CIRM unveils RAPID funding programme for rare diseases

RAPID builds on CIRM’s previous work, including a recent genetic therapy developed within six months for CPS1 deficiency. Credit: warut pothikit / Shutterstock.com. The California Institute for Regenerative Medicine (CIRM) in the US has approved the Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) funding programme, setting aside $100m over two years. The initiative aims to speed up the development of platform-based genetic therapies for rare diseases in the US. Go deeper with GlobalDataReports LOA and PTSR Model - Celyvir in Rhabdomyosarcoma Reports LOA and PTSR Model - IMS-001 in Relapsing Remitting Multiple Sclero... Go deeper with GlobalData The gold standard of business intelligence. Find out more Discover B2B Marketing That Performs Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms. Find out more It also aims to create a scalable model to deliver these therapies, addressing the needs of more than 30 million people affected by rare conditions. RAPID builds on CIRM’s previous work, including a recent genetic therapy developed within six months for carbamoyl phosphate synthetase 1 (CPS1) deficiency. The programme will focus on accelerating treatments for rare diseases, most of which currently lack approved therapies. It will fund validation and innovation awards, of which the former target projects that have received US Food and Drug Administration (FDA) pre-investigational new drug (IND) feedback. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Validation awards fund activities through the completion of first-in-human clinical trials while innovation awards aim to minimise testing requirements or broaden applicability to various technologies and rare diseases. CIRM will require RAPID awardees to share knowledge in near-real time within its network and publish data promptly. This approach is intended to accelerate learning and regulatory alignment across related projects. CIRM preclinical development associate vice-president Shyam Patel said: “RAPID is designed to fundamentally reshape how we advance treatments for people with rare diseases. By focusing on scalable platform technologies, we’re accelerating individual projects while building an infrastructure that enables faster, more efficient development across entire categories of genetic conditions. “These therapies not only have the potential to reduce lifetime healthcare costs, but also will strengthen partnerships, streamline pathways to the clinic, and ultimately ensure that promising therapies reach patients who otherwise have no options.”