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.

Ajax Therapeutics is a New York City-based biotechnology company that applies computational chemistry and structure-based technologies to develop novel small molecules for treating hematologic malignancies.

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.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

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 specializes in developing precision medicines tailored to homogeneous rare patient populations. Its pipeline focuses on creating new drugs designed to enhance clinical success rates and ultimately deliver treatments to all patients in need, including those with challenging-to-cure illnesses.

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.

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 New York City-based biotechnology company that applies computational chemistry and structure-based technologies to develop novel small molecules for treating hematologic malignancies.

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.

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.

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 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.