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

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

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

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.

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

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

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

Regeneron Pharmaceuticals has teamed up with Tessera Therapeutics to develop and commercialise Tessera’s rare disease in vivo ...

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

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' ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...