EcoR1 Capital

EcoR1 Capital, LLC is an employee owned hedge fund sponsor. The firm provides its services to pooled investment vehicles. The firm also manages separate client-focused equity and fixed income portfolios. The firm invests in the public equity markets of the United States. It employs long/short strategy to make its investments. The firm typically invests in companies in biotechnology sector. EcoR1 Capital, LLC was founded in 2012 and is based in San Francisco, California.

Oleg Nodelman

Founder and Portfolio Manager

Scott Perlen

CFO

Caroline Stout

Partner

135 past transactions

Nurix Therapeutics

Venture Round in 2020
Nurix develops small molecule inhibitors for the treatment of proliferative and degenerative diseases. The company’s product engine leverages a deep mechanistic understanding of the UPS to deliver effective small molecule drug candidates with the potential to make a dramatic difference for patients. Nurix drugs control ubiquitin E3 ligases, the key enzymes responsible for protein breakdown in human cells, as a unique therapeutic approach to treat a broad range of diseases. The company discovers drugs that harness the body's natural process to control protein levels. Their focus is on developing drugs to treat cancer including the novel, small molecule immuno-oncology agents. Nurix was founded in 2009 by internationally recognized experts in the ubiquitin-proteasome field and is funded by leading life science investors, Third Rock Ventures, and The Column Group. The company is headquartered in San Francisco, California.

aTyr Pharma

Series E in 2015
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.

Scholar Rock

Series C in 2018
Scholar Rock, Inc., a biotechnology company, discovers and develops a class of biologic therapies that selectively target dysregulated growth factors in the disease microenvironment by modulating supracellular activation. The company’s drug discovery programs target growth factors which are present in the microenvironments of significant diseases, including fibrosis, musculoskeletal diseases, immuno-oncology, and autoimmune diseases. Its lead products include SRK-015, a selective and local inhibitor of latent myostatin activation for the treatment of primary myopathies. The company was incorporated in 2012 and is based in Cambridge, Massachusetts.

Syndax Pharmaceuticals

Series C in 2015
Syndax Pharmaceuticals is a clinical-stage pharmaceutical company that focuses on an HDAC inhibitor for solid tumors and hematological tumors. It offers therapies to treat solid tumors and lung, breast, and hematological cancer. The company is developing axatilimab, a monoclonal antibody that blocks the colony-stimulating factor 1 (CSF-1) receptor, in development for chronic Graft versus Host Disease, and entinostat, a class I HDAC inhibitor. Syndax Pharmaceuticals pipeline includes and SNDX-5613, a highly selective inhibitor of the Menin–MLL binding interaction, in development for the treatment of patients with MLLr and NPM1 acute leukemias.

Naurex

Series C in 2014
Naurex Inc. is a clinical-stage company developing novel therapies for depression and other CNS disorders based on a new mechanism of action for modulating the N-methyl-D-aspartic acid receptor (NMDAR). Using these discoveries, Naurex researchers have generated novel chemical drug classes known as glycine-site functional partial agonists (GFPAs), which modulate the receptor in a different way than existing NMDAR agents. The company's drug development programs based on its GFPA NMDAR modulators include the first-generation molecule, GLYX-13, the second-generation NRX-1050 small molecule series and additional compounds from Naurex's platform of novel NMDAR modulators.

Pardes Biosciences

Post in 2021
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.

Atreca

Private Placement in 2018
Atreca is a biopharmaceutical that develops a technology to identify the set of antibodies produced during an immune response, without prior knowledge of an antigen. Its product candidate, ATRC-101, is a monoclonal antibody in preclinical development with a novel mechanism of action and target-derived from an antibody identified using its discovery platform.

Omniox

Series D in 2018
Omniox is a biopharmaceutical company developing new medicines for hypoxic diseases. Hypoxia, defined as insufficient levels of oxygen in tissues, is known to trigger or impact a range of serious illnesses and conditions including many cancers, cardiovascular diseases, and trauma. At the center of Omniox’ work is a protein-based platform technology (H-NOX), that is engineered to reverse hypoxic disease states by delivering oxygen or nitric oxide preferentially to hypoxic tissues.

ElevateBio

Private Placement in 2019
ElevateBio is a operator of a portfolio of cell and gene therapy companies intended to develop, manufacture and commercialize life-transforming medicines. The company creates a portfolio through partnerships with the world's most innovative scientists and inventors as well as it created a centralized facility that integrates all the elements that translate cell and gene therapy R&D into commercially viable therapies quickly and efficiently, enabling patients to get medical products to treat severe diseases.

