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.
AlloVir, Inc. is a clinical-stage cell therapy company based in Cambridge, Massachusetts, specializing in the development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies for viral-associated diseases. Its lead product, Viralym-M, targets several viruses including BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. The company's pipeline also includes candidates for treating respiratory syncytial virus, influenza, SARS-CoV-2, hepatitis B, and human herpesvirus-8. AlloVir's proprietary VST therapy platform aims to provide accessible treatment options for patients facing severe viral infections, addressing the pressing need for effective therapies in this area. Founded in 2013, AlloVir was previously known as ViraCyte, Inc. and rebranded in May 2019.
Gossamer Bio is a San Diego-based company focused on the discovery and development of novel and differentiated therapeutic products, to address high unmet needs amongst various targeted patient populations. Founded by the former Receptos executive team, Gossamer Bio’s strategy will be to leverage an asset-rich in-licensing environment, with a focus on areas of high unmet need, utilizing a team with a strong track record of execution in immunology, inflammation, fibrosis and oncology.
Tempest Therapeutics is a biotechnology company that focuses on small molecule therapeutics that modulate anti-tumor immunity pathways. The company has a balanced and deep pipeline consisting of first-in-class and best-in-class small molecule therapeutics, which modulate distinct pathways relevant to mounting an effective anti-tumor response.
Tempest Therapeutics was established in 2011 and is headquartered in San Francisco, California.
22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.
Dewpoint Therapeutics, Inc. is a Boston-based biotechnology company focused on the research and development of biomolecular condensates to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunology, cardiovascular issues, women's health, and virology. Founded in 2018, the company utilizes a proprietary, AI-powered drug discovery platform that employs machine learning and advanced image analysis to visualize and manipulate biomolecular condensates. This technology aids in identifying novel drug candidates and has applications across various therapeutic areas. Dewpoint Therapeutics collaborates with leading academic and pharmaceutical partners to enhance its research and development efforts, aiming to deliver impactful healthcare solutions.
Pardes Biosciences is a biotechnology company that develops and commercializes direct-acting antivirals for the treatment of coronavirus. The Carlsbad, California-based company was established by Uri Lopatin in 2020.
Cyclerion Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery, development, and commercialization of innovative treatments for serious and orphan diseases. The company focuses on soluble guanylate cyclase (sGC) pharmacology to harness the therapeutic potential of next-generation sGC stimulators. Its key product candidates include Olinciguat, which is undergoing Phase II studies for sickle cell disease, and Praliciguat, currently in Phase II trials for diabetic nephropathy and heart failure with preserved ejection fraction. Additionally, Cyclerion is developing IW-6463, an orally administered central nervous system-penetrant sGC stimulator in Phase I trials aimed at treating neurodegenerative diseases. The company is also exploring liver-targeted and lung-targeted sGC stimulators to expand its therapeutic offerings. Cyclerion Therapeutics was incorporated in 2018 and is dedicated to addressing unmet medical needs through its innovative pipeline.
Ajax Therapeutics is an NYC-based biotechnology company applying computational chemistry and structure-based technologies to develop novel small molecules for hematologic malignancies
Corvus Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Burlingame, California, that specializes in developing innovative immuno-oncology therapies. The company's lead product candidate, Ciforadenant (CPI-444), is an oral small molecule antagonist of the A2A receptor and is currently undergoing Phase Ib/2 clinical trials targeting adenosine, an important immune checkpoint. Additionally, Corvus is advancing CPI-006, an anti-CD73 monoclonal antibody, which is in Phase I/Ib trials to inhibit adenosine production and stimulate immune responses, and is also being evaluated for its potential use in COVID-19. Other candidates in development include an antagonist of the A2B receptor and CPI-818, a small molecule inhibitor targeting interleukin-2-inducible T-cell kinase, which is also in Phase I/Ib trials. The company has established a strategic collaboration with Angel Pharmaceuticals to further develop its pipeline of targeted investigational medicines.
Janux Therapeutics develops immunotherapies that generate immune responses to prevent tumors and not affecting a patient’s healthy tissue. Janux technology targets all three stages of the anti-tumor immune response. Janux Therapeutics, Inc. was founded in 2017 by David Campbell and is based in La Jolla, California.
Tectonic is a privately held biotechnology company that aims to transform the discovery of novel GPCR-targeted drugs and unlock the therapeutic utility the most challenging receptors in the class. Tectonic’s founders are at the forefront of scientific research in biochemistry and molecular pharmacology with an extensive track record of entrepreneurial achievement.
