EcoR1 Capital

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs that target G protein-coupled receptors (GPCRs). With a focus on therapeutic proteins and antibodies, Tectonic aims to address the complexities of GPCR-targeted drug discovery through its proprietary technology platform, GEODe. The company is committed to unlocking the therapeutic potential of challenging receptors, striving to create biologic medicines that can effectively modify disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic combines a strong scientific foundation with a history of entrepreneurial success to advance its mission in the biopharmaceutical landscape.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs that target G protein-coupled receptors (GPCRs). With a focus on therapeutic proteins and antibodies, Tectonic aims to address the complexities of GPCR-targeted drug discovery through its proprietary technology platform, GEODe. The company is committed to unlocking the therapeutic potential of challenging receptors, striving to create biologic medicines that can effectively modify disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic combines a strong scientific foundation with a history of entrepreneurial success to advance its mission in the biopharmaceutical landscape.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

Kura Oncology is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing targeted therapies for cancer treatment. The company’s primary product candidate, Tipifarnib, is an orally available farnesyl transferase inhibitor currently undergoing Phase II clinical trials for solid tumors, peripheral T-cell lymphomas, and various hematologic malignancies. In addition to Tipifarnib, Kura is advancing other small molecule candidates, including KO-947, which targets dysregulated extracellular signal-related kinases, and KO-539, aimed at inhibiting the menin-mixed lineage leukemia protein interaction. Kura Oncology emphasizes precision medicine, leveraging insights from cancer genomics to create targeted treatments for patients with specific genetic profiles, addressing significant unmet medical needs across various cancer types, including lung, colorectal, thyroid, pancreatic cancers, and blood cancers such as lymphoma and leukemia. The company was founded in 2014 and operates primarily within the United States.

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.

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.

Actio Biosciences focuses on the development of precision medicines tailored for homogeneous rare patient populations. The company is engaged in creating a pipeline of innovative drugs aimed at treating rare diseases, with the potential to extend their application to more common illnesses. By utilizing biotechnology to modulate the activity of proteins linked to rare diseases, Actio Biosciences aims to enhance the likelihood of clinical success and deliver effective treatments for conditions that are often difficult to cure. Their commitment to addressing both rare and common diseases positions them to make a significant impact in the field of medicine.

Paratus Sciences is a biotechnology company focused on enhancing human health and health security through the study of bat biology. By exploring the unique aspects of the bat genome, the company aims to identify and develop therapeutics for a variety of diseases. Paratus Sciences seeks to unlock the secrets of bat biology to facilitate innovative treatments, ultimately helping patients combat the health challenges they encounter.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing gene and cell therapies for life-threatening rare genetic diseases. Its lead programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa; ABO-102, an adeno-associated virus-based gene therapy for Sanfilippo syndrome type A; and ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B. The company also develops therapies for Batten disease, cystic fibrosis, and genetic eye disorders. Additionally, Abeona Therapeutics is advancing the AIM Vector Platform, which includes over 100 next-generation AAV capsids for delivering gene therapies to various organs. The company operates a manufacturing facility producing therapies and vectors for preclinical and clinical studies.

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.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs that target G protein-coupled receptors (GPCRs). With a focus on therapeutic proteins and antibodies, Tectonic aims to address the complexities of GPCR-targeted drug discovery through its proprietary technology platform, GEODe. The company is committed to unlocking the therapeutic potential of challenging receptors, striving to create biologic medicines that can effectively modify disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic combines a strong scientific foundation with a history of entrepreneurial success to advance its mission in the biopharmaceutical landscape.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

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.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

Neogene Therapeutics, Inc. is a biotechnology company based in New York, founded in 2018, that focuses on developing T cell therapies for cancer treatment. The company specializes in creating personalized engineered T cells that target neo-antigens, which are mutated proteins present in cancer cells due to DNA mutations. By leveraging advanced technologies, Neogene isolates T cell receptor (TCR) genes specific to these neo-antigens from tumor biopsies routinely collected from patients. This process utilizes high-sensitivity DNA sequencing and genetic screening to identify TCRs that can effectively recognize and attack cancer cells. Neogene's innovative approach aims to enhance the precision and efficacy of cancer therapies, offering improved treatment options for patients facing a broad spectrum of cancers.

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.

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.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

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.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Codiak BioSciences is a clinical-stage biopharmaceutical company dedicated to developing exosome-based therapeutics, a novel class of medicines with the potential to address various diseases with significant unmet medical needs. The company employs its proprietary engEx Platform, which allows for the engineering and manufacturing of exosomes, leveraging their natural role as intercellular messengers. Codiak's pipeline includes product candidates such as exoSTING and exoIL-12, targeting diverse therapeutic areas including oncology, neuro-oncology, neurology, neuromuscular diseases, and infectious diseases. Additionally, the company collaborates with the Ragon Institute to explore the potential of its exoVACC platform for vaccines against SARS-CoV-2 and HIV. Founded in 2015 and headquartered in Cambridge, Massachusetts, Codiak aims to transform therapeutic approaches by harnessing the unique properties of exosomes.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

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.

Kura Oncology is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing targeted therapies for cancer treatment. The company’s primary product candidate, Tipifarnib, is an orally available farnesyl transferase inhibitor currently undergoing Phase II clinical trials for solid tumors, peripheral T-cell lymphomas, and various hematologic malignancies. In addition to Tipifarnib, Kura is advancing other small molecule candidates, including KO-947, which targets dysregulated extracellular signal-related kinases, and KO-539, aimed at inhibiting the menin-mixed lineage leukemia protein interaction. Kura Oncology emphasizes precision medicine, leveraging insights from cancer genomics to create targeted treatments for patients with specific genetic profiles, addressing significant unmet medical needs across various cancer types, including lung, colorectal, thyroid, pancreatic cancers, and blood cancers such as lymphoma and leukemia. The company was founded in 2014 and operates primarily within the United States.