Gossamer Bio

Post in 2022
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.

AlloVir

Post in 2022
AlloVir focuses on the development of cell therapies and focuses on restoring natural immunity against the virus-associated diseases. It provides an ideal environment for ViraCyte to develop and advance its product pipeline from discovery through late-stage clinical trials. The company was founded in 2013 and headquartered in Houston, Texas.
Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development of novel, first-in-class agents that target the immune system to treat patients with cancer. With accomplished and talented scientists, and top-tier investors, we are well positioned in an exciting new era of immuno-oncology.

Kezar Life Sciences

Series B in 2017
Kezar Life Sciences company focused on the discovery and development of drugs targeting protein homeostasis for autoimmune disorders. In parallel with this work, Christopher and Kezar co-founder Jack Taunton, Ph.D. collaborated on several research projects involving protein secretion. This collaboration inspired a belief in the strong synergies between protein degradation and protein secretion, two fertile areas for drug discovery with platform potential.

Morphic Therapeutic

Series B in 2018
Morphic Therapeutic is a biotechnology company developing a new generation of oral integrin therapies. Drawing on discoveries made in the lab of Scientific Founder Tim Springer, Morphic was created in 2015 with co-founders Polaris Partners, T.A. Springer and Schrödinger, Inc., along with ShangPharma Investment Group.
Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

Shattuck Labs

Series B in 2020
Shattuck Labs, Inc., a clinical-stage biotechnology company, develops therapeutics for the treatment of cancer and autoimmune disease in the United States. The company’s lead product candidate is SL-172154, which is in Phase 1 clinical trial for the treatment of ovarian cancer. It also holds a collaboration agreement with Takeda Pharmaceuticals to develop SL-279252 that is in Phase 1 clinical trial in patients with advanced solid tumors and lymphoma. The company was founded in 2016 and is headquartered in Austin, Texas.

Janux Therapeutics

Series B in 2021
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.

Fulcrum Therapeutics

Private Placement in 2020
Fulcrum Therapeutics is a biotechnology company developing new medicines to deliver a new future to patients. The company develops a proprietary product engine that employs to identify and validate cellular drug targets that can modulate gene expression. Its product candidate, losmapimod, is a small molecule that was developed for the treatment of FSHD, a muscle-wasting disorder that leads to physical impairments and disability. It is focused on improving the lives of patients with genetically defined diseases in areas of high unmet medical need, with an initial focus on rare diseases.

Ventyx Biosciences

Post in 2022
Ventyx Biosciences is a biotechnology company with a diverse portfolio of best-in-class highly differentiated clinical and pre-clinical stage programs focused on high-value targets.

Alpine Immune Sciences

Funding Round in 2020
Alpine Immune Sciences is an immunotherapy startup focused on developing recombinant, protein-based therapeutic solutions. It was founded on January 23, 2015, and is based in Seattle, Washington, United States.

Freenome

Private Placement in 2020
Freenome, Inc., is an artificial intelligence (AI) genomics biotech company. The company develops and provides a medical arti?cial intelligence platform for early detection of cancer. It develops blood tests for early-cancer screening and treatment selection. The company also provides clinical research services. Freenome, Inc. was founded in 2014 and is based in South San Francisco, California.

Nuvation Bio

Private Placement in 2019
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.

Prevail Therapeutics

Private Placement in 2019
Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company’s lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.

AlloVir

Private Placement in 2019
Allovir, Inc., a clinical-stage cell therapy company, engages in the research and development of allogeneic, off-the-shelf multi-virus specific T cell (VST) therapies to prevent and treat devastating viral-associated diseases. The company’s lead product is Viralym-M, an allogeneic, off-the-shelf VST therapy, to treat BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, and human herpesvirus 6. Its preclinical and clinical development product candidates include ALVR106 for respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus; ALVR109 to treat SARS-CoV-2 and COVID-19; ALVR107 for treating hepatitis B; and ALVR108 to treat human herpesvirus-8, including Kaposi’s sarcoma, primary effusion lymphoma, and multicentric Castleman’s diseases. The company was formerly known as ViraCyte, Inc. and changed its name to Allovir, Inc. in May 2019. Allovir, Inc. was founded in 2013 and is based in Cambridge, Massachusetts.

