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 ...

Sarepta Therapeutics said Tuesday that a 16-year-old boy died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy, the first known death linked to the treatment. The ...

21-year-old Sebastien Beauzile from Laurelton, Long Island, has become the first person in New York State to be cured of sickle cell anemia using the innovative Lyfgenia gene therapy. This ...

Pfizer is pulling further away from the gene therapy field with its decision to discontinue hemophilia product Beqvez. The New York pharma is ending global development and commercialization of ...

Roche has recently launched a “fundamental reorganization” of Spark Therapeutics, the gene therapy unit the Swiss pharma bought for $4.3 billion in 2019. Roche described the restructuring in ...

An indigenously developed gene therapy for specific blood cancers has shown a 73 per cent response rate among patients in India, according to results of clinical trials published in The Lancet ...

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 ...

Scientists have created a new gene therapy for a debilitating genetic disorder called maple syrup urine disease (MSUD). The treatment can prevent recurrence of deadly symptoms in a cow calf born ...

Regeneron’s investigational gene therapy has been tied to notable hearing improvements in 10 of 11 children who were treated for a rare genetic condition that causes hearing loss. “You can see ...

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene therapy, data that are prompting the biotech to seek accelerated approval ...

The University of Bristol research team used animal models to compare patient responses to AAV therapy, studying male and female eye cells across different age groups (young, middle-aged and older ...