Aktis Oncology is a biotechnology company focused on developing targeted radiopharmaceuticals for the treatment of various solid tumor cancers. The company has created proprietary platforms that generate tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered to achieve high penetration into tumors and prolonged residence time, allowing for effective tumor elimination while reducing side effects associated with treatment. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing the precision of cancer treatment.
Notch Therapeutics Inc. is an immune cell therapy company based in Toronto, Canada, focused on developing gene-edited allogenic T cell therapies for cancer treatment. Established in 2018, the company utilizes an induced pluripotent stem cell (iPSC) platform that enables precise control of Notch signaling. This innovative technology addresses key limitations in cell therapy development by allowing for the design and manufacture of a consistent supply of therapeutic T cells. Notch Therapeutics specializes in iPSC AlloCAR therapy products aimed at treating conditions such as non-Hodgkin lymphoma, leukemia, and multiple myeloma, enabling healthcare professionals to engineer stem cells tailored to the specific biological challenges presented by complex diseases.
Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, that specializes in developing vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. This approach allows for the combination of protective polysaccharides and proteins in a single vaccine, aiming to enhance the immune response against various infectious diseases. Affinivax focuses on a range of pathogens, including Streptococcus pneumoniae, which poses significant health risks to both children and adults. The company seeks to create vaccines that provide broader disease coverage and address infectious diseases lacking effective immunization strategies. Founded in 2014, Affinivax benefits from insights provided by leading experts in infectious diseases and vaccines, and it has secured support from the Bill & Melinda Gates Foundation, along with exclusive licensing rights from Boston Children’s Hospital for its MAPS technology.
Gritstone Bio is a biotechnology company focused on developing personalized cancer immunotherapies aimed at treating various cancer types and infectious diseases. The company is advancing its lead product candidates, GRANITE and SLATE, both of which are in Phase I/II clinical trials. GRANITE targets solid tumors, including metastatic non-small cell lung cancer and gastroesophageal cancer, while SLATE serves as an off-the-shelf immunotherapy for common solid tumors and mutation-positive cancers. Gritstone is committed to identifying therapeutic neo-antigens from individual patients' tumors to create novel treatments, leveraging the expertise of its scientific founders and management team. Founded in 2015 and headquartered in Emeryville, California, the company is also engaged in strategic collaborations to enhance its research and development efforts.
Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.
Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.
Cellares Corporation, founded in 2019 and based in South San Francisco, California, focuses on advancing cell therapy manufacturing for cancer treatment. The company aims to address the significant challenges associated with the production of cell therapies, which are often expensive and time-consuming due to their current one-patient-at-a-time manufacturing model. Cellares is developing The Cell Shuttle, an innovative automated and closed end-to-end manufacturing solution designed to enhance scalability and reduce costs. This system allows for the simultaneous production of multiple patient doses, increasing efficiency by tenfold compared to traditional methods. Additionally, The Cell Shuttle is expected to decrease process failure rates by three times and reduce manufacturing costs per patient by up to 70%. By integrating advanced technologies into the production process, Cellares is committed to accelerating access to life-saving therapies for patients in need.
Dewpoint Therapeutics, Inc. is a Boston-based biotechnology company focused on the research and development of biomolecular condensates to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunology, cardiovascular issues, women's health, and virology. Founded in 2018, the company utilizes a proprietary, AI-powered drug discovery platform that employs machine learning and advanced image analysis to visualize and manipulate biomolecular condensates. This technology aids in identifying novel drug candidates and has applications across various therapeutic areas. Dewpoint Therapeutics collaborates with leading academic and pharmaceutical partners to enhance its research and development efforts, aiming to deliver impactful healthcare solutions.
Silverback Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing tissue-targeted therapeutics for cancer, chronic viral infections, and other serious diseases. The company's lead product candidate, SBT6050, is currently in a Phase I/Ib clinical trial. It is a TLR8 agonist linker-payload conjugated to a HER2-directed monoclonal antibody, specifically targeting tumors such as breast, gastric, and non-small cell lung cancers. Additionally, Silverback is advancing SBT6290, a preclinical candidate that targets Nectin4, expressed in various cancers, and SBT8230, aimed at treating chronic hepatitis B virus infection. Utilizing its proprietary ImmunoTAC technology, Silverback aims to develop a new generation of therapies that can be systemically delivered yet act locally at disease sites, thereby modulating critical disease pathways that were previously considered inaccessible.
Eledon Pharmaceuticals, Inc. is a specialty pharmaceutical company dedicated to developing products for patients with ear, nose, and throat disorders. The company's lead product, OP0201, is a surfactant-based nasal aerosol designed for individuals at risk of or suffering from otitis media, which is inflammation of the middle ear. In addition to OP0201, Eledon has developed foam-based drug delivery technologies, OP0101 and OP0102, aimed at administering medications to the ear, nose, and sinus cavities. Founded in 2004 and initially known as Novus Therapeutics, the company rebranded in January 2021 and is headquartered in Irvine, California.
