RAPID Awards: Accelerating the Development of Genetic Therapies for Patients with Rare Diseases

RAPID Awards: Accelerating the Development of Genetic Therapies for Patients with Rare Diseases is sponsored by California Institute for Regenerative Medicine (CIRM). Supports the development and delivery of platform-based genetic therapies for rare diseases in California.

Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) Awards Accelerating the Development of Genetic Therapies for Patients with Rare Diseases Application period is July to September 2026. The Request for Applications (RFA) is now available and the consultation period is closed.

Photo Credit: Stanford University Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) Program at CIRM The Rare Disease Acceleration Through Platform Innovation and Delivery (RAPID) program advances the development and delivery of platform-based genetic therapies for Americans with rare diseases. Current approaches to developing rare disease therapies are slow, expensive, and inefficient.

RAPID tackles the challenge of 10,000+ rare diseases—most genetic, many affecting children, and largely without approved therapies—by funding research that builds a scalable, efficient path to move multiple treatments faster into clinical trials, and eventually to patients.

By funding both validation of nascent platform approaches and innovation in novel platform technologies, RAPID aims to accelerate technical and regulatory progress so more patients can access life-changing therapies. The program also emphasizes knowledge and data sharing within its awardee network and with the public, fostering collective learning and faster regulatory alignment. RAPID Program: Key Information RAPID Validation.

The expected outcome for RAPID Validation awards is the completion of the First-in-Human trial (FIH) of at least three in vivo genetic therapy candidates that demonstrate clinical proof of concept (POC) for the platform. RAPID Innovation. The expected outcome for RAPID Innovation awards is master protocol IND clearance for a FIH trial of at least three in vivo genetic therapy candidates.

The maximum award duration for RAPID Validation is 6 years. The maximum award duration of RAPID Innovation is 3. 5 years.

RAPID Validation and RAPID Innovation do not have maximum award amounts. Below is a list of eligibility requirements for reference. Applications must meet the complete list of eligibility requirements defined in the RAPID RFA in order to be considered for CIRM funding.

The application must propose a platform comprising at least three in vivo genetic therapy candidates targeting rare diseases. The application must demonstrate that the project is at an appropriate stage of readiness at the time of application. The application must propose studies designed to achieve the expected outcome of the applicable RAPID award type.

CIRM anticipates funding 2-3 RAPID awards in fiscal year 2026-2027. Download the RAPID Request for Applications (RFA) for more information on award eligibility, review criteria, and the application process. Important: All prospective applicants MUST participate in a consultation.

How to Request a Mandatory Consultation: Please note that RAPID application is only available to applicants who have had a consultation with the Preclinical Development Team. To request a 30-minute consultation, please email the Preclinical team at preclinical@cirm. ca.

gov with the subject line “RAPID consultation request. ” The RAPID consultation period is closed. 1.

Go to the Grants Management Portal 2. After logging in, click on the Menu tab Select the tab labeled “Open Programs” under the section labeled “RFAs and Programs Open for Applications,” and click on the “Start a Grant Application” link for the selected program. 3.

Complete each section of the Application Click on the appropriate link and follow the instructions. Proposal templates can be located and submitted under the “Document Uploads” section. 4.

To submit your Application Click on the “Done with Application” button. After completing all the mandatory sections, the “Done with Application” button will be enabled. Note: Once submitted, you can no longer make changes to your application.

5. To confirm submission of your Application Select the tab labeled “Your Applications” and check the table under the section labeled “Your Submitted Applications. ” Once the submission process has been completed, you will see your application number and project title listed.

The application period for the RAPID program opens in July and closes in September 2026.

CIRM Grants Policies, FAQs, and Primers Data Sharing and Management Requirements Patient Access Planning Requirements Allowable Costs and co-funding FAQ Commercialization Rights Primer (IP, Revenue Sharing, Pricing, Access, March-in Rights) Grants Administration Policy for Clinical Stage Projects Clinical Development Resources Industry Resource Partner Program CIRM California Academic Cell and Gene Therapy Manufacturing Network CIRM Alpha Clinics Network RAPID Program Application Resources Application Selection Process: Volume Control Video Application Selection Process: Volume Control Video Deck [PDF] What is a “platform” in RAPID; how many candidates must be included?

A platform in RAPID is a common set of technologies that can be leveraged for more resource-efficient development, manufacture, clinical delivery, and regulatory review across multiple related therapies. RAPID is intended to support programs where candidates share meaningful preclinical, CMC, and clinical development elements in a way that reduces redundant testing and supports scalable addition of new candidates.

Platforms must comprise at least three in vivo genetic therapy candidates and maintain that minimum throughout the project. In practice, RAPID is not looking for a loose collection of related assets. Applicants will need to show that the candidates are connected through a robust development platform that creates time and cost efficiencies and supports expansion to additional candidates with reduced incremental burden.

What is the difference between RAPID Innovation and RAPID Validation? How should applicants decide which type of award is the right fit? Both award types are for platform-based in vivo genetic therapy programs for rare diseases, require a platform with at least three candidates, disease-modifying activity for at least one candidate, and at least a plausible biological mechanism for the others.

Both also require platform-relevant FDA engagement covering all proposed candidates. The main differences are stage, regulatory readiness, supported activities, and expected outcome: RAPID Validation is for projects that have already completed a pre-IND meeting and have preliminary FDA alignment on the platform approach.

Validation awards will support all activities required for master protocol IND clearance and to conduct a FIH master protocol clinical trial. The expected outcome is FIH clinical trial completion and generation of clinical proof of concept for the platform. RAPID Innovation is for earlier-stage platform projects.

Applicants must have completed or requested an INTERACT meeting at the time of application. Innovation awards will support activities from platform optimization through IND-enabling work and submission of a master protocol IND but not conduct of the clinical trial beyond startup activities. The expected outcome is master protocol IND clearance.

Essentially, applicants should choose Innovation if the platform is still earlier and needs to be derisked through early FDA engagement and preclinical/IND-enabling work and should choose Validation if the platform is more mature, has already had pre-IND interaction, and is ready to move toward a master protocol FIH trial. Is there a preference for specific types of rare diseases? RAPID does not prioritize specific disease areas.

Instead, it is intended to support a diverse portfolio of rare diseases that are a strong fit for a platform-based approach. The key consideration is whether the disease(s) can be efficiently addressed using a shared platform, not the specific indication. Note: CIRM defines rare disease as a disease with a prevalence of <200,000 patients in the U.S. If one candidate fails, does the entire platform fail?

Not necessarily. RAPID is structured around a platform, not a single candidate. The rationale for the program is that shared platform components generate cumulative knowledge that can support multiple related candidates and strengthen future development.

A single candidate setback would not automatically mean the platform fails, but the impact would depend on whether the issue undermines the broader platform and whether the three-candidate minimum can be maintained throughout the project. Additional candidates may be added during the project with prior CIRM approval and FDA alignment. Requests will be evaluated on a case-by-case basis.

Can an investigator be included in more than one RAPID application? The principal investigator may not apply for more than one RAPID award within the same funding cycle. The same individual may be named as key personnel on multiple RAPID applications, including those submitted within the same funding cycle.

Are pre-application consultations required? How do I schedule one? Yes.

All prospective applicants must participate in a mandatory consultation with CIRM staff before submitting an application. The purpose is to ensure that proposed platform-based therapies meet RAPID eligibility, scope, and readiness requirements and to provide guidance on application expectations. Prior to the consultation, applicants are required to submit key information about their proposed project.

This includes completion of the Eligibility Upload Document, where applicants should provide relevant data and supporting information demonstrating candidate and readiness eligibility. Please visit our program page to request a consultation.