EcoR1 Capital

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.

Syros Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead candidates, SY-1425, a selective retinoic acid receptor alpha agonist currently undergoing Phase II trials for specific patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor in Phase I trials for select advanced solid tumors. Syros employs a proprietary platform that integrates assay technologies, bioinformatics, and biological insights to map gene regulatory circuits and modulate gene expression, positioning itself as a leader in gene control research. Additionally, Syros has established collaborations, including a target discovery agreement with Incyte Corporation focused on myeloproliferative neoplasms, and a licensing agreement with TMRC Co. Ltd. for tamibarotene's development and commercialization. Founded in 2011, Syros Pharmaceuticals aims to address significant unmet medical needs in oncology and beyond.

Neogene Therapeutics, Inc. is a biotechnology company based in New York that focuses on the development of T cell therapies to treat cancer. Founded in 2018, the company specializes in creating personalized engineered T-cells that target neo-antigens, which are mutated proteins resulting from DNA alterations in cancer cells. Neogene's innovative platform allows for the isolation of T cell receptor (TCR) genes specific to these neo-antigens from tumor biopsies, which are routinely collected from patients. By utilizing advanced DNA sequencing and genetic screening technologies, Neogene can accurately identify and engineer TCRs that enhance the ability of T-cells to detect and eliminate cancer cells. This approach aims to improve treatment outcomes for cancer patients by providing therapies tailored to their unique tumor profiles.

Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.

Neurogene Inc. is a clinical-stage biotechnology company focused on developing genetic medicines for neurological diseases. Founded in 2018 and based in New York, the company employs adeno-associated virus (AAV) technology to create treatments for conditions such as Charcot-Marie Tooth disease (CMT4J), Aspartylglucosaminuria (AGU), and various lysosomal storage diseases. Neurogene aims to improve patient outcomes by providing effective gene therapies for rare neurological disorders that currently lack viable treatment options. Through its innovative approaches, the company seeks to enhance the quality of life for patients and their families affected by these debilitating conditions.

Avidity Biosciences is a biopharmaceutical company specializing in the development of oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disorder. The company's development pipeline also includes programs focused on treating various muscle diseases, such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Additionally, Avidity is exploring therapeutic applications for immune and other cell types, leveraging its proprietary AOC platform to reach previously undruggable tissues and effectively target the genetic underpinnings of these conditions. Founded in 2012, Avidity Biosciences is headquartered in La Jolla, California.

Prevail Therapeutics Inc. is a biotechnology company based in New York that specializes in developing gene therapies for neurodegenerative diseases. Founded in 2017 with support from The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, the company is focused on creating innovative adeno-associated virus (AAV)-based treatments. Its lead candidate, PR001, is currently undergoing Phase I/II clinical trials aimed at treating Parkinson's disease linked to GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is developing PR006 for frontotemporal dementia associated with GRN mutations and PR004 for various synucleinopathies. The company is dedicated to advancing therapies that can modify the course of these debilitating conditions.

Codiak is a clinical-stage biopharmaceutical company focused on pioneering the development of exosome-based therapeutics, a new class of medicines with the potential to transform the treatment of a wide spectrum of diseases with high unmet medical need. By leveraging the biology of exosomes as natural intercellular transfer mechanisms, Codiak has developed its proprietary engEx Platform to expand upon the innate properties of exosomes to design, engineer and manufacture novel exosome therapeutic candidates. Codiak has utilized its engEx Platform to generate a deep pipeline of engineered exosomes aimed at treating a broad range of disease areas, spanning oncology, neuro-oncology, neurology, neuromuscular disease and infectious disease.

Dicerna Pharmaceuticals is a biopharmaceutical company dedicated to the discovery and development of innovative treatments primarily for liver-related diseases, including rare inherited conditions, chronic liver diseases, and viral infections. Utilizing its proprietary GalXC RNA interference technology, Dicerna focuses on silencing disease-causing genes to address various health issues. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and belcesiran for genetic liver disease, among others. The company's extensive research efforts also encompass treatments for cardiovascular diseases and neurodegeneration. Dicerna has established strategic collaborations with prominent pharmaceutical companies to enhance its development capabilities. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna is at the forefront of advancing RNA-based therapeutics.

Intellia Therapeutics is a genome editing company based in Cambridge, Massachusetts, focused on developing curative gene-editing treatments using the CRISPR/Cas9 technology. The company aims to address severe and life-threatening genetic diseases through both in vivo and ex vivo therapeutic approaches. Its pipeline includes programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia is developing engineered cell therapies for oncological and autoimmune diseases. The company has established collaborations with various entities, including leading pharmaceutical firms and research institutions, to enhance its research and development efforts. Founded in 2014, Intellia Therapeutics holds a comprehensive intellectual property platform for the therapeutic application of CRISPR technology, positioning it at the forefront of gene editing innovation.