Prevail Therapeutics

Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company, is a gene therapy company focused on developing disease-modifying treatments for rare conditions and neurodegenerative diseases. The company utilizes adeno-associated virus (AAV)-based gene therapy technology to deliver healthy copies of genes to specific cells, potentially restoring the function of disease-causing genes. Prevail's lead candidates include PR001, which is being evaluated for Gaucher disease and Parkinson's disease with GBA1 mutations, and PR006 for frontotemporal dementia with GRN mutations. The company's approach involves using AAV9 vectors to transport genetic material directly into the body (in vivo) for targeted treatment. In January 2021, Eli Lilly acquired Prevail Therapeutics for approximately USD 1.04 billion, establishing a new gene therapy program within Lilly's research efforts. Prevail's technology platform includes methods for administering gene therapies through various routes, including suboccipital injections into the cisterna magna and intravenous delivery, with specific dosages for optimal therapeutic effects.

Key customers and partnerships

In January 2023, Prevail Therapeutics entered into a partnership with Capsida Biotherapeutics to develop genetic medicines for central nervous system diseases. As part of this collaboration, Prevail agreed to use Capsida's adeno-associated virus technology to create gene therapies capable of targeting the central nervous system while limiting exposure to the liver. The deal included an upfront payment of USD 55 million to Capsida, with the potential for up to USD 685 million in additional payments based on research, development, and commercial milestones. This partnership aims to advance Prevail's gene therapy programs by leveraging Capsida's expertise in AAV vector development.