Notch Therapeutics

Series A in 2021
Notch Therapeutics Inc., an immune cell therapy company, develops gene-edited allogenic T cell therapies for the treatment of cancer. The company offers induced pluripotent stem cell (iPSC) AlloCAR therapy products for non-Hodgkin lymphoma, leukemia, and multiple myeloma. Notch Therapeutics Inc. was incorporated in 2018 and is based in Toronto, Canada.

Neurogene

Private Placement in 2019
Neurogene is a developer of generic medicines intended to improve patient outcomes for neurological disorders.The company's offerings include advancing genetic medicines for many neurological disorders where no effective treatment options exist, providing patients and families affected by rare neurological disorders with gene therapy that improves quality of life.

Scholar Rock

Series A in 2014
Scholar Rock, Inc., a biotechnology company, discovers and develops a class of biologic therapies that selectively target dysregulated growth factors in the disease microenvironment by modulating supracellular activation. The company’s drug discovery programs target growth factors which are present in the microenvironments of significant diseases, including fibrosis, musculoskeletal diseases, immuno-oncology, and autoimmune diseases. Its lead products include SRK-015, a selective and local inhibitor of latent myostatin activation for the treatment of primary myopathies. The company was incorporated in 2012 and is based in Cambridge, Massachusetts.

Neogene Therapeutics

Series A in 2020
Neogene Therapeutics, Inc. develops T cell therapies to treat cancer. The company was incorporated in 2018 and is headquartered in New York, New York.

Dewpoint Therapeutics

Series B in 2020
Dewpoint Therapeutics, Inc., a biotech company, engages in research of biomolecular condensates and cellular function to develop treatments for diseases like cancer, neurodegenerative disorders, immunology, cardiovascular, women’s health, and virology. The company was incorporated in 2018 and is based in Boston, Massachusetts.

Accent Therapeutics

Series A in 2018
Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.

ElevateBio

Series B in 2020
ElevateBio is a operator of a portfolio of cell and gene therapy companies intended to develop, manufacture and commercialize life-transforming medicines. The company creates a portfolio through partnerships with the world's most innovative scientists and inventors as well as it created a centralized facility that integrates all the elements that translate cell and gene therapy R&D into commercially viable therapies quickly and efficiently, enabling patients to get medical products to treat severe diseases.

Neurogene

Series A in 2019
Neurogene is a developer of generic medicines intended to improve patient outcomes for neurological disorders.The company's offerings include advancing genetic medicines for many neurological disorders where no effective treatment options exist, providing patients and families affected by rare neurological disorders with gene therapy that improves quality of life.

AlloVir

Series B in 2019
AlloVir focuses on the development of cell therapies and focuses on restoring natural immunity against the virus-associated diseases. It provides an ideal environment for ViraCyte to develop and advance its product pipeline from discovery through late-stage clinical trials. The company was founded in 2013 and headquartered in Houston, Texas.

Ashvattha Therapeutics

Series A in 2019
Ashvattha Therapeutics creates products that selectively target and treat human diseases. Ashvattha enables precision targeting for therapeutics eliminating off-target toxicity, systemic side effects, and immunogenicity seen in other platforms. Ashvattha has a pipeline of nine therapeutics in neurology, oncology, and ophthalmology.

Clementia Pharmaceuticals

Venture Round in 2015
Clementia is a clinical-stage company innovating treatments for people with ultra-rare bone disorders. The company develops disease-modifying treatments for patients suffering from debilitating bone and other diseases with a high unmet medical need. The company's lead product candidate, palovarotene, is an oral small molecule that has shown potent activity in preventing abnormal new bone formation as well as fibrosis in a variety of tissues. Clementia Pharmaceuticals was founded in 2012 and is headquartered in Montréal, Quebec.

Regulus Therapeutics

Private Placement in 2019
Regulus Therapeutics Is a biopharmaceutical company leading the discovery of a new class of high-impact medicines based on microRNAs. The discovery of MicroRNA in humans is one of the most exciting scientific breakthroughs in the last decade. MicroRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. It was founded in 2007 and headquartered in Carlsbad, California.

Dewpoint Therapeutics

Private Placement in 2020
Dewpoint Therapeutics, Inc., a biotech company, engages in research of biomolecular condensates and cellular function to develop treatments for diseases like cancer, neurodegenerative disorders, immunology, cardiovascular, women’s health, and virology. The company was incorporated in 2018 and is based in Boston, Massachusetts.

