uniQure

Marty Makary Steps Down as FDA Commissioner After Complaints, Adding to Regulatory Uncertainties

UniQure Pauses Higher Doses in Fabry Study; Aro Shares Pompe Data

4 Spaces Primed for the Next Wave of Gene Therapies

Replimune Cries Foul on Regulatory Flexibility. But Many Americans Want a Stricter FDA

World Hemophilia Day 2026: New Treatment Era and Improving Diagnosis

Latus Bio Secures $97 Million Series A to Scale Gene‑Therapy Access

Advanced Therapies Week 2026: ‘Solving for Science’ and Weathering Global Complexity

Deep Dive: Huntington’s at a Crossroads

UniQure Pushes Forward As FDA Rare Disease Controversies Continue

FDA Bolsters Bespoke Therapy Framework with New Draft Safety Guidelines

Seven Biotech Companies to Know in the Netherlands 

Liver Toxicities Force Pause on Two Dose Groups of UniQure’s Mid-Stage Fabry Study

Eisai Licenses Nuvation Lung Cancer Pill; Alnylam's Stock Dips After Sales Data Report

FDA, After Turbulent Year, Leaves Drugmakers Guessing on Its Direction

STAT+: A Huntington’s Researcher on the UniQure-FDA Fray

Opinion: Prasad’s FDA Exit Good for Rare Diseases but New CBER Head Must Repair Eroded Trust

FDA Reconsiders Ebvallo Cell Therapy After Surprise Rejection

Latus Bio Secures $97 Million Series A to Scale Gene‑Therapy Manufacturing

Biotech Investors Bet on a 2026 Rebound as Deal Activity Accelerates

Rare Disease Advocacy Group Urges Trump Administration to Restore FDA Clarity

Kurma Partners Closes €215 Million Biofund IV to Back European BioTech Ventures From Paris

Kennedy’s Vaccine Agenda Stalled, Structure’s ‘Competitive’ Obesity Pill, Novo’s Warning Letter

FDA Reversals in Rare Disease Space Highlight Confusion Around External Controls

Cassidy Names Areas of Improvement for FDA’s Regulatory ‘Black Box’