CIRM Approves $100 Million for Platform-Based Gene Therapies for Rare Diseases

Rare Daily Staff

The California Institute for Regenerative Medicine is launching a new funding initiative aimed at accelerating the development of genetic therapies for rare diseases by shifting away from traditional one-drug, one-disease models.

The program, known as RAPID, is designed to support platform-based approaches that can be applied across multiple conditions, while reducing redundant testing requirements and improving coordination with regulators. The initiative emphasizes scalable technologies, including in vivo gene therapies, with the goal of speeding development timelines and expanding patient access.

A central feature of RAPID is its structured approach to data sharing. Award recipients will be required to share study designs, emerging data, and regulatory strategies within the CIRM network in near real time. They also must publicly disclose key regulatory feedback, including responses from the U.S. Food and Drug Administration, within six months of pre-investigational new drug meetings.

CIRM officials say the approach is intended to build a shared evidence base that can inform multiple programs simultaneously, helping align clinical and regulatory strategies across related therapies.

CIRM’s RAPID program will fund two types of awards: validation and innovation. Both will seek proposals that advance multiple

CIRM Approves $100 Million for Platform-Based Gene Therapies for Rare Diseases

Rare Daily Staff

The California Institute for Regenerative Medicine is launching a new funding initiative aimed at accelerating the development of genetic therapies for rare diseases by shifting away from traditional one-drug, one-disease models.

The program, known as RAPID, is designed to support platform-based approaches that can be applied across multiple conditions, while reducing redundant testing requirements and improving coordination with regulators. The initiative emphasizes scalable technologies, including in vivo gene therapies, with the goal of speeding development timelines and expanding patient access.

A central feature of RAPID is its structured approach to data sharing. Award recipients will be required to share study designs, emerging data, and regulatory strategies within the CIRM network in near real time. They also must publicly disclose key regulatory feedback, including responses from the U.S. Food and Drug Administration, within six months of pre-investigational new drug meetings.

CIRM officials say the approach is intended to build a shared evidence base that can inform multiple programs simultaneously, helping align clinical and regulatory strategies across related therapies.

CIRM’s RAPID program will fund two types of awards: validation and innovation. Both will seek proposals that advance multiple in vivo genetic therapies for 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.

The program builds on CIRM’s existing portfolio, in which roughly half of its clinical-stage investments target rare diseases such as leukocyte adhesion deficiency type 1, muscular dystrophies, Danon syndrome, Machado-Joseph disease, and Pitt-Hopkins syndrome. Historically, those efforts have followed a conventional model centered on a single therapeutic candidate for a single indication, each requiring a full set of preclinical and clinical studies.

RAPID aims to move beyond that framework by encouraging shared learnings and reusable development strategies, potentially lowering costs and shortening timelines across programs. CIRM officials also say the model could improve regulatory consistency and strengthen collaboration among developers.

“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.”

genetic therapies for 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.

The program builds on CIRM’s existing portfolio, in which roughly half of its clinical-stage investments target rare diseases such as leukocyte adhesion deficiency type 1, muscular dystrophies, Danon syndrome, Machado-Joseph disease, and Pitt-Hopkins syndrome. Historically, those efforts have followed a conventional model centered on a single therapeutic candidate for a single indication, each requiring a full set of preclinical and clinical studies.

RAPID aims to move beyond that framework by encouraging shared learnings and reusable development strategies, potentially lowering costs and shortening timelines across programs. CIRM officials also say the model could improve regulatory consistency and strengthen collaboration among developers.

“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.”