Kezar Life Sciences

Series A in 2015
Kezar Life Sciences company focused on the discovery and development of drugs targeting protein homeostasis for autoimmune disorders. In parallel with this work, Christopher and Kezar co-founder Jack Taunton, Ph.D. collaborated on several research projects involving protein secretion. This collaboration inspired a belief in the strong synergies between protein degradation and protein secretion, two fertile areas for drug discovery with platform potential.

aTyr Pharma

Post in 2017
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.

Magenta Therapeutics

Series C in 2018
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.

Pieris Pharmaceuticals

Private Placement in 2019
Pieris Pharmaceuticals, Inc. is a, clinical-stage biotechnology company applying its proprietary Anticalin® technology to create differentiated drugs that can help patients suffering from cancer, severe asthma, anemia and other medical conditions with a high unmet medical need. Anticalins are recombinantly engineered versions of lipocalins, human proteins that naturally bind, store and transport a wide spectrum of molecules. Our libraries of more than 100 billion different Anticalins can virtually bind to any target of interest.

Ardelyx

Post in 2016
Ardelyx, Inc., a biopharmaceutical company, develops and sells medicines for the treatment of cardiorenal diseases in the United States and internationally. The company’s lead product candidate is tenapanor, which has completed Phase 3 clinical trial for the treatment of patients with irritable bowel syndrome with constipation, as well as in Phase 3 clinical trial for the treatment of hyperphosphatemia in end-stage renal disease patients on dialysis. It also develops RDX013, a small molecule potassium secretagogue program for the treatment of patients with hyperkalemia. The company was formerly known as Nteryx, Inc. and changed its name to Ardelyx, Inc. in June 2008. Ardelyx, Inc. was founded in 2007 and is headquartered in Fremont, California.

Prevail Therapeutics

Series B in 2019
Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company’s lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.

Gritstone bio

Private Placement in 2020
Gritstone Oncology is a cancer immunotherapy company developing next-generation personalized cancer therapeutics. Gritstone brings together distinguished scientific founders, an experienced and diverse management team, a seasoned and successful board of directors, and very deep financial backing to tackle fundamental challenges at the intersection of cancer biology, immunology, and immunotherapy design. The company’s initial goal is to identify and deploy therapeutic neo-antigens from individual patients’ tumors to develop novel treatments for lung cancer.

Kura Oncology

Venture Round in 2015
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.

Neurogene

Private Placement in 2020
Neurogene is a developer of generic medicines intended to improve patient outcomes for neurological disorders.The company's offerings include advancing genetic medicines for many neurological disorders where no effective treatment options exist, providing patients and families affected by rare neurological disorders with gene therapy that improves quality of life.

Intellia Therapeutics

Series B in 2015
Intellia Therapeutics was formed in 2014 to lead the industry in one of the most promising new areas of therapeutic development: gene editing and repair using CRISPR-Cas9 technology. Intellia holds exclusive access to one of the most comprehensive intellectual property platforms available for the therapeutic use of CRISPR-Cas9. The company is advancing a broad pipeline toward clinical development, including ex vivo and in vivo approaches. Intellia closed a Series A round in 2014 led by Atlas Venture and Novartis.

UNITY Biotechnology

Private Placement in 2018
Unity Biotechnology designs therapeutics that prevent, halt, and reverse various diseases of aging. The company focuses on clearing senescent cells; and designs senolytic medicines. Its medicines target vulnerabilities unique to senescent cells to clear those cells from the human body while leaving normal cells unaffected.

Arcus Biosciences

Series C in 2017
Arcus Biosciences is biotech company founded on a vision of creating new cancer therapeutics through the utilization of emerging insights in immunology. Arcus was formed in 2015 by a group of seasoned researchers from the biotechnology and pharmaceutical industries and is located in the San Francisco bay area, in the heart of the world’s largest biotechnology research hub.

Morphic Therapeutic

Private Placement in 2018
Morphic Therapeutic is a biotechnology company developing a new generation of oral integrin therapies. Drawing on discoveries made in the lab of Scientific Founder Tim Springer, Morphic was created in 2015 with co-founders Polaris Partners, T.A. Springer and Schrödinger, Inc., along with ShangPharma Investment Group.

