CIRM has launched the Rare Disease Acceleration Platform and Innovation and Delivery (RAPID), allocating $100 million over two years to fast-track platform-based genetic therapies in the US. The programme aims to develop scalable delivery models to reach more than 30 million people with rare conditions and builds on rapid-development work such as a six-month CPS1 therapy. RAPID will issue validation awards to move FDA pre-IND–endorsed projects through first-in-human trials and innovation awards to broaden applicability or reduce testing burdens. Awardees must share data rapidly to speed collective learning and regulatory alignment.
CIRM Launches $100M RAPID Programme to Accelerate Platform Genetic Therapies for Rare Diseases
RAPID builds on CIRM’s previous work
The California Institute for Regenerative Medicine (CIRM) has approved the Rare Disease Acceleration Platform and Innovation and Delivery (RAPID), committing $100 million across two years to accelerate development of platform-based genetic therapies for rare diseases in the United States.
Programme Goals
RAPID aims to both speed individual therapy development and create scalable delivery models so that genetically based treatments can reach the more than 30 million Americans affected by rare conditions. The initiative builds on CIRM’s recent rapid-development work, including a genetic therapy developed within six months for carbamoyl phosphate synthetase 1 (CPS1) deficiency, demonstrating what platform approaches can achieve.
Funding Streams
The programme will award two types of grants:
- Validation Awards: Target projects that have received pre-investigational new drug (pre-IND) feedback from the US Food and Drug Administration (FDA). These awards support work through the completion of first-in-human clinical trials.
- Innovation Awards: Support strategies that reduce preclinical testing requirements or broaden a technology’s applicability across multiple rare diseases and platforms, increasing scalability and efficiency.
Open Sharing To Accelerate Progress
CIRM will require RAPID awardees to share knowledge in near–real time within its network and to publish data promptly. This open-sharing requirement is designed to accelerate collective learning, improve regulatory alignment across related projects and reduce duplicated effort.
“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,”
Implications and Next Steps
By prioritising platform approaches and enforced data sharing, RAPID could shorten timelines to clinic, strengthen collaborative partnerships, and ultimately increase the number of rare-disease therapies reaching patients. CIRM is expected to publish specific application timelines, eligibility criteria and programme details on its website for prospective applicants.
Note: This article summarizes CIRM’s announcement for informational purposes and is not medical or investment advice.
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