Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

Two years ago, he was diagnosed with a terminal illness called Batten disease -- often leading to blindness, seizures, and ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease.

Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only ...

A new FDA pathway could speed bespoke gene therapies, but key questions over scope and commercial viability remain.

Please refer to the ASH 2025 online program for full session details and visit the Genetix booth (#1261) onsite.

Itvisma, an intrathecal version of Zolgensma, can be used in children, teens and adults to stabilize or improve motor ...

The new intrathecal formulation means the one-shot gene therapy is now available to all SMA patients, regardless of their age ...

By adding a microRNA molecule to ALS models, Tel Aviv University, Ben-Gurion University and Weizmann Institute researchers say the damaged nerve cells 'even regenerated' ...

Novartis is pricing Itvisma at a wholesale acquisition cost of $2.59 million, a company spokesperson told Fierce Pharma. At ...

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the heart can pump and dramatically improves ...

Biotechs in the charter city of Próspera aim to cure aging, but experts question the feasibility of their goals.