Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft ...

A groundbreaking gene therapy has restored sight in four young children born with severe blindness due to a rare genetic ...

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic ...

YolTech's gene therapy is intended as a one-time life-long cure for patients living with PH1, which can lead to kidney ...

The global Gene Therapy market, valued at US$7.21 billion in 2023, is forecasted to grow at a robust CAGR of 19.4%, reaching ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

The Interministerial Commission for the Pricing of Medicines in Spain has backed access by the Spanish National Health System ...

The children were born with a severe impairment to their sight due to a rare genetic deficiency that affects the AIPL1 gene. The condition, a form of retinal dystrophy, means those affected are born ...

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic ...

Spinal muscular atrophy (SMA) is a debilitating genetic condition that’s usually fatal by a few years of age. But an ...