Zymeworks

Post in 2023
Zymeworks Inc. is a privately held biotherapeutics company based in Vancouver, British Columbia. Zymeworks is committed to developing best-in-class protein therapeutics for the treatment of cancer, autoimmune and inflammatory diseases. Zymeworks' approach combines proprietary molecular modeling and simulation software with high-performance computing to create an environment for in silico experimentation and predictive protein optimization. Using this approach Zymeworks has developed multiple platform technologies to advance the field of biologics drug discovery and optimization.

Terremoto Biosciences

Series B in 2023
Terremoto Biosciences is a biotechnology company leveraging the power of lysine-based covalency to develop optimized medicines.

Aligos Therapeutics

Post in 2023
Aligos Therapeutics, Inc. is a clinical stage biopharmaceutical company that was founded in 2018 with the mission to become a world leader in the treatment of viral infections and liver diseases. Aligos is focused on the development of targeted antiviral therapies for chronic hepatitis B (CHB) and coronaviruses as well as leveraging its expertise in liver diseases to create targeted therapeutics for nonalcoholic steatohepatitis (NASH). Aligos’ strategy is to harness the deep expertise and decades of drug development experience its workforce has in liver disease, particularly viral hepatitis, to rapidly advance its pipeline of potentially best-in-class molecules.

BioAtla

Post in 2022
BioAtla, Inc., a biopharmaceutical company, develops specific and selective antibody-based therapeutics for the treatment of various cancers. Its lead product candidate is BA3011, a conditionally active biologic (CAB) antibody-drug conjugate (ADC) for soft tissue and bone sarcoma tumors, non-small cell lung cancer (NSCLC), and other tumor types. It also develops BA3021, a CAB ADC for NSCLC and Melanoma; and BA3071, which is a CAB anti-cytotoxic T-lymphocyte-associated antigen 4 antibody for renal cell carcinoma, NSCLC, small cell lung cancer, hepatocellular carcinoma, melanoma, bladder cancer, gastric cancer, and cervical cancer. The company was founded in 2007 and is based in San Diego, California.
SpringWorks Therapeutics is a biotechnology company that specializes in identifying, developing, and commercializing therapies for underserved patient populations suffering from severe rare diseases and cancer. The firm's pipeline product includes Mirdametinib, Nirogacestat, and others. The company was founded in 2017 and headquartered in Connecticut, United States.

ReCode Therapeutics

Series B in 2023
ReCode Therapeutics is a development-stage biopharmaceutical company focused on the correction of the nonsense mutations that account for 1/3 of all inherited diseases, including approximately 10% of cystic fibrosis. They have combined selected tRNAs with their unique and diverse proprietary delivery platforms to create and develop first-in-class suppressor tRNA-nanoparticle therapeutics for correcting nonsense mutations. ReCode’s diverse pipeline includes lead programs for primary ciliary dyskinesia (PCD) and nonsense mutations in cystic fibrosis (CF). The company’s proprietary non-viral lipid nanoparticle platform enables the delivery of a variety of payloads, and precise organ targeting of nucleic acid and protein payloads via both systemic and local delivery. ReCode is leveraging its nanoparticle and nucleic acid technologies across multiple modalities, including mRNA-mediated protein replacement therapies, first-in-class tRNA NanoCorrectors for diseases caused by nonsense mutations, and gene editing. The company was founded in 2015 and is based in Dallas, Texas.

Abeona Therapeutics

Post in 2022
Abeona Therapeutics is developing gene therapy-based potential cures for the deadly childhood diseases Sanfilippo (SF) Syndrome Type A and Type B. Its multi-platform expertise across the manufacture, delivery, development, and discovery of the novel gene and cell therapies has it uniquely positioned for success. Underpinning the Company’s robust pipeline is its fully-operational manufacturing facility producing therapies and vectors for preclinical and clinical studies. Abeona is also developing the AIM Vector Platform: 100+ next-generation AAV capsids for delivering gene therapies targeting a wide range of organs and multiple routes of delivery.

Gritstone bio

Post in 2020
Gritstone Oncology is a cancer immunotherapy company developing next-generation personalized cancer therapeutics. Gritstone brings together distinguished scientific founders, an experienced and diverse management team, a seasoned and successful board of directors, and very deep financial backing to tackle fundamental challenges at the intersection of cancer biology, immunology, and immunotherapy design. The company’s initial goal is to identify and deploy therapeutic neo-antigens from individual patients’ tumors to develop novel treatments for lung cancer.

