California’s CIRM aims to speed therapies’ move from lab to clinic
Agency funds $100M effort to build platform for gene therapy delivery
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The California Institute for Regenerative Medicine (CIRM) approved a $100 million funding program designed to speed the development of gene therapies for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency.
The Rare Disease Acceleration Platform and Innovation and Delivery (RAPID) program will invest the funds over two years to create a scalable, platform-based model for delivering genetic therapies.
The goal is to move treatments from the lab to patients far more quickly than traditional drug development allows. CIRM said it aims to establish a model that could make speed, collaboration, and scalability central to rare disease drug development, offering new hope to patients who have long been left without viable treatment options.
“RAPID represents a pivotal step in transforming how we deliver genetic therapies for rare diseases,” Rosa Canet-Avilés, PhD, CIRM chief science officer, said in a CIRM press release. “Traditional development models can’t keep pace with the sheer number and diversity of rare conditions. 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.”
CIRM said RAPID builds on the recent success of a gene therapy developed in six months for Baby KJ, the first infant successfully treated for CPS1 deficiency, a rare and life-threatening metabolic disorder. That rapid turnaround highlighted how platform-based approaches could be used to tackle many rare conditions more efficiently.
Awards for validation, innovation
The program will offer two types of awards — validation and innovation — with the overall goal of funding proposals that advance multiple in vivo gene therapies rather than a single treatment for a single disease.
Validation awards will support proposals that have received early feedback from the U.S. Food and Drug Administration (FDA) through a pre-investigational new drug review, a step that occurs before a company formally applies for permission to test a new therapy in people. This feedback indicates early regulatory agreement with a project’s platform-based genetic therapy approach. The funds will help finance first-in-human clinical trials to show that the platform-based gene therapies can be delivered safely and efficiently to patients.
Innovation awards will back proposals that aim to push the boundaries of platform development, such as reducing testing requirements or expanding a single platform’s use across multiple rare diseases or genetic technologies.
A key feature of RAPID is its emphasis on near-real-time knowledge sharing. Awardees will be required to share study designs, emerging data, and regulatory strategies within the CIRM-funded network, as well as make certain information public on defined timelines.
For example, FDA feedback from pre-IND meetings must be publicly shared within six months. CIRM says this approach will help align regulatory strategies across related therapies and allow both funded and non-funded projects to benefit from shared learnings, accelerating progress across the field.
CIRM has a history of supporting rare disease research, with about half of its clinical trial funding going toward rare conditions such as muscular dystrophies. Traditionally, however, these efforts have focused on one therapy for one disease at a time, each requiring a full and separate development pathway.
RAPID is intended to change that model by driving technical and regulatory innovations that allow multiple therapies to be developed, tested, and delivered using shared platforms.
“RAPID is designed to fundamentally reshape how we advance treatments for people with rare diseases,” said Shyam Patel, PhD, CIRM 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. 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.”
