More than two years after its approval in Europe, PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US.
Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric
Lexeo Therapeutics' gene therapy for Alzheimer's has shown that it can reduce biomarkers of the disease in a small study, including reductions in disease biomarkers.
Astellas has added another gene therapy to its pipeline, taking an option on a clinical-stage candidate for a rare genetic form of dementia from UK biotech AviadoBio for around $50 million.
BioMarin Pharma has decided not to divest its haemophilia A gene therapy Roctavian, which has struggled to build much traction in the market, but is reining back its ambitions for the busin