CIRM unveils RAPID funding programme for rare diseases

The initiative aims to speed up the development of platform-based genetic therapies for rare diseases in the US.

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.

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.”

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