Silverback Therapeutics

Series C in 2020
Silverback Therapeutics is a biopharmaceutical company that develops ImmunoTAC therapies targeting previously inaccessible disease pathways. They developing a new generation of systemically delivered, locally active therapies that potently modulate fundamental disease pathways once deemed inaccessible. Silverback Therapeutics is building a platform technology capable of unlocking potent immuno-modulatory pathways using an established antibody-guided approach for targeting disease sites. The company's proprietary ImmunoTAC technology and integrated R&D approach enables the design of product candidates that can be administered systemically, but that act only at the sites of disease.

Aktis Oncology

Series A in 2021
Aktis Oncology is a biotechnology company pioneering the discovery and development of a new class of targeted radiopharmaceuticals to treat a broad range of solid tumor cancers. Founded and incubated by MPM Capital, the company has developed proprietary platforms to generate tumor-targeting agents with ideal properties for alpha radiotherapy. Designed for high tumor penetration and long residence time, Aktis Oncology's molecules will quickly clear other areas of the body, thereby maximizing tumor elimination while minimizing the side effects of treatment. This approach would also enable clinicians to visualize and verify target engagement prior to exposure to therapeutic radioisotopes.

Dewpoint Therapeutics

Series C in 2022
Dewpoint Therapeutics, Inc., a biotech company, engages in research of biomolecular condensates and cellular function to develop treatments for diseases like cancer, neurodegenerative disorders, immunology, cardiovascular, women’s health, and virology. The company was incorporated in 2018 and is based in Boston, Massachusetts.
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.

Chinook Therapeutics

Private Placement in 2020
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.

2seventy bio

Post in 2022
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.

Nurix Therapeutics

Private Placement in 2020
Nurix develops small molecule inhibitors for the treatment of proliferative and degenerative diseases. The company’s product engine leverages a deep mechanistic understanding of the UPS to deliver effective small molecule drug candidates with the potential to make a dramatic difference for patients. Nurix drugs control ubiquitin E3 ligases, the key enzymes responsible for protein breakdown in human cells, as a unique therapeutic approach to treat a broad range of diseases. The company discovers drugs that harness the body's natural process to control protein levels. Their focus is on developing drugs to treat cancer including the novel, small molecule immuno-oncology agents. Nurix was founded in 2009 by internationally recognized experts in the ubiquitin-proteasome field and is funded by leading life science investors, Third Rock Ventures, and The Column Group. The company is headquartered in San Francisco, California.

Magenta Therapeutics

Private Placement in 2018
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.

Nabriva Therapeutics

Series B in 2015
Nabriva Therapeutics AG operates as a biopharmaceutical company that engages in the discovery and development of antibiotics for use in community and hospital infections. The company focuses on antibiotic classes, such as pleuromutilins designed for oral and topical application; and injectable broad-spectrum anti-MRSA cephalosporins. Its products include Oral Pleuromutilins BC-3205 and BC-3781 for multi-drug resistant (MDR) pathogens, including methicillin resistant Staphylococcus aureus (MRSA), MDR Streptococcus pneumonia, and vancomycin resistant Enterococcus faecium; Topical Pleuromutilin BC-7013 for the treatment of various bacterial associated, or derived dermatological diseases; and Cephalosporins for the treatment of hospital infections. The company was formerly known as Antibiotic Research Institute and changed its name to Nabriva Therapeutics AG in February, 2006. Nabriva Therapeutics AG was founded in 2001 is based in Vienna, Austria.

Accent Therapeutics

Series B in 2020
Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.

UNITY Biotechnology

Series C in 2018
Unity Biotechnology designs therapeutics that prevent, halt, and reverse various diseases of aging. The company focuses on clearing senescent cells; and designs senolytic medicines. Its medicines target vulnerabilities unique to senescent cells to clear those cells from the human body while leaving normal cells unaffected.
Dicerna Pharmaceuticals is a biotechnology company involved in the discovery and development of innovative treatments for rare inherited diseases affecting the liver, along with cancers, and other therapeutic areas to address liver problems. It is using ribonucleic acid interference (RNA) technology platform to develop its products. The product candidates are DCR-PHXC (for primary hyperoxaluria), DCR-undisclosed (for orphan genetic disease), DCR-PCSK9 (for cardiovascular disease), and DCR-HBV (for hepatitis B virus) among others. The company was founded in 2007 and is headquartered in Lexington, Massachusetts.

