Written by Holly MacCormick
South San Francisco, CA, January 30, 2026 – As many as 1 in 10 people in the U.S. has a rare disease, many of which have no known cure. A new program by the California Institute for Regenerative Medicine (CIRM) could change this by funding novel ways to develop multiple genetic therapies for multiple rare diseases at the same time.
The rare disease funding program was one item that came before the January 29 meeting of CIRM’s governing board, known as the Independent Citizens Oversight Committee (ICOC). At this same meeting, board members received updates from two grantees who are running CIRM-funded clinical trials, heard preliminary results of a new process for prioritizing applications, and approved both new reimbursement rates for awardees as well as revisions to the time commitment of the Chair.
Support for rare disease therapies
The new rare disease funding program approved by the board will allocate $100 million over two years to support multiple platform technologies for rare diseases. These platforms each represent one approach that could ultimately result in treatments for many diseases. That’s good news for those patients and their families who might otherwise have no options. This new approach for developing therapies for rare diseases is known as the Rare Disease Acceleration Platform and Innovation and Delivery (RAPID).
“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 streamline the path of therapies to the clinic and ultimately ensure that promising therapies reach patients who otherwise have no options.”
Preference setting
RAPID is the latest research funding concept to arise from CIRM’s new strategic allocation framework, which sets priorities for CIRM spending over the lifetime of the remaining funds allocated by Proposition 14 in 2020. The new framework defines a funding cadence and priorities that will accelerate new therapies and enable CIRM to put cell and gene therapies within reach of people in California.
To help CIRM maximize the impact of remaining funds, achieve goals laid out in the new framework, and handle the large volume of applications, the agency adopted a step in which staff review applications against a set of preferences. Those that met the most preferences moved on to full scientific review by the Grants Working Group.
At the January meeting, Rosa Canet-Avilés, CIRM Chief Scientific Officer, discussed this new mechanism and gave an overview of the initial results of using it to filter applications.
“CIRM’s challenge is not whether there is strong science to fund, but how to prioritize among many meritorious applications to maximize the likelihood of delivering impactful therapies to patients within CIRM’s remaining years,” said Canet-Avilés. “Preferences help us navigate the realities of finite funds and a limited runway.”
- Read the memo, including preliminary results
Closer to Cures
This meeting marked the start of a new speaker series, called Closer to Cures, which will feature presentations from CIRM-funded grantees at each ICOC meeting.
Crystal Mackall, MD, the founding director of the Stanford Medicine Center for Cancer Cell Therapy and director of the Parker Institute for Cancer Immunotherapy at Stanford, discussed results of a clinical trial testing a new possible treatment for brain cancer that involves modifying a patient’s own immune system cells so they will identify and destroy cancer cells — an approach known as CAR T. Cory Nicholas, PhD, the CEO and co-founder of Neurona Therapeutics, presented results of a clinical trial testing a stem cell-based therapy for epilepsy.
Mackall’s Presentation:
Nicholas’ Presentation:
Additional action items
The board also approved updated facilities reimbursement rates for awardees, which is part of the new Award Management Policy approved at the December board meeting. The new reimbursement rates simplify the Facilities rate structure for For-Profit Awardees and Non-Profit Awardees that do not already have a federally negotiated rate.
Lastly, the board approved a request from ICOC Chair Vito Imbasciani regarding a reduction in percent effort and salary from 100% to 50% starting March 1, 2026. The ICOC heard how the duties of the Chair could be accomplished at 50% effort, and how the remainder of the activities could be accomplished with existing Board and CIRM team resources.
The Stem Cellar 