Neogene Therapeutics, Inc. is a biotechnology company based in New York that focuses on the development of T cell therapies to treat cancer. Founded in 2018, the company specializes in creating personalized engineered T-cells that target neo-antigens, which are mutated proteins resulting from DNA alterations in cancer cells. Neogene's innovative platform allows for the isolation of T cell receptor (TCR) genes specific to these neo-antigens from tumor biopsies, which are routinely collected from patients. By utilizing advanced DNA sequencing and genetic screening technologies, Neogene can accurately identify and engineer TCRs that enhance the ability of T-cells to detect and eliminate cancer cells. This approach aims to improve treatment outcomes for cancer patients by providing therapies tailored to their unique tumor profiles.
Chinook Therapeutics is a biotechnology company that specializes in the fields of therapeutics and life science. The company focuses on developing precision medicines for kidney diseases. Its services offers utilization of novel translational platforms and patient stratification tools, and emergence of accelerated regulatory pathways based on surrogate endpoints.
The company was founded in 2019 and headquartered in Alberta, Canada.
Alpine Immune Sciences, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, specializing in the discovery and development of protein-based immunotherapies aimed at treating cancer, autoimmune, and inflammatory disorders. The company's lead programs include ALPN-101, an antagonist targeting the inducible T cell costimulator and CD28 pathways for autoimmune and inflammatory diseases, and ALPN-202, which combines programmed cell death protein ligand 1 and cytotoxic T-lymphocyte associated protein 4 antagonism with PD-L1 dependent CD28 costimulation for cancer treatment. Additionally, Alpine has a collaboration with Kite Pharma, Inc. to develop immunotherapies that target the immune synapse for cancer therapy. The company utilizes a proprietary platform to transform native immune system proteins into innovative, multi-targeted therapeutics.
Shattuck Labs, Inc. is a clinical-stage biotechnology company based in Austin, Texas, focused on developing innovative therapeutics for cancer and autoimmune diseases. Founded in 2016, the company leverages its proprietary Agonist Redirected Checkpoint (ARC) platform to create dual-function fusion proteins that enhance immune responses against tumors. Its lead product candidate, SL-172154, is currently undergoing a Phase 1 clinical trial for ovarian cancer, designed to inhibit the CD47/SIRPa checkpoint interaction while activating the CD40 costimulatory receptor. Additionally, Shattuck collaborates with Takeda Pharmaceuticals on SL-279252, also in Phase 1 trials, which targets the PD-1/PD-L1 interaction and activates the OX40 receptor to improve anti-tumor immunity. Through its innovative approaches, Shattuck Labs aims to advance the field of immuno-oncology and provide effective treatments for various malignancies.
Private Equity Round in 2020
Surface Oncology was created to advance next-generation approaches to cancer immunotherapy based on proprietary insights about novel immunotherapy targets and emerging areas of cancer immuno-biology. Together with world-leading scientific founders, the Company is advancing multiple preclinical programs that target novel mechanisms shown to play vital roles in tumor immune-evasion. Surface is based in Cambridge, Mass.
Accent Therapeutics, Inc. is a biopharmaceutical company based in Lexington, Massachusetts, focused on developing small molecule therapies for oncology through the innovative field of epitranscriptomics. Founded in 2017, the company explores post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting RNA-modifying proteins associated with cancer, Accent Therapeutics aims to translate advanced scientific research into effective treatments for patients, ultimately providing new therapeutic options in the fight against cancer.
ElevateBio, LLC, based in Cambridge, Massachusetts, is a holding company focused on the development of cell and gene-based therapies. Established in 2017, ElevateBio operates a portfolio of subsidiary companies that aim to create innovative treatments for cancer and viral infections. The company partners with leading scientists and inventors to foster its portfolio and has established a centralized facility designed to streamline the translation of research and development into commercially viable therapies. This integrated approach enables the efficient development, manufacturing, and commercialization of life-transforming medicines, ultimately helping to provide patients with effective treatments for severe diseases.
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.
Pieris Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on discovering and developing anticalin-based therapeutics. Anticalins are low molecular-weight proteins derived from lipocalins, which are naturally occurring proteins in the human body. The company’s lead respiratory candidate, PRS-060, is in Phase I trials for asthma and other inflammatory diseases, while its lead immuno-oncology candidate, PRS-343, is also in Phase I trials targeting various cancers. Additionally, Pieris is advancing PRS-080, a treatment for functional iron deficiency in patients with chronic kidney disease, currently in Phase IIa trials, and PRS-344, a bispecific anticalin-antibody fusion protein in preclinical development for oncology. The company collaborates with several partners, including Les Laboratoires Servier, AstraZeneca, and Seattle Genetics, and has research collaborations with academic institutions such as the University of Pittsburgh. Founded in 2000, Pieris Pharmaceuticals aims to address critical medical needs through its innovative drug development approach.
