Sarepta Therapeutics

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, specializing in the development of adeno-associated viral (AAV) vector technologies and therapeutics aimed at treating rare diseases. Founded in 2015, the company employs a unique STRucture Inspired DEsign approach that merges structural knowledge with accelerated evolution to create innovative AAV capsids. This technology is designed to evade neutralizing antibodies, thereby enhancing gene transfer efficiency. StrideBio's platform supports various gene therapy modalities, including gene addition, gene silencing, and gene editing, with applications across a range of rare genetic disorders.

AavantiBio, Inc. is dedicated to developing gene transfer therapy and gene editing technologies aimed at improving the lives of individuals afflicted by rare and fatal diseases. The company primarily focuses on Friedreich’s Ataxia, a rare inherited genetic disorder that leads to significant cardiac and central nervous system dysfunction. Founded in 2019 and based in Gainesville, Florida, AavantiBio is committed to advancing innovative gene therapies to address unmet medical needs in the treatment of rare genetic diseases and autoimmune conditions. Through its research and development efforts, the company seeks to transform patient care and enhance treatment options for those affected by these serious health challenges.

Myonexus Therapeutics is a clinical stage, rare disease gene therapy company developing first ever treatments for Limb-girdle muscular dystrophy (LGMD) Types 2D, 2B, 2E, 2L, and 2C based on research at Nationwide Children’s Hospital, a global leader in muscular dystrophy gene therapy discovery and translational research.

Lacerta Therapeutics, Inc., a clinical-stage gene therapy company, develops treatments for central nervous system and lysosomal storage diseases using a constellation of adeno-associated virus (AAV) vector technologies. The company was incorporated in 2017 and is based in Alachua, Florida.

Myonexus Therapeutics is a clinical stage, rare disease gene therapy company developing first ever treatments for Limb-girdle muscular dystrophy (LGMD) Types 2D, 2B, 2E, 2L, and 2C based on research at Nationwide Children’s Hospital, a global leader in muscular dystrophy gene therapy discovery and translational research.

Eisai Inc., a subsidiary of Tokyo-based Eisai Co., Ltd, is a research-driven pharmaceutical company dedicated to human health care. Established in 1995 and headquartered in Woodcliff Lake, New Jersey, Eisai Inc. has developed a robust portfolio that includes products for oncology, neurology, and neurodegenerative diseases. The company began marketing in the United States in 1997 and has since become well-regarded for its commitment to addressing unmet healthcare needs. Eisai India, a wholly-owned subsidiary founded in 2004, focuses on prescription medicines across various therapeutic areas and operates an integrated manufacturing and research facility in Visakhapatnam, which is recognized by multiple regulatory agencies. Eisai's operations emphasize collaboration with healthcare professionals to ensure they are equipped with the latest product information, ultimately aiming to enhance patient care. The company is characterized by its values of integrity, respect, professionalism, quality, and teamwork, positioning it as an innovative and efficient player in the pharmaceutical market.

Ercole Biotech, Inc., a research stage biopharmaceutical company, creates oligonucleotide drugs that achieve their therapeutic effect by directing the alternative splicing of target genes. The company was founded in 2002 and is based in Research Triangle Park, North Carolina. As of March 20, 2008, Ercole Biotech, Inc. operates as a subsidiary of AVI Biopharma, Inc.