ElevateBio

Private Placement in 2020
ElevateBio is a operator of a portfolio of cell and gene therapy companies intended to develop, manufacture and commercialize life-transforming medicines. The company creates a portfolio through partnerships with the world's most innovative scientists and inventors as well as it created a centralized facility that integrates all the elements that translate cell and gene therapy R&D into commercially viable therapies quickly and efficiently, enabling patients to get medical products to treat severe diseases.
Novus Therapeutics is a pharmaceutical company focusing on the acquisition, development, and commercialization of ear, nose, and throat (ENT) products. The company has two technologies, each of which has the potential to be developed for multiple ENT indications. It was founded in 2004 and headquartered in Irvine, California.

Tectonic Therapeutic

Series A in 2021
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.

Cellares

Series A in 2020
Cellares Corporation develops cell therapy for the treatment of cancer. The company develops solution to overcome limitation of manufacturing to make cell therapies for patients. Cellares Corporation was founded in 2019 and is based in South San Francisco, California.

Prevail Therapeutics

Series A in 2018
Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company’s lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.

Scholar Rock

Private Placement in 2017
Scholar Rock, Inc., a biotechnology company, discovers and develops a class of biologic therapies that selectively target dysregulated growth factors in the disease microenvironment by modulating supracellular activation. The company’s drug discovery programs target growth factors which are present in the microenvironments of significant diseases, including fibrosis, musculoskeletal diseases, immuno-oncology, and autoimmune diseases. Its lead products include SRK-015, a selective and local inhibitor of latent myostatin activation for the treatment of primary myopathies. The company was incorporated in 2012 and is based in Cambridge, Massachusetts.

Ajax Therapeutics

Venture Round in 2021
Ajax Therapeutics is an NYC-based biotechnology company applying computational chemistry and structure-based technologies to develop novel small molecules for hematologic malignancies

Flex Pharma

Venture Round in 2014
Flex Pharma, Inc. is a biotechnology company that is developing innovative and proprietary treatments for nocturnal leg cramps, cramps and spasms associated.

Affinivax

Series C in 2021
Affinivax, Inc., a biotechnology company, focuses on developing multiple antigen presenting system (MAPS) technology based vaccines that provide protection against various infectious diseases afflicting children and adults around the globe. Its MAPS enables the binding of protective polysaccharides and proteins in a single vaccine and induces a protective immune response. The company develops its vaccine programs to target various pathogens, such as streptococcus pneumoniae (pneumococcus), bacterial nosocomial infections, salmonella typhi, and staphylococcus aureus. Affinivax, Inc. was incorporated in 2014 and is based in Lexington, Massachusetts.

Accent Therapeutics

Private Placement in 2020
Accent Therapeutics is a biopharmaceutical company developing oncology-focused, small molecule therapies in the emerging field of epitranscriptomics. This field of biology encompasses post-transcriptional chemical modifications of RNA that provide cells with a unique mechanism for regulating proteins critical for cellular growth and differentiation. By targeting cancer-linked RNA-modifying proteins (RMPs) with precision therapies, they aim to translate extraordinary science into life-changing therapies for patients.

Metacrine

Series A in 2015
Metacrine is a clinical-stage biopharmaceutical company focused on building an innovative pipeline of best-in-class drugs to treat liver and gastrointestinal (GI) diseases. The most advanced program is focused on the farnesoid X receptor (FXR) an important drug target in multiple liver and GI diseases. Beyond the FXR program, a pipeline of novel drug candidates against other drug targets is being explored by taking advantage of internal drug discovery and development capabilities.

Cellares

Private Placement in 2019
Cellares Corporation develops cell therapy for the treatment of cancer. The company develops solution to overcome limitation of manufacturing to make cell therapies for patients. Cellares Corporation was founded in 2019 and is based in South San Francisco, California.

Eledon Pharmaceuticals

Private Placement in 2020
Novus Therapeutics is a pharmaceutical company focusing on the acquisition, development, and commercialization of ear, nose, and throat (ENT) products. The company has two technologies, each of which has the potential to be developed for multiple ENT indications. It was founded in 2004 and headquartered in Irvine, California.

Atreca

Series C in 2018
Atreca is a biopharmaceutical that develops a technology to identify the set of antibodies produced during an immune response, without prior knowledge of an antigen. Its product candidate, ATRC-101, is a monoclonal antibody in preclinical development with a novel mechanism of action and target-derived from an antibody identified using its discovery platform.

