered in Newton, Massachusetts.
Rhythm Metabolic
Series A in 2015
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 early stage, privately-funded biotechnology company developing Probodiesâ„¢, proteolytically-activated antibodies. Probodies, by their ability to site-direct the activity of antibodies, will result in an improved therapeutic index for validated targets as well enable the drugging of targets with broad tissue expression. Their mission, alone and with the help of partners, is to provide patients with more effective and less toxic therapies for severe illnesses such as cancer and inflammatory diseases.
Unum Therapeutics is a biopharmaceutical company focused on developing immunotherapy products for cancer treatment, utilizing proprietary T-cell engineering technology. The company’s lead programs, based on its Antibody-Coupled T-cell Receptor (ACTR) technology, are in Phase I clinical trials. These include ACTR707 and ACTR087, both being tested in combination with rituximab for relapsed or refractory CD20+ non-Hodgkin lymphoma, as well as ACTR087 combined with SEA-BCMA for relapsed multiple myeloma. Additionally, ACTR707 is being evaluated with trastuzumab for HER2+ solid tumors. Unum Therapeutics is also exploring BOXR1030, a pre-clinical candidate targeting glypican-3 to enhance T cell functionality in the solid tumor microenvironment. Founded in 2014 and headquartered in Cambridge, Massachusetts, Unum aims to activate the body’s immune system to effectively combat cancer through its innovative therapies.
Jounce Therapeutics is a clinical-stage immunotherapy company focused on developing innovative cancer treatments that harness the immune system to target and eliminate cancer cells. The company's lead product, vopratelimab, is a monoclonal antibody currently in Phase II trials for patients with non-small cell lung cancer and urothelial cancer, particularly those who have not responded to PD-1/PD-L1 inhibitors. Additionally, Jounce is advancing several other therapeutic candidates, including JTX-4014, an anti-PD-1 antibody intended for combination therapy, and JTX-1811, which aims to selectively deplete T regulatory cells in the tumor microenvironment. The company also develops JTX-8064, an antibody targeting a receptor on macrophages. Founded in 2013 by experts in immunobiology and cancer research, Jounce Therapeutics is headquartered in Cambridge, Massachusetts, and is committed to improving patient outcomes through its proprietary immunotherapy platform.
Voyager Therapeutics is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe neurological diseases. The company's lead candidate, VY-AADC, is currently undergoing an open-label Phase 1b clinical trial for Parkinson's disease. In addition to this, Voyager's preclinical pipeline includes therapies targeting amyotrophic lateral sclerosis, Huntington's disease, Friedreich's ataxia, and various tauopathies and synucleinopathies, which encompass conditions like Alzheimer's disease and Lewy Body Dementia. Voyager Therapeutics emphasizes advancing adeno-associated virus (AAV) gene therapy through innovations in vector optimization, dosing techniques, and production processes. The company has established strategic collaborations with the University of Massachusetts and ClearPoint Neuro, along with partnerships with Brammer Bio and Fujifilm Diosynth Biotechnologies to enhance its gene therapy programs. Furthermore, Voyager has a collaboration agreement with Neurocrine Biosciences for the research, development, and commercialization of AAV-based gene therapies. Founded in 2013, Voyager Therapeutics is committed to addressing critical unmet medical needs in the field of central nervous system disorders.
Nivalis Therapeutics is focused on developing innovative therapies aimed at treating cystic fibrosis (CF) by preserving intracellular S-nitrosoglutathione (GSNO), a molecule involved in cell signaling and implicated in the disease's pathophysiology. The company's lead candidate, N91115, specifically targets patients with the F508del mutation, the most prevalent mutation associated with CF. Founded in 2007 and headquartered in Boulder, Colorado, Nivalis Therapeutics is committed to improving the lives of individuals affected by cystic fibrosis and supporting their families through its research and development efforts.
Blueprint Medicines Corporation is a biopharmaceutical company that specializes in developing small molecule kinase inhibitors aimed at treating cancers and rare diseases driven by genomic abnormalities. The company’s pipeline includes avapritinib, which targets systemic mastocytosis and gastrointestinal stromal tumors, and BLU-263, an oral KIT inhibitor for indolent systemic mastocytosis and other mast cell disorders. Additionally, fisogatinib is in Phase I trials for advanced hepatocellular carcinoma, while pralsetinib targets RET-altered non-small cell lung cancer and medullary thyroid carcinoma. Other investigational compounds include BLU-782, aimed at treating fibrodysplasia ossificans progressive. Established in 2008 and based in Cambridge, Massachusetts, Blueprint Medicines utilizes its proprietary chemical library and Insights-to-Validation Platform to develop personalized therapies that address specific molecular drivers of cancer and the challenges posed by resistance mechanisms. The company has formed collaboration agreements with several pharmaceutical entities to enhance its research and development efforts.
Invitae is a genetic information company focused on integrating genetic testing into routine medical practice to enhance healthcare quality worldwide. It specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates a wide array of genetic tests into one platform, ensuring faster turnaround times and lower costs compared to traditional single-gene tests. Invitae's test offerings cover various hereditary conditions, including cancers, neurological disorders, cardiovascular diseases, pediatric and metabolic disorders. The company aims to make genetic testing more affordable and accessible, develop a secure infrastructure for genome management, and foster a global community for sharing genetic information to advance scientific research and medical practices. By lowering the barriers to obtaining diagnostic genetic information, Invitae seeks to improve healthcare outcomes for millions of individuals.
Adaptimmune Therapeutics plc is a clinical-stage biopharmaceutical company based in Abingdon, United Kingdom, specializing in innovative cell therapies for cancer treatment, particularly targeting solid tumors. The company utilizes its specific peptide enhanced affinity receptor (SPEAR) T-cell platform to identify and genetically engineer T-cell receptors that enhance the immune response against cancerous cells. Current clinical trials are exploring various therapeutic candidates, including ADP-A2M4 for multiple solid tumor types and ADP-A2AFP for hepatocellular carcinoma. Adaptimmune has formed strategic collaborations with several organizations, including GSK for the development of NY-ESO T-cell therapy and Noile-Immune Biotech Inc. for combining SPEAR T-cells with PRIME technology. Founded in 2008, Adaptimmune is committed to advancing T-cell therapies to improve patient outcomes in oncology and beyond.
Civitas Therapeutics is a biopharmaceutical company dedicated to developing therapies for pulmonary delivery. The company focuses on creating innovative treatments for central nervous system and respiratory disorders, with a particular emphasis on conditions such as multiple sclerosis and spinal cord injuries. Civitas Therapeutics aims to improve the health and quality of life for patients across the United States through its specialized drug delivery systems.
Spark Therapeutics, Inc. is dedicated to developing gene therapy products aimed at treating debilitating genetic diseases. The company is known for LUXTURNA, a treatment for patients with biallelic RPE65 mutation-associated retinal dystrophy, and has an extensive pipeline that includes candidates like SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. Additionally, Spark is exploring liver-directed gene therapies such as SPK-3006 for Pompe disease and has programs targeting neurodegenerative diseases, including Huntington's disease and a form of Batten disease. Spark Therapeutics also engages in collaborations, notably with Pfizer for the SPK-FIX program for hemophilia B, and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Established in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical studies across various therapeutic areas.