South San Francisco, Calif. — January 30, 2026 — Leads & Copy — The California Institute for Regenerative Medicine (CIRM) has approved a new funding program allocating $100 million over two years to expedite therapies for rare diseases. The program aims to establish a scalable model for rapidly delivering transformative, platform-based genetic therapies to millions of Americans affected by rare diseases.
The Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) program is based on the recent success of a genetic therapy developed in six months for Baby KJ, the first infant successfully treated for CPS1 deficiency, a rare, life-threatening disorder.
RAPID is anticipated to accelerate the creation of new treatments for many of the Americans affected by rare diseases. According to the press release, 95% of rare diseases have no approved therapy. While each of the at least 10,000 unique rare diseases may have a small patient population, cumulatively, they affect over 30 million people in the U.S., about half of whom are young children with limited expected lifespans.
CIRM Chief Science Officer Rosa Canet-Avilés, PhD, stated that RAPID marks a pivotal step in transforming the delivery of genetic therapies for rare diseases. She added that traditional development models cannot keep pace with the number and diversity of rare conditions. CIRM is investing in platform-based approaches to create a scalable, efficient pathway that can accelerate multiple treatments from scientific discovery to transforming the lives of patients and their families. Canet-Aviles said this framework positions CIRM to champion innovation where it is most urgently needed, with speed, impact, and equity.
CIRM’s RAPID program will fund two types of awards: validation and innovation. Both awards will seek proposals that will 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 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 to knowledge sharing includes clear requirements and timelines that go beyond other CIRM funding programs to accelerate collective learning and regulatory alignment. For example, sharing study designs, emerging data, and regulatory strategies within the CIRM awardee network may help RAPID projects, as well as other CIRM projects, align and coordinate testing and regulatory strategies across related genetic therapies. Similarly, public sharing of FDA feedback within six months of a pre-investigational new drug meeting helps ensure that other projects, whether CIRM-funded or not, collectively benefit from the learnings and build the evidence base for platform-based genetic therapy development.
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 diseases, including LAD-1, forms of muscular dystrophy, Danon Syndrome, Machado-Joseph disease, Pitt Hopkins syndrome, and many others. These programs are focused on the traditional way of developing new therapies, which is to focus a project on a single candidate for a single indication, do the full suite of testing for every one of those projects, and to conduct a clinical trial for a single candidate.
Although CIRM has significant pre-existing investments in rare disease clinical trials, RAPID is designed to take a new approach and 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 Associate Vice President of Preclinical Development Shyam Patel, PhD, said that RAPID is designed to reshape how treatments are advanced for people with rare diseases. By focusing on scalable platform technologies, CIRM is accelerating individual projects while building an infrastructure that enables faster, more efficient development across entire categories of genetic conditions. Patel added that these therapies have the potential to reduce lifetime healthcare costs, strengthen partnerships, streamline pathways to the clinic, and ensure that promising therapies reach patients who otherwise have no options.
CIRM was created to fund stem cell and gene therapy research with the goal of accelerating treatments for patients with unmet medical needs. CIRM supports stem cell and gene therapy discoveries from inception through clinical trials, trains a workforce in California, and creates infrastructure to make clinical trials accessible for people throughout California. All of CIRM’s programs are designed to benefit the people of California.
Source: California Institute for Regenerative Medicine (CIRM)
