FDA defends its demand for new trial for rare brain disease therapy

A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new ...
Fierce Biotech

UniQure shares crash 40% as FDA rejects early approval path for Huntington's gene therapy

Shares of uniQure plummeted more than 40% in premarket trading Monday after the company confirmed the FDA has rejected its current data package for AMT-130, a highly anticipated gene therapy for ...
Scientific American

Pioneering gene therapy may treat a deadly seizure disorder

A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...
FierceBiotech

Ocugen posts midphase gene therapy data, charting course for rivalry with Apellis, Astellas

Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may outperform existing treatments from Apellis Pharmaceuticals and Astellas ...
Forbes

Gene Editing Or Gene Silencing: The Therapies Poised To Transform Medicine

Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...