Strategic Execution for Cell and Gene Therapy Programs
RareMoon supported the first RMAT ever granted by the FDA in 2017 and has secured approaching a dozen ( as of June 2025) RMAT designations with a 100% success rate since. Our success is driven by sustained, strategic collaboration with the FDA’s Office of Therapeutic Products (OTP) and a state-of-the-art understanding of RMAT eligibility, regulatory expectations, and post-designation strategy.
Our RMAT submissions are guided by regulatory experts who have served in pivotal FDA roles on both sides of the table — including Dr. Rachel Witten and Dr. Bruce Schneider, former FDA clinical leaders who reviewed and designated RMATs while at the Agency, and who now lend their in depth insight and expertise to RareMoon’s client programs.
Established by the FDA under the 21st Century Cures Act to expedite the development and review of regenerative medicine products, RMAT offers sponsors earlier and more intensive interaction with the FDA — similar to Breakthrough Therapy Designation — while introducing greater flexibility in clinical development and review.
Programs granted RMAT may benefit from:
- Priority Review eligibility at the time of marketing application
- Accelerated Approval based on surrogate endpoints or intermediate clinical measures
- Early and frequent FDA interactions to align on trial design, CMC, and long-term follow-up
- Increased flexibility in regulatory development pathways for advanced therapies
How we partner with you:
- Strategic assessment and feasibility of RMAT eligibility.
- Authoring and submission of successful designation requests.
- Post-designation regulatory planning, including FDA meetings and planning.
- Cross-functional alignment across CMC, clinical, nonclinical, and long-term follow-up requirements.
RareMoon brings unmatched insight, proven execution, and sustained strategic guidance to help you progress your therapy from RMAT request to regulatory approval.
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