Copyright: © 2025 Published by Elsevier Ltd. The roll-out of gene therapy has not been without difficulties, mostly related to side-effects and complex technology ...

Pfizer’s decision to halt further marketing of Beqvez is further sign of the sparse patient interest in gene therapies for the bleeding condition. The pioneering gene therapy developer will be ...

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of ...

The company made its debut last year with a $51 million Series A and a licensing agreement with Astellas for a gene therapy candidate (KT430) for X-linked myotubular myopathy. Shortly after that ...

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 ...

Medics in the UK have become the first in the world to successfully administer a pioneering gene therapy that preserves sight in young children with an extremely rare genetic condition that usually ...

Children with a rare form of eye disorder who were born blind can now see thanks to a "remarkable" gene therapy breakthrough.

The outcomes of the new treatment, reported in The Lancet, show that gene therapy at an early age can dramatically improve sight for children with this condition – one that is rare and ...

Jundishapur University of Medical Sciences and the University of Meisan have signed a memorandum of understanding (MOU) to ...

CRISPR epigenome editing successfully reactivates silenced genes in Prader-Willi Syndrome, highlighting potential for ...

New research shows people in specific Polynesian communities have a much higher rate of a form of Parkinson’s disease considered rare elsewhere.

It’s like a weather map, charting shifts in the atmosphere ... Mitalipov, director of the university’s Center for Embryonic Cell and Gene Therapy, had no intention of carrying the experiment ...