AlloVir, Inc. is a clinical-stage cell therapy company based in Cambridge, Massachusetts, specializing in the development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies for viral-associated diseases. Its lead product, Viralym-M, targets several viruses including BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. The company's pipeline also includes candidates for treating respiratory syncytial virus, influenza, SARS-CoV-2, hepatitis B, and human herpesvirus-8. AlloVir's proprietary VST therapy platform aims to provide accessible treatment options for patients facing severe viral infections, addressing the pressing need for effective therapies in this area. Founded in 2013, AlloVir was previously known as ViraCyte, Inc. and rebranded in May 2019.
ElevateBio, LLC, based in Cambridge, Massachusetts, is a holding company focused on the development of cell and gene-based therapies. Established in 2017, ElevateBio operates a portfolio of subsidiary companies that aim to create innovative treatments for cancer and viral infections. The company partners with leading scientists and inventors to foster its portfolio and has established a centralized facility designed to streamline the translation of research and development into commercially viable therapies. This integrated approach enables the efficient development, manufacturing, and commercialization of life-transforming medicines, ultimately helping to provide patients with effective treatments for severe diseases.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Ashvattha Therapeutics, Inc. is a pharmaceutical company focused on developing innovative products for neurology, ophthalmology, and inflammation-related conditions. Established in 2015 and headquartered in Redwood City, California, with research facilities in Baltimore, Maryland, the company aims to create therapeutics that precisely target human diseases. This approach is designed to minimize off-target toxicity, systemic side effects, and immunogenicity often associated with other treatment platforms. Ashvattha Therapeutics has developed a diverse pipeline of nine therapeutics that span the fields of neurology, oncology, and ophthalmology, reflecting its commitment to advancing targeted treatment options for patients.
Peloton Therapeutics, Inc. is a clinical stage biopharmaceutical company based in Dallas, Texas, focused on developing innovative medicines for cancer and other serious conditions. The company's lead candidate, PT2977, is an oral HIF-2a inhibitor currently undergoing Phase 2 clinical trials for advanced or metastatic clear cell renal cell carcinoma. Additionally, Peloton is exploring PT2977 in various other indications, including von Hippel-Lindau disease-associated renal cell carcinoma and glioblastoma multiforme, as well as in combination therapies for metastatic renal cell carcinoma. Furthermore, Peloton is developing PT2567, another oral HIF-2a inhibitor in preclinical stages aimed at non-oncology applications, notably pulmonary arterial hypertension. Founded in 2010 and initially known as Damascus Pharmaceutics, Peloton Therapeutics has formed strong partnerships with academic institutions and secured funding from organizations like the Cancer Prevention Research Institute of Texas, positioning itself as a key player in the biotech sector.
Neurogene Inc. is a clinical-stage biotechnology company focused on developing genetic medicines for neurological diseases. Founded in 2018 and based in New York, the company employs adeno-associated virus (AAV) technology to create treatments for conditions such as Charcot-Marie Tooth disease (CMT4J), Aspartylglucosaminuria (AGU), and various lysosomal storage diseases. Neurogene aims to improve patient outcomes by providing effective gene therapies for rare neurological disorders that currently lack viable treatment options. Through its innovative approaches, the company seeks to enhance the quality of life for patients and their families affected by these debilitating conditions.
Dewpoint Therapeutics, Inc. is a Boston-based biotechnology company focused on the research and development of biomolecular condensates to create innovative treatments for a range of diseases, including cancer, neurodegenerative disorders, immunology, cardiovascular issues, women's health, and virology. Founded in 2018, the company utilizes a proprietary, AI-powered drug discovery platform that employs machine learning and advanced image analysis to visualize and manipulate biomolecular condensates. This technology aids in identifying novel drug candidates and has applications across various therapeutic areas. Dewpoint Therapeutics collaborates with leading academic and pharmaceutical partners to enhance its research and development efforts, aiming to deliver impactful healthcare solutions.
