Jace’s mum DJ, 35, told the PA news agency: “Pre-surgery, at around two years old, you could have held up any object, even a ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Less than a year after approval, Pfizer said it plans to discontinue its hemophilia B gene therapy fidanacogene elaparvovec ...

Medics in the UK have become the first in the world to successfully administer a pioneering gene therapy that preserves sight ...

Christmas has come a few days early for Spark Therapeutics after the FDA approved its ground-breaking gene therapy for a rare eye disease. It looked likely that the FDA would approve the US ...

Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...

This achievement follows Bahrain’s landmark decision on 2 December 2023 to become the second country globally and the first ...

Understand the critical steps involved in translating laboratory research into clinical-grade cell and gene therapies. Explore how innovations in cell and gene therapy are paving the way for more ...

A second gene therapy candidate that spurs antigen-specific ... following myocardial infarction was deemed due to a previous history of coronary artery disease and severe aortic stenosis rather ...

The firm is seeking accelerated approval for UX111 using reductions in levels of heparan sulfate in cerebrospinal fluid as a surrogate endpoint.

Pharmaceutical announced new data demonstrating treatment with UX111 AAV gene therapy led to a statistically significant ...