By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...

A baby boy with a rare illness is making “incredible” progress after he became the first ever patient to receive a new gene ...

Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...