Relay Therapeutics is a clinical-stage precision medicines company based in Cambridge, Massachusetts, focused on transforming the drug discovery process, particularly in targeted oncology. The company employs an innovative allosteric drug-discovery platform that integrates computational techniques with experimental methods to analyze protein motion and interactions. This approach aids in the development of therapies for complex diseases, including cancer. Relay's product candidates include RLY-1971, an oral small molecule inhibitor targeting SHP2, currently undergoing Phase 1 trials for advanced solid tumors; RLY-4008, a selective inhibitor of FGFR2 for tumors with specific oncogenic alterations; and RLY-PI3K1047, which targets cancer-associated mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, Relay Therapeutics is dedicated to enhancing small molecule therapeutic discovery through its advanced platform and targeted treatment strategies.
Avidity Biosciences is a biopharmaceutical company specializing in the development of oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disorder. The company's development pipeline also includes programs focused on treating various muscle diseases, such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Additionally, Avidity is exploring therapeutic applications for immune and other cell types, leveraging its proprietary AOC platform to reach previously undruggable tissues and effectively target the genetic underpinnings of these conditions. Founded in 2012, Avidity Biosciences is headquartered in La Jolla, California.
Morphic Therapeutic is a biotechnology company focused on developing oral small-molecule integrin therapeutics for various medical conditions, including autoimmune diseases, cardiovascular issues, metabolic disorders, fibrosis, and cancer. Its lead candidates in clinical development include MORF-720, an avß6 specific integrin inhibitor targeting idiopathic pulmonary fibrosis and primary sclerosing cholangitis, and MORF-057, an a4ß7 specific integrin inhibitor for inflammatory bowel disease. The company is also exploring additional integrin inhibitors for similar applications. Founded in 2015, Morphic Therapeutic has established collaborations with various organizations, including Engitix Ltd, Schrödinger, LLC, AbbVie Biotechnology Ltd, and Janssen Pharmaceuticals, to advance its research and development efforts. The company is headquartered in Waltham, Massachusetts, and leverages scientific discoveries made by its founder Tim Springer to innovate in the field of integrin therapies.
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.
Accent Therapeutics, Inc. is a biopharmaceutical company based in Lexington, Massachusetts, focused on developing small molecule therapies for oncology through the innovative field of epitranscriptomics. Founded in 2017, the company explores post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting RNA-modifying proteins associated with cancer, Accent Therapeutics aims to translate advanced scientific research into effective treatments for patients, ultimately providing new therapeutic options in the fight against cancer.
Magenta is focused on improving the patient experience in transplant medicine, with the goal of bringing this lifesaving procedure to more patients. Over the past 50 years, stem cell transplantation has been used in more than 1M patients worldwide. It is currently the only treatment with the potential to cure some of the most devastating hematologic cancers and autoimmune diseases. Despite the increased use and clinical advancements over the past several decades, stem cell transplant remains a high-risk last-resort therapeutic option due to the toxicity and potential long-term side effects.
Omniox is a biopharmaceutical company focused on developing innovative treatments for hypoxic diseases, which occur when tissues receive insufficient oxygen. This condition is linked to serious health issues, including various cancers and cardiovascular diseases. The company utilizes a proprietary protein-based platform technology known as H-NOX, which is designed to deliver oxygen and nitric oxide directly to hypoxic tissues. By specifically targeting these areas, Omniox aims to restore normal tissue function, potentially improving patient outcomes and offering new therapeutic options. Through its work, Omniox seeks to advance oxygen-based therapies that address the challenges posed by diseases associated with low oxygen levels.
Unity Biotechnology is a biotechnology company dedicated to the research and development of therapeutics aimed at extending human health span by addressing diseases associated with aging. The company specializes in clearing senescent cells through the creation of senolytic medicines, which target vulnerabilities specific to these cells while preserving normal ones. Its lead drug candidates include UBX0101, currently undergoing Phase II trials for musculoskeletal disorders and Phase Ib trials for moderate-to-severe knee osteoarthritis, and UBX1325, which is in Phase I trials for diabetic macular edema. Additionally, Unity is exploring treatments for various age-related conditions, including ophthalmologic, pulmonary, kidney, and liver diseases. Founded in 2009 and based in South San Francisco, California, Unity Biotechnology was formerly known as Forge, Inc. and rebranded in 2015.
Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.
Scholar Rock is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapies targeting growth factor signaling in serious diseases. The company’s lead product candidate, SRK-015, is a selective inhibitor of myostatin activation and is currently undergoing Phase II clinical trials for spinal muscular atrophy. Additionally, Scholar Rock is advancing SRK-181, which is in Phase I trials for treating cancers resistant to checkpoint inhibitors. The company employs a proprietary platform to design monoclonal antibodies that selectively modulate the activation of growth factors in disease microenvironments, addressing challenges associated with traditional growth factor inhibition. Scholar Rock is also developing a pipeline of candidates for a variety of conditions, including neuromuscular disorders, cancer, fibrosis, and anemia. Furthermore, the company has a collaboration with Gilead Sciences to explore specific inhibitors of transforming growth factor beta for fibrotic diseases. Founded in 2012, Scholar Rock aims to improve patient outcomes through its targeted therapeutic approach.
