EcoR1 Capital

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Atara Biotherapeutics, Inc. is a biotechnology company focused on developing off-the-shelf T-cell immunotherapies for patients with cancer, autoimmune diseases, and viral infections. The company is advancing tabelecleucel, a T-cell immunotherapy currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, as well as other related hematologic and solid tumors, including nasopharyngeal carcinoma. Atara is also working on next-generation CAR T immunotherapies targeting various indications, including ATA2271 and ATA3271 for mesothelin, and ATA2431 and ATA3219 for B-cell lymphomas. Additionally, its pipeline includes ATA188 for multiple sclerosis and several other candidates for conditions such as acute myeloid leukemia and cytomegalovirus. Founded in 2012, Atara Biotherapeutics is headquartered in South San Francisco, California, and collaborates with leading academic institutions to enhance its research and development efforts.

CRISPR Therapeutics specializes in developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. This technology enables precise, directed changes to genomic DNA for treating serious human diseases. The company focuses on various disease areas including hemoglobinopathies, oncology, regenerative medicine, and rare diseases.

Accent Therapeutics, Inc. is a biopharmaceutical company focused on developing small molecule therapies for oncology, utilizing the emerging field of epitranscriptomics. Founded in 2017 and headquartered in Lexington, Massachusetts, the company explores the post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting cancer-associated RNA-modifying proteins, Accent Therapeutics aims to innovate and deliver transformative therapies that can significantly improve patient outcomes in cancer treatment.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Paratus Sciences focuses on advancing human health and health security by studying the biology of bats. It integrates cell biology, genomics, informatics, and large-scale data inputs to compare disease resistance patterns in bats with those in humans, aiming to develop therapeutics that mimic bats' adaptations.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy-based treatments for severe and life-threatening rare genetic diseases. Its pipeline includes therapies for Sanfilippo syndrome types A and B, epidermolysis bullosa, Batten disease, cystic fibrosis, and genetic eye disorders.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

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.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Notch Therapeutics is a Toronto-based company developing immune cell therapies using induced pluripotent stem cell technology to create gene-edited allogeneic T cells. Its platform enables precise control of Notch signaling during T-cell development, enabling scalable manufacture of a uniform supply of therapeutic T cells for cancer indications such as non-Hodgkin lymphoma, leukemia, and multiple myeloma. The approach aims to overcome bottlenecks in cell therapy development by integrating advanced product design with commercially compatible processes, supporting a next-generation pipeline of cell therapies.

Gritstone bio is a biotechnology company focused on developing immunotherapies for cancer and infectious diseases. The company initially concentrated on tumor-specific neoantigens but has expanded its programs to include viral antigens found on the surface of infected cells. Gritstone bio's approach leverages the body's natural immune system recognition of targets on abnormal cells, which is applicable to both anti-tumor and anti-viral immunity. Their pipeline includes several product candidates for treating solid tumors, such as GRANITE, SLATE, and CORAL. The company operates in a field that has seen significant advancements with the development and commercialization of immunotherapy drugs like checkpoint inhibitors, transforming cancer treatment.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Neogene Therapeutics, Inc., founded in 2018 and based in New York, develops innovative T cell therapies aimed at treating cancer. The company specializes in creating personalized engineered T cells that target mutated proteins, known as neo-antigens, found in cancer cells as a result of DNA mutations. By leveraging advanced technologies, Neogene isolates neo-antigen specific T cell receptor genes from tumor biopsies, which are routinely collected during cancer treatment. These tumor-infiltrating lymphocytes often express T cell receptors that can recognize and attack cancer cells displaying neo-antigens. Utilizing state-of-the-art DNA sequencing, synthesis, and genetic screening, Neogene's proprietary platform enables the identification of these specific T cell receptors with high sensitivity and at scale, ultimately enhancing the efficacy of cancer treatments.

