CIRM Approves $100 Million Plan to Accelerate Genetic Therapies for Rare Diseases

Rare Daily Staff

The California Institute for Regenerative Medicine has approved a new funding program to accelerate therapies for rare diseases, setting aside $100 million over two years to create a scalable model that rapidly delivers transformative, platform-based genetic therapies for the millions of people in the United States living with rare conditions.

The Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) program builds on the recent success of a genetic therapy developed in just six months for Baby KJ, the first infant successfully treated for carbamoyl phosphate synthetase I (CPS1) deficiency, a rare and life-threatening disorder.

“RAPID represents a pivotal step in transforming how we deliver genetic therapies for rare diseases. Traditional development models can’t keep pace with the sheer number and diversity of rare conditions,” said Rosa Canet-Avilés, CIRM’s chief science officer. “By investing in platform-based approaches, we’re creating a scalable, efficient pathway that can accelerate multiple treatments from scientific discovery to transforming the lives of patients and their families.”

RAPID builds on CIRM’s history of funding research and development of regenerative medicine therapies for patients with rare diseases. About half of all CIRM awards that support clinical trials are for rare conditions.

Although CIRM has significant existing investments in rare disease clinical trials, RAPID is designed to take a new approach. The program will drive the technical and regulatory innovations needed to accelerate and streamline the development, delivery, and accessibility of multiple platform-based genetic therapies for patients with rare diseases.

CIRM’s RAPID program will fund two types of awards: validation and innovation. Both will seek proposals to advance multiple in vivo genetic therapies to patients with rare diseases.

Validation awards will support projects that have already received FDA pre-IND feedback with preliminary alignment on their platform approach. These awards will fund activities through completion of a first-in-human clinical trial to help demonstrate that platform-based in vivo genetic therapies can be efficiently delivered to patients.

Innovation awards will support projects that push the boundaries of what constitutes a platform by reducing testing requirements or expanding applicability across multiple rare diseases and in vivo genetic technologies.

RAPID will also include requirements for near-real-time knowledge sharing within the CIRM awardee network, as well as timely public sharing of data and knowledge. This structured approach includes clear requirements and timelines that go beyond other CIRM funding programs to accelerate collective learning and regulatory alignment.

“RAPID is designed to fundamentally reshape how we advance treatments for people with rare diseases,” said Shyam Patel, CIRM’s associate vice president of preclinical development. “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.”