Relay Therapeutics is a clinical-stage precision medicines company based in Cambridge, Massachusetts, focused on transforming the drug discovery process, particularly in targeted oncology. The company employs an innovative allosteric drug-discovery platform that integrates computational techniques with experimental methods to analyze protein motion and interactions. This approach aids in the development of therapies for complex diseases, including cancer. Relay's product candidates include RLY-1971, an oral small molecule inhibitor targeting SHP2, currently undergoing Phase 1 trials for advanced solid tumors; RLY-4008, a selective inhibitor of FGFR2 for tumors with specific oncogenic alterations; and RLY-PI3K1047, which targets cancer-associated mutant variants of phosphoinositide 3-kinase alpha. Founded in 2015, Relay Therapeutics is dedicated to enhancing small molecule therapeutic discovery through its advanced platform and targeted treatment strategies.
Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.
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.
aTyr Pharma is a biotherapeutics company that discovers and develops protein biologics for human therapeutics. The company has established a dominant intellectual property estate surrounding Physiocrine-based compositions and potential therapeutic applications. aTyr’s key programs are focused on immunomodulation disorders in the areas of inflammation and immunity and are in preclinical development.
The company's primary focus is ATYR1923, a clinical-stage product candidate which binds to the neuropilin-2 receptor and is designed to down-regulate immune engagement in interstitial lung diseases.
The privately held biotech was founded by The Scripps Research Institute Professor Paul Schimmel, a leading aminoacyl tRNA synthetase scientist, and is backed by top life sciences investors Alta Partners, Cardinal Partners, Domain Associates and Polaris Ventures.
Kezar Life Sciences, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing novel small molecule therapeutics for unmet medical needs in autoimmunity and cancer. The company’s lead candidate, KZR-616, is a selective immunoproteasome inhibitor currently undergoing Phase 2 clinical trials for multiple autoimmune conditions, including lupus nephritis and autoimmune hemolytic anemia, as well as Phase 1b/2 trials for systemic lupus erythematosus. In addition to KZR-616, Kezar is advancing preclinical products, including KZR-TBD, aimed at treating oncology and autoimmune disorders. Founded in 2015, Kezar Life Sciences leverages insights into protein homeostasis, particularly the interplay between protein degradation and secretion, to drive its drug discovery efforts.
Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.
Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.
ORIC Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing therapies to overcome resistance in cancer treatment. Founded in 2014 and based in South San Francisco, California, the company is advancing several product candidates, including ORIC-101, a small molecule antagonist of the glucocorticoid receptor associated with resistance to various cancer therapies in solid tumors. Another key candidate, ORIC-533, is an orally bioavailable small molecule inhibitor of CD73, which plays a significant role in resistance to chemotherapy and immunotherapy regimens. In addition to these, ORIC is working on multiple precision medicines aimed at addressing other cancer resistance mechanisms. The company's founders, Charles Sawyers and Scott Lowe, are recognized for their contributions to identifying novel cancer targets that have led to innovative treatment options.
Intellia Therapeutics is a genome editing company based in Cambridge, Massachusetts, focused on developing curative gene-editing treatments using the CRISPR/Cas9 technology. The company aims to address severe and life-threatening genetic diseases through both in vivo and ex vivo therapeutic approaches. Its pipeline includes programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia is developing engineered cell therapies for oncological and autoimmune diseases. The company has established collaborations with various entities, including leading pharmaceutical firms and research institutions, to enhance its research and development efforts. Founded in 2014, Intellia Therapeutics holds a comprehensive intellectual property platform for the therapeutic application of CRISPR technology, positioning it at the forefront of gene editing innovation.
Syndax Pharmaceuticals is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of innovative cancer therapies. Its lead product candidate, entinostat, is a class I HDAC inhibitor currently undergoing Phase III trials for advanced hormone receptor-positive, HER2-negative breast cancer. Additionally, the company is developing SNDX-5613, an inhibitor targeting the Menin-MLL binding interaction, in Phase I/II trials for specific types of acute leukemia. Syndax's pipeline also includes SNDX-6352, a monoclonal antibody designed to block the CSF-1 receptor, which is being tested for chronic graft versus host disease and various solid tumors. The company is pursuing multiple clinical collaborations and agreements with notable organizations, including MSD International and the National Cancer Institute, to enhance its research and development efforts. Founded in 2005, Syndax is dedicated to advancing treatment options for patients with solid tumors and hematological cancers.
Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.
