genetic diseases

News

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

R&D

Michelle Werner, Director of Alltrna, discusses her company’s approach of leveraging transfer RNA (tRNA) to shift the paradigm in genetic medicine.

News

Investor panic has poleaxed Quince Therapeutics share price, after a regulatory filing revealed the biotech's cash is running out.

News

The FDA said it needs more information before it can approve Regenxbio's gene therapy for Hunter syndrome, in a devastating decision for patients.

News

Servier has pushed its emerging neurology pipeline into the clinical trials stage by licensing a drug for Fragile X syndrome from the UK's Kaerus.

News

PTC Therapeutics' treatment for the rare disease PKU, Sephience, will launch in its first market next month after getting EU approval.