At the recent DIA 2025Global Annual Meeting, Catalyst’s CEO Nancy Myers led a dynamic panel of distinguishedexperts who discussed how innovative Master Protocol trial designs—particularlyumbrella trials—can accelerate the path to “platformize” cell and genetherapies (CGTs) to meet the needs of the rare disease patient community. Joining us were Dr. KiranMusunuru (Professor of Medicine at University of Pennsylvania), a leading voicein Cell & Gene Therapy and a pioneer in base editing; Dr. PJ Brooks (DeputyDirector of the Division of Rare Diseases Research Innovation at NIH), adistinguished thought leader in the generation of streamlined GT developmentplatforms; and Dr. Jeff Allen (President and CEO of Friends of CancerResearch), an esteemed oncology expert and advocate.

Throughout the discussion,Nancy and panelists discussed lessons learned from the oncology field, applications ofumbrella and basket trials, and opportunities that are emerging under the newFDA administration. The conversation spotlighted umbrella trials, which evaluate multiple targetedtherapies for a single disease under one Master Protocol, enabling efficiencies via sharedinfrastructure and adaptive trial arms to speed results and approvals. These designs lay the groundworkfor platform therapies, which reuse key components across multipletreatments, allowing existing data to be reused to support the rapiddevelopment and approval of new therapies. 

Although the FDA’s Platform Technology Designation draft guidance permits data sharing acrosstherapies, it requires the first therapy of the platform to be fullyFDA-approved prior to data sharing. Many in the CGT field are eager to bypassthis prerequisite, especially in the GT space. GTs often use the same deliveryvector (e.g., viral or lipid nanoparticles) to carry different healthy genes orgene-editing instructions (i.e., guide RNAs, “gRNAs”). Platformizing GT vectorscould mean swapping in new healthy genes or editing instructions without theneed for re-evaluation and re-validation of vector safety—dramatically reducingtimelines for rare disease treatments and offering options to those withlife-threatening rare diseases. 

Panelist Dr. Musunuru’sground-breaking work exemplifies this game-changing potential: his FDAcollaboration led to the rapid, life-saving treatment of “Baby KJ” for CPS-1 deficiency and pavedthe way for FDA to endorse a novel umbrella/platform trial framework tofast-track similar therapies for other rare inherited metabolic disorders—atransformative step for the future of CGTs.

It was a pleasureconnecting with everyone who joined our DIA panel, and we’re eager to see thecontinued progress in advancing CGTs through innovative platform trialapproaches.

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