Accent Therapeutics, Inc. is a biopharmaceutical company focused on developing small molecule therapies for oncology, utilizing the emerging field of epitranscriptomics. Founded in 2017 and headquartered in Lexington, Massachusetts, the company explores the post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting cancer-associated RNA-modifying proteins, Accent Therapeutics aims to innovate and deliver transformative therapies that can significantly improve patient outcomes in cancer treatment.

Nurix Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing oral small-molecule therapies that modulate cellular protein levels to treat cancer and immune disorders. It operates DELigase, an integrated discovery platform that targets E3 ligases to alter protein abundance within cells. The company's product pipeline includes targeted protein degraders such as NX-2127, an oral Bruton's tyrosine kinase degrader for relapsed or refractory B-cell malignancies, and NX-1607, an oral Casitas B-lineage lymphoma proto-oncogene-B inhibitor for immuno-oncology indications; these are in preclinical stages. Nurix also explores inhibitors of CBL-B, an E3 ligase that regulates T cell activation. The company collaborates with major industry partners, including Gilead Sciences and Sanofi, and was founded in 2009 and is headquartered in San Francisco.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients' muscles. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Accent Therapeutics, Inc. is a biopharmaceutical company focused on developing small molecule therapies for oncology, utilizing the emerging field of epitranscriptomics. Founded in 2017 and headquartered in Lexington, Massachusetts, the company explores the post-transcriptional chemical modifications of RNA, which play a crucial role in regulating proteins essential for cellular growth and differentiation. By precisely targeting cancer-associated RNA-modifying proteins, Accent Therapeutics aims to innovate and deliver transformative therapies that can significantly improve patient outcomes in cancer treatment.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Atara Biotherapeutics, Inc. is a biotechnology company focused on developing off-the-shelf T-cell immunotherapies for patients with cancer, autoimmune diseases, and viral infections. The company is advancing tabelecleucel, a T-cell immunotherapy currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, as well as other related hematologic and solid tumors, including nasopharyngeal carcinoma. Atara is also working on next-generation CAR T immunotherapies targeting various indications, including ATA2271 and ATA3271 for mesothelin, and ATA2431 and ATA3219 for B-cell lymphomas. Additionally, its pipeline includes ATA188 for multiple sclerosis and several other candidates for conditions such as acute myeloid leukemia and cytomegalovirus. Founded in 2012, Atara Biotherapeutics is headquartered in South San Francisco, California, and collaborates with leading academic institutions to enhance its research and development efforts.

Atara Biotherapeutics, Inc. is a biotechnology company focused on developing off-the-shelf T-cell immunotherapies for patients with cancer, autoimmune diseases, and viral infections. The company is advancing tabelecleucel, a T-cell immunotherapy currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, as well as other related hematologic and solid tumors, including nasopharyngeal carcinoma. Atara is also working on next-generation CAR T immunotherapies targeting various indications, including ATA2271 and ATA3271 for mesothelin, and ATA2431 and ATA3219 for B-cell lymphomas. Additionally, its pipeline includes ATA188 for multiple sclerosis and several other candidates for conditions such as acute myeloid leukemia and cytomegalovirus. Founded in 2012, Atara Biotherapeutics is headquartered in South San Francisco, California, and collaborates with leading academic institutions to enhance its research and development efforts.

Atara Biotherapeutics, Inc. is a biotechnology company focused on developing off-the-shelf T-cell immunotherapies for patients with cancer, autoimmune diseases, and viral infections. The company is advancing tabelecleucel, a T-cell immunotherapy currently in Phase III clinical trials for treating Epstein-Barr virus-associated post-transplant lymphoproliferative disorder, as well as other related hematologic and solid tumors, including nasopharyngeal carcinoma. Atara is also working on next-generation CAR T immunotherapies targeting various indications, including ATA2271 and ATA3271 for mesothelin, and ATA2431 and ATA3219 for B-cell lymphomas. Additionally, its pipeline includes ATA188 for multiple sclerosis and several other candidates for conditions such as acute myeloid leukemia and cytomegalovirus. Founded in 2012, Atara Biotherapeutics is headquartered in South San Francisco, California, and collaborates with leading academic institutions to enhance its research and development efforts.