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.
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.
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.
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.
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.
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.
LianBio is a biopharmaceutical company based in Shanghai, China, with an additional office in Princeton, New Jersey. Founded in 2019, the company focuses on the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as other medical conditions. LianBio's mission is to enhance access to innovative medicines by partnering with leading organizations in the field to leverage advanced scientific discoveries. The company is dedicated to addressing unmet medical needs in Greater China and other Asian markets by in-licensing assets and building a diverse pipeline of clinically validated product candidates. Its portfolio aims to establish new standards of care across various therapeutic areas, including cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory conditions.
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.
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.
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.
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.
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.
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.
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.
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.
Mersana Therapeutics employs its biodegradable polymer platform to create new and better medicines. They are advancing their own clinical-stage pipeline of novel compounds with the potential to address multiple oncology indications.
It was founded in 2005 and headquartered in Cambridge, Massachusetts.
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.
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.
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.
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.