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uniQure's “game changing” data announced in September, which showed significant slowing of Huntington’s disease (HD) progression in patients treated with its gene therapy candidate AMT-130, may not be enough to secure FDA approval. We also discuss Recursion's pivotal leadership transitions, as Najat Khan, PhD, chief R&D officer and chief commercial officer, is set to take over as the company's CEO effective January 1. The AI drug developer has made big bets filling the biology data gap and recently announced a "Google Map of the brain" to advance neurodegenerative disease targets. In open-source AI for drug discovery, the release of the latest Boltz model, BoltzGen, advances the platform from structural predictions to the design of "any" therapeutic modality, all available for commercial use.
Join GEN editors Corinna Singleman, PhD, Alex Philippidis, Fay Lin, PhD, and Uduak Thomas for a discussion of the latest biotech and biopharma news.
Listed below are links to the GEN stories referenced in this episode of Touching Base:
uniQure Staggers as FDA Questions Data for Huntington’s Gene Therapy Candidate
By Alex Philippidis, GEN Edge, November 3, 2025
StockWatch: uniQure Shares Reach Five-Year High on “Game Changing” Huntington’s Data
Alex Philippidis, GEN Edge, September 28, 2025
Gene Therapy Significantly Slows Huntington Disease Progression
GEN, September 24, 2025
Recursion, Roche Unveil Microglia Map of Neuro Disease Targets
By Alex Philippidis, GEN Edge, October 29, 2025
BoltzGen Democratizes AI Therapeutic Design, Expands Druggable Universe
By Fay Lin, PhD, GEN, October 27, 2025
The State of AI in Drug Discovery On Demand
Hosted by Corinna Singleman, PhD
Hosted by Jonathan D. Grinstein, PhD
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