The California Institute for Regenerative Medicine (CIRM) has approved a new funding programme to accelerate therapies for rare diseases, which sets aside $100m over two years to create a scalable model that rapidly delivers transformative, platform-based genetic therapies for the millions of people in the US living with rare diseases.
The Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) programme builds on the recent success of a genetic therapy developed in six months for Baby KJ, the first infant successfully treated for a rare, life-threatening disorder called CPS1 deficiency.
RAPID is expected to accelerate the development of new treatments for many of the 1 in 10 Americans affected by rare diseases, 95% of which have no approved therapy. There are at least 10,000 unique rare diseases, each of which may have a small population, but cumulatively they affect over 30m people in the US, about half of whom are young children with limited expected lifespan.
“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 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. This framework positions CIRM to champion innovation where it’s most urgently needed, and to do so with speed, impact, and equity at the forefront.”
CIRM’s RAPID programme 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 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 to knowledge sharing includes clear requirements and timelines that go beyond other CIRM funding programmes 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 programmes 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 pre-existing investments in rare disease clinical trials, RAPID is designed to take a new approach. RAPID 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.
“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,” said Shyam Patel, CIRM associate vice president of preclinical development.