uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy
uniQure; AMT-130; Huntington’s disease; gene therapy; FDA; accelerated approval; regulatory setback; clinical trial
Roche Pays $55M Upfront to Manifold Bio in $2B+ Deal for AI-Guided Next-Gen Brain Shuttles
Roche; Manifold Bio; blood-brain barrier; BBB shuttles; neurological diseases; AI drug discovery; research deal; upfront payment; milestones; therapeutic payloads
‘Trying to take me out’: Tidmarsh’s exit at FDA pushes an agency in transition toward turmoil
FDA; George Tidmarsh; resignation; personal conduct probe; Kevin Tang; Center for Drug Evaluation and Research (CDER); agency turmoil; Vinay Prasad; leadership transitions; regulatory authority
Zag Bio Launches with $80M to Teach the Immune System to Make Better Tregs
Zag Bio; Tregs; regulatory T cells; autoimmune diseases; thymus-targeted therapy; Series A funding; immune tolerance; Type 1 diabetes; AbbVie; Regeneron; Sanofi
Expert Flags Potential ‘Honeymoon Effect’ as Rapport Heads Towards Phase III Epilepsy Trials
KOL Bulletin; honeymoon effect; Rapport Therapeutics; RAP-219; Phase III trials; epilepsy; drug-resistant focal seizures; seizure reduction; mid-stage data; biomarker
Dyne Therapeutics’ z-rostudirsen Shows Durable Efficacy, Supporting Positive Regulatory Outlook
Dyne Therapeutics; z-rostudirsen; DELIVER trial; Duchenne muscular dystrophy (DMD); durable efficacy; regulatory approval; biomarkers; functional improvement; accelerated approval
ESMO 2025 KEYNOTE-671 Update: Perioperative Pembrolizumab Reinforces NSCLC Standard
KEYNOTE-671; pembrolizumab; KEYTRUDA; perioperative immunotherapy; NSCLC; overall survival; event-free survival; ESMO Congress; standard of care
Otsuka Acquires Asia-Pacific Rights to 4D Molecular Therapeutics’ Retinal Gene Therapy 4D-150 for $85 Million
Otsuka; 4D Molecular Therapeutics; 4D-150; Asia-Pacific rights; gene therapy; wet age-related macular degeneration; diabetic macular edema; $85 million; strategic partnership; ophthalmology
Gilead Cell Therapy Sales Drop 11% in Q3 2025, HIV Franchise Drives Growth
Gilead Sciences; cell therapy sales; Q3 2025 financial results; HIV franchise; Yescarta; Tecartus; Biktarvy; Yeztugo; Livdelzi; Trodelvy; Veklury
Researchers Publish Roadmap of FDA Interactions After Baby KJ’s Groundbreaking Gene Editing Therapy, as FDA Signals New Pathway for Patient-Specific Treatments
Baby KJ; CRISPR gene editing; CPS1 deficiency; FDA approval pathway; personalized medicine; rare disease; regulatory roadmap; platform trials; N-of-1 therapies; rare disease gene therapies