This quarter, the Alliance for Regenerative Medicine released its Sector Snapshot, capturing early progress across delivery platforms, clinical programs and regulatory pathways in 2025.
In vivo delivery continues to advance, including the first trial of a CAR gene therapy in Europe, the first in vivo beta-thalassemia trial using gene editing, and only the second in vivo CRISPR program to reach Phase 3. Delivery systems such as lipid nanoparticles and smaller CRISPR enzymes are helping in vivo approaches reach more disease areas, with signs of improved efficiency.
Clinical results this quarter show encouraging movement in areas where standard care is limited. Bayer and BlueRock’s Parkinson’s program has moved into Phase III, and Aspen Neuroscience and AskBio reported early signals of motor improvement. Sana Biotechnology shared data from its islet cell therapy in type 1 diabetes, with no immunosuppression required. Solid Biosciences’ Duchenne muscular dystrophy program showed early indications of cardiac benefit.
The FDA’s Accelerated Approval pathway is beginning to play a bigger role. Six therapies could be approved through this route in 2025 or 2026. Europe may also see a shift, with several regulatory decisions expected after two slower years.
While commercial uptake remains concentrated in a small number of therapies, the broader pipeline continues to build. The focus now is on scaling delivery platforms, generating consistent clinical data, and moving through regulatory review without delay.
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