CIRM RAPID: Funding for Rare Disease Platform Projects
Below is a brief summary. Please check the full solicitation before applying (link in resources section).
Quick Answer
CIRM's RAPID program (Rare Disease Acceleration Through Platform Innovation and Delivery) funds California organizations developing platform-based in vivo genetic therapies for rare diseases (fewer than 200,000 US patients). There is no maximum award amount. The program has two tracks: RAPID Innovation (pre-IND through IND clearance, up to 3.5 years) for earlier-stage platforms, and RAPID Validation (IND-enabling through first-in-human trial completion, up to 6 years) for platforms with completed FDA Pre-IND alignment. Applicants must propose at least three in vivo genetic therapy candidates sharing common development infrastructure, commit a Principal Investigator (15% effort minimum) and Project Manager (50% effort minimum), and complete a mandatory pre-application consultation with CIRM before submitting. CIRM expects to fund 2–3 awards in the FY 26-27 cycle.
What Is the CIRM RAPID Program?
The California Institute for Regenerative Medicine (CIRM) launched RAPID to accelerate the development of platform-based genetic therapies for rare diseases. Rather than funding one therapy at a time, RAPID supports platforms: a shared set of technologies, manufacturing processes, and clinical infrastructure that can be reused across multiple related therapeutic candidates. The goal is to reduce per-indication development cost and timeline by letting each new candidate leverage work already done for the platform, ultimately advancing 4–7 rare disease programs toward a Biologics License Application (BLA).
CIRM positions itself as an active development partner, not just a funder — awardees gain access to the CIRM Awardee Knowledge Network, the CIRM Product Development (PDEV) Expert Network of contracted CMC, clinical, nonclinical, and regulatory advisors, and optional use of the CIRM Clinical Network (Alpha Clinics) and CIRM Cell and Gene Therapy Manufacturing Network.
RAPID Validation vs. RAPID Innovation: Which Track Fits Your Program?
RAPID Innovation is for earlier-stage platforms spanning pre-IND through IND-enabling activities. It requires that an FDA INTERACT meeting has been completed, or that the meeting request has been submitted, at the time of application. The expected outcome is Master Protocol IND clearance for at least three in vivo candidates, and the maximum award duration is 3.5 years. RAPID Innovation does not fund clinical trial conduct — only start-up activities are allowable, with no patient recruitment, screening, or enrollment.
RAPID Validation is for more advanced platforms that have already completed an FDA Pre-IND meeting covering the platform and all proposed candidates. It supports activities spanning IND-enabling studies through full conduct of a first-in-human (FIH) master protocol clinical trial, with a maximum award duration of 6 years. The expected outcome is a completed FIH trial demonstrating clinical proof of concept for the platform.
Neither track specifies a maximum award amount. Both require the platform to maintain at least three shared in vivo genetic therapy candidates throughout the project period.
Who Is Eligible to Apply?
To be considered, an application must satisfy all 13 eligibility criteria in the RFA, most notably:
Platform scope — at least three in vivo genetic therapy candidates for rare diseases sharing preclinical, CMC, and clinical development elements.
Stage readiness — reproducible disease-modifying activity demonstrated in a relevant preclinical model for at least one candidate, plus a plausible mechanism of action for the additional candidates.
Regulatory readiness — a completed FDA Pre-IND meeting (RAPID Validation) or a submitted/completed FDA INTERACT meeting request (RAPID Innovation) covering the full platform.
Study design aligned to the applicable track's expected outcome (IND clearance for Innovation; completed FIH trial for Validation).
Principal Investigator committing a minimum of 15% effort.
Project Manager with relevant experience, committing a minimum of 50% effort.
Data Project Manager on the team (role may be shared with other Key Personnel).
IND sponsorship — the CIRM applicant organization (or PI, for an investigator-sponsored IND) must be the named IND sponsor.
California trial site — at least one clinical trial site located in California, with justification required for any out-of-state sites.
90-day start — the applicant must be ready to initiate award activities within 90 days of ARS approval.
Complete, accurate application with no false or inaccurate information.
California Organization status per CIRM's standard definition.
Solvency (for-profit applicants) and good standing for all applicants and affiliates.
Both non-profit and for-profit California organizations may apply. An institution may submit more than one RAPID application per funding cycle.
What Does CIRM Fund — and What Won't It Fund?
Fundable activities span both tracks and include:
IND-enabling nonclinical GLP studies (pharmacodynamics, pharmacokinetics, safety/toxicology, mechanism-of-action and efficacy confirmation)
Platform expansion studies requested by FDA to add candidates or indications
GMP process development, technology transfer, and manufacturing of therapeutic candidates
Analytical assay development and qualification, QTPP/CQA/CPP development
Clinical protocol development, clinical operations planning, and (for RAPID Validation) full conduct of the FIH master protocol trial
Preparation for and conduct of FDA Pre-IND/INTERACT meetings and master protocol IND submission
Patient access, market access, and reimbursement strategy planning
Data management, preservation, and sharing activities
Not funded under RAPID:
Clinical trial conduct beyond start-up activities under RAPID Innovation (no patient recruitment, screening, or enrollment)
Activities already budgeted, paid, or funded under another CIRM award or outside funder
Costs for work performed by an out-of-state organization that retains independent IP or publication rights to project outputs
Costs incurred on or before the date of ICOC (CIRM governing board) approval
See Appendix A of the RFA for a full category-by-category breakdown of allowable nonclinical, CMC, clinical, regulatory, and patient-access-planning activities by track.