ElevateBio

Series A in 2019
ElevateBio is a operator of a portfolio of cell and gene therapy companies intended to develop, manufacture and commercialize life-transforming medicines. The company creates a portfolio through partnerships with the world's most innovative scientists and inventors as well as it created a centralized facility that integrates all the elements that translate cell and gene therapy R&D into commercially viable therapies quickly and efficiently, enabling patients to get medical products to treat severe diseases.

Alpine Immune Sciences

Private Placement in 2020
Alpine Immune Sciences is an immunotherapy startup focused on developing recombinant, protein-based therapeutic solutions. It was founded on January 23, 2015, and is based in Seattle, Washington, United States.

Solid Biosciences

Private Placement in 2019
Solid Biosciences Inc., a life science company, engages in developing therapies for duchenne muscular dystrophy (DMD) in the United States. The company’s lead product candidate is SGT-001, a gene transfer candidate, which is in a Phase I/II clinical trial to derive functional dystrophin protein expression in patients’ muscles. Its portfolio also comprises Anti-LTBP4, a complementary disease modifying program that identifies and develops a monoclonal antibody intended to reduce fibrosis and inflammation by targeting and stabilizing the LTBP4 protein. In addition, the company engages in developing biomarkers and sensors; and Solid Suit program that includes the development of wearable assistive devices that focus on providing functional and therapeutic benefits. Solid Biosciences Inc. has strategic collaboration with Ultragenyx to develop and commercialize new gene therapies for Duchenne Muscular Dystrophy. Solid Biosciences Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.

Verastem

Private Placement in 2020
Verastem Oncology is a biopharmaceutical company committed to the development and commercialization of medicines to improve the lives of patients diagnosed with cancer. Its portfolio consists of small molecule kinase inhibitors that inhibit critical signaling pathways, which promote cancer cell survival and tumor growth.

Dewpoint Therapeutics

Series A in 2019
Dewpoint Therapeutics, Inc., a biotech company, engages in research of biomolecular condensates and cellular function to develop treatments for diseases like cancer, neurodegenerative disorders, immunology, cardiovascular, women’s health, and virology. The company was incorporated in 2018 and is based in Boston, Massachusetts.

Peloton Therapeutics

Private Placement in 2019
A peloton is a team of bicyclists who achieve great efficiency of motion through cooperative action. It is also the root of platoon, a nimble military unit with capabilities that exceed the sum of its component parts. Founded by Chairman of Biochemistry Steven L. McKnight of UT Southwestern Medical Center, Peloton Therapeutics represents a unique combination of innovative science, strong financial support from the Cancer Prevention Research Institute of Texas (CPRIT), and seasoned investors and management. Working in close collaboration with scientists at UT Southwestern, Peloton is advancing a promising discovery and development pipeline, comprised of several small molecule-based programs, each with a distinct target and mechanism of action. Peloton's vision is to become a major biotech company in Dallas with a passion for discovering and developing novel cancer drugs.

Prevail Therapeutics

Private Placement in 2018
Prevail Therapeutics Inc., a gene therapy company, focuses on developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company’s lead product candidate is PR001, which is in Phase I/II clinical trial for the treatment of Parkinson’s disease with GBA1 mutation and neuronopathic Gaucher disease. It is also developing PR006 for the treatment of frontotemporal dementia with GRN mutation; and PR004 for the treatment of synucleinopathies. Prevail Therapeutics Inc. was founded in 2017 and is based in New York, New York.

Atara Biotherapeutics

Series B in 2014
Atara Biotherapeutics, Inc. (@Atarabio) is an off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune, and viral diseases. Atara’s technology platform leverages research collaborations with leading academic institutions with the Company’s scientific, clinical, regulatory, and manufacturing expertise. Atara’s pipeline includes tab-cel® (tabelecleucel), which is in Phase 3 development for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disorder (EBV+ PTLD) as well as in earlier stage development for other EBV-associated hematologic malignancies and solid tumors, including nasopharyngeal carcinoma (NPC); T-cell immunotherapies targeting EBV antigens believed to be important for the potential treatment of multiple sclerosis; and next-generation chimeric antigen receptor T-cell (CAR T) immunotherapies. The company was founded in 2012 and is co-located in South San Francisco and Southern California. Our Southern California hub is anchored by the state-of-the-art Atara T-cell Operations and Manufacturing (ATOM) facility in Thousand Oaks, California. For additional information about the company, please visit atarabio.com.