Clementia Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Montreal, Canada, focused on developing innovative treatments for patients with ultra-rare bone disorders and other diseases characterized by high unmet medical needs. Founded in 2010, the company specializes in disease-modifying therapies, with its lead product candidate being palovarotene, an oral small molecule. Palovarotene has demonstrated significant potential in preventing abnormal bone formation and fibrosis across various tissues. The candidate is currently undergoing evaluation in the Phase III MOVE trial, targeting conditions such as fibrodysplasia ossificans progressive and multiple osteochondromas, as well as dry eye disease. Clementia's commitment to addressing debilitating health issues highlights its role in advancing treatment options for affected patients.
Kezar Life Sciences, Inc. is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing novel small molecule therapeutics for unmet medical needs in autoimmunity and cancer. The company’s lead candidate, KZR-616, is a selective immunoproteasome inhibitor currently undergoing Phase 2 clinical trials for multiple autoimmune conditions, including lupus nephritis and autoimmune hemolytic anemia, as well as Phase 1b/2 trials for systemic lupus erythematosus. In addition to KZR-616, Kezar is advancing preclinical products, including KZR-TBD, aimed at treating oncology and autoimmune disorders. Founded in 2015, Kezar Life Sciences leverages insights into protein homeostasis, particularly the interplay between protein degradation and secretion, to drive its drug discovery efforts.
Nabriva Therapeutics is a biopharmaceutical company focused on developing and commercializing innovative anti-infective agents to address serious bacterial infections. The company's lead product, lefamulin, is a semi-synthetic pleuromutilin antibiotic designed to treat community-acquired bacterial pneumonia and acute bacterial skin infections. It is currently undergoing clinical trials for additional applications, including pediatric infections, sexually transmitted infections, osteomyelitis, and prosthetic joint infections. Nabriva is also advancing CONTEPO, an epoxide antibiotic aimed at treating complicated urinary tract infections, and it is in clinical trials for peri-operative prophylaxis. Founded in 2005 and headquartered in Dublin, Ireland, Nabriva Therapeutics has evolved from its earlier identity as a research institute focused on antibiotic development.
aTyr Pharma is a biotherapeutics company that discovers and develops protein biologics for human therapeutics. The company has established a dominant intellectual property estate surrounding Physiocrine-based compositions and potential therapeutic applications. aTyr’s key programs are focused on immunomodulation disorders in the areas of inflammation and immunity and are in preclinical development.
The company's primary focus is ATYR1923, a clinical-stage product candidate which binds to the neuropilin-2 receptor and is designed to down-regulate immune engagement in interstitial lung diseases.
The privately held biotech was founded by The Scripps Research Institute Professor Paul Schimmel, a leading aminoacyl tRNA synthetase scientist, and is backed by top life sciences investors Alta Partners, Cardinal Partners, Domain Associates and Polaris Ventures.
Kura Oncology, Inc., a clinical-stage biopharmaceutical company, develops medicines for the treatment of cancer. Its pipeline consists of small molecule product candidates that target cancer. The company’s lead product candidate is Tipifarnib, an orally bioavailable inhibitor of farnesyl transferase that is in Phase II clinical trials for the treatment of solid tumors, peripheral T-cell lymphomas, and other hematologic malignancies. It is also developing KO-947, a small molecule inhibitor of extracellular signal related kinase used for the treatment of patients with tumors that have dysregulated activity due to mutations or other mechanisms in the mitogen-activated protein kinase pathway; and KO-539, a small molecule inhibitor of the menin-mixed lineage leukemia protein-protein interaction. Kura Oncology, Inc. was founded in 2014 and is headquartered in San Diego, California.
Collegium Pharmaceutical is a specialty pharmaceutical company that develops and commercializes products aimed at treating chronic pain while addressing the concerns of prescription drug abuse. The company utilizes its patented DETERx platform technology to create abuse-deterrent formulations that maintain the extended-release profiles and safety of medications despite potential misuse methods such as chewing or crushing. Collegium's product lineup includes Xtampza ER, an extended-release oral formulation of oxycodone designed to deter abuse, as well as Nucynta ER and Nucynta IR, which are extended-release and immediate-release formulations of tapentadol respectively, for managing severe pain and acute pain in adults. Founded in 2002 and headquartered in Stoughton, Massachusetts, Collegium focuses on delivering innovative solutions for patients with chronic pain, utilizing formulation improvements backed by strong intellectual property protections.