How to Apply: Step by Step
Complete a mandatory pre-application consultation. Email preclinical@cirm.ca.gov with the subject line "RAPID Consultation Request" to receive and submit the online Consultation Form (core team, project title, award type, platform description, mechanism of action, indication, prior regulatory interactions, prior CIRM funding, and an Eligibility Upload Document using CIRM's template). This step is required before the online application will open to you.
Create a login at the CIRM Grants Management Portal (https://grants.cirm.ca.gov). Only the PI can access and submit the application, and a PI may submit only one RAPID application per review cycle.
Complete the online application, including the eligibility form, Key Personnel listing, and budget.
Prepare the Application Proposal using CIRM's required templates, covering: Project Summary, Target Product Profile, Value Proposition, Scientific Rationale, Preclinical and Clinical Studies summaries, Project Plan (preclinical, CMC, clinical), Milestones and Gantt timeline, FDA Correspondence summary, Patient Access and Commercialization Plans, Team Organization, Risk Mitigation and Financial Contingency plans, Resources and Project Environment, and a Data Sharing Overview.
Build an activities-based budget with justification detailed enough for CIRM budget staff to assess reasonableness; subcontracts over $500,000 require at least three comparative proposals or a sole-source justification.
Review Process and Timeline
The GWG — fifteen outside-California scientific experts plus patient advocate/nurse members of CIRM's governing board (ICOC) — scores applications 1–100 on scientific and technical merit. A median score of 85 or above is required to be recommended for funding, subject to fund availability. Patient advocate and nurse GWG members separately assign a 1–5 Patient Perspective Score, which does not determine the funding recommendation directly but can influence GWG discussion and the ARS's final funding decision.
Scientific review criteria cover five areas: Value Proposition, Rationale, Project Plan & Design, Project Team and Resources, and Population Impact. Patient Perspective criteria cover Relevance, Patient Benefit, Patient Centeredness, Patient Engagement, and California Benefit.
Award Size, Duration, and Budget Rules
No maximum award amount for either track; total costs must be justified and are subject to adjustment by the GWG, CIRM staff, or the ARS.
RAPID Innovation: up to 3.5 years. RAPID Validation: up to 6 years.
CIRM expects to fund 2–3 awards in the FY 26-27 cycle.
Supplemental funding may be requested at defined milestones (e.g., after a pre-IND meeting, or to add new candidates) but is not guaranteed.
Direct Facilities Costs: limited to federally negotiated rates for non-profits with an existing agreement; capped at 35% of direct project costs for for-profit applicants or non-profits without a negotiated rate.
Indirect Costs: capped at 20% of allowable direct research funding costs for non-profits; for-profit organizations may not claim indirect costs.
Ongoing Awardee Obligations
90-day start rule: award activities must begin within 90 days of ARS approval.
Operational Milestones: funds are disbursed against CIRM-defined milestones; missing a milestone by more than four months (without CIRM-approved resolution) can result in termination.
Data and knowledge sharing: awardees must manage, preserve, and share Applicable Data under a Data Sharing and Management Plan (DSMP), participate in the CIRM Awardee Knowledge Network, and publicly share FDA regulatory feedback (e.g., Pre-IND meeting minutes) within 90 days of receipt.
PDEV Expert Network review: all regulatory strategies and submission packages must be reviewed with CIRM and PDEV advisors before FDA submission; non-participation can result in termination.
Reporting: periodic written progress and financial reports; RAPID Validation awardees must also report clinical trial enrollee demographic data.
Frequently Asked Questions
Is there a maximum RAPID award amount? No. CIRM does not specify a funding cap for RAPID Validation or RAPID Innovation awards; the total requested amount must be well-justified and is subject to review and adjustment.
How many in vivo genetic therapy candidates must be included in a platform? At least three, maintained throughout the entire project period, sharing preclinical, CMC, and clinical development elements.
Do I need an FDA meeting before applying? Yes. RAPID Validation requires a completed FDA Pre-IND meeting covering the platform and all candidates. RAPID Innovation requires that an FDA INTERACT meeting request has been submitted (or completed) at the time of application.
Can RAPID Innovation funds be used to run a clinical trial? No. RAPID Innovation supports activities through Master Protocol IND clearance and trial start-up only — patient recruitment, screening, and enrollment are not allowable.
Who can apply? Non-profit or for-profit organizations that meet CIRM's definition of a California Organization, with at least one clinical trial site located in California.
Is a pre-application consultation really required? Yes — CIRM requires applicants to complete a consultation with the Preclinical Development team, including submission of an Eligibility Upload Document, before the online application becomes accessible.
How is scientific merit scored? The Grants Working Group scores applications 1–100. A median score of 85 or above is needed for a funding recommendation, subject to available funds.
What happens to FDA feedback shared with CIRM? RAPID has enhanced knowledge-sharing requirements: FDA Pre-IND meeting feedback must generally be shared publicly within 90 days of receipt to support broader regulatory learning across the field.
BW&CO Consulting advises deep-tech, biotech, and healthtech companies on non-dilutive federal and state funding strategy, including CIRM, SBIR/STTR, and other non-contingency-fee funding pathways. Contact us to evaluate your platform's fit for the RAPID program.