Naurex Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for depression and other central nervous system disorders. The company specializes in a new approach to modulating the N-methyl-D-aspartic acid receptor (NMDAR), resulting in the creation of a unique class of compounds called glycine-site functional partial agonists (GFPAs). These GFPAs interact with the NMDAR in a manner distinct from traditional agents, potentially offering new treatment options for patients. Naurex's drug development pipeline includes several programs, highlighted by the first-generation molecule GLYX-13 and the second-generation NRX-1050 series, along with additional compounds derived from its platform of novel NMDAR modulators. Through this research, Naurex aims to address the challenges posed by various CNS disorders.
Scholar Rock is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapies targeting growth factor signaling in serious diseases. The company’s lead product candidate, SRK-015, is a selective inhibitor of myostatin activation and is currently undergoing Phase II clinical trials for spinal muscular atrophy. Additionally, Scholar Rock is advancing SRK-181, which is in Phase I trials for treating cancers resistant to checkpoint inhibitors. The company employs a proprietary platform to design monoclonal antibodies that selectively modulate the activation of growth factors in disease microenvironments, addressing challenges associated with traditional growth factor inhibition. Scholar Rock is also developing a pipeline of candidates for a variety of conditions, including neuromuscular disorders, cancer, fibrosis, and anemia. Furthermore, the company has a collaboration with Gilead Sciences to explore specific inhibitors of transforming growth factor beta for fibrotic diseases. Founded in 2012, Scholar Rock aims to improve patient outcomes through its targeted therapeutic approach.
Flex Pharma, Inc. is a biotechnology company focused on developing and commercializing treatments for muscle cramps, spasms, and spasticity linked to neurological conditions and exercise. The company operates in two segments: Consumer Operations and Drug Development. Its lead drug candidate, FLX-787, is a dual transient receptor potential V1/A1 ion channel activator, which has completed an exploratory Phase II clinical trial in Australia targeting multiple sclerosis patients. Additionally, Flex Pharma offers HOTSHOT, a consumer beverage designed to prevent and treat exercise-associated muscle cramps. The company markets HOTSHOT products online through its direct-to-consumer website and third-party platforms that provide international shipping. Founded in 2014 and based in Boston, Massachusetts, Flex Pharma was acquired by Salarius Pharmaceuticals in 2019.
SAGE Therapeutics is a biopharmaceutical company focused on developing and commercializing innovative treatments for central nervous system (CNS) disorders. Its lead product, ZULRESSO, is an intravenous formulation of brexanolone designed for postpartum depression. The company's pipeline includes SAGE-217, a neuroactive steroid in Phase III trials for various depressive disorders and anxiety, and SAGE-324, currently in Phase II trials for essential tremors and previously tested for other neurological conditions. Additionally, SAGE-718, which targets multiple CNS disorders, has completed Phase I trials, while SAGE-904 and SAGE-689 are in early clinical stages for NMDA-related disorders. SAGE Therapeutics has formed strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. for the development and commercialization of its key product candidates. Founded in 2010 and headquartered in Cambridge, Massachusetts, the company aims to address the unmet needs of patients affected by debilitating CNS disorders through scientific innovation and strategic partnerships.
Atara Biotherapeutics is an off-the-shelf T-cell immunotherapy company based in South San Francisco, California, focused on developing innovative treatments for patients with cancer, autoimmune diseases, and viral infections. The company's lead product, tabelecleucel (tab-cel), is currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, with additional development for other EBV-related hematologic malignancies and solid tumors, such as nasopharyngeal carcinoma. Atara is also advancing next-generation CAR T immunotherapies, addressing various conditions including mesothelin-targeted therapies for solid tumors and treatments for B-cell lymphomas. Other notable candidates include therapies for multiple sclerosis and several viral infections. The company collaborates with esteemed institutions such as Memorial Sloan Kettering Cancer Center and Bayer AG to enhance its research and development efforts. Founded in 2012, Atara Biotherapeutics is committed to transforming the treatment landscape for serious diseases through its robust clinical and scientific initiatives.
Atara Biotherapeutics is an off-the-shelf T-cell immunotherapy company based in South San Francisco, California, focused on developing innovative treatments for patients with cancer, autoimmune diseases, and viral infections. The company's lead product, tabelecleucel (tab-cel), is currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, with additional development for other EBV-related hematologic malignancies and solid tumors, such as nasopharyngeal carcinoma. Atara is also advancing next-generation CAR T immunotherapies, addressing various conditions including mesothelin-targeted therapies for solid tumors and treatments for B-cell lymphomas. Other notable candidates include therapies for multiple sclerosis and several viral infections. The company collaborates with esteemed institutions such as Memorial Sloan Kettering Cancer Center and Bayer AG to enhance its research and development efforts. Founded in 2012, Atara Biotherapeutics is committed to transforming the treatment landscape for serious diseases through its robust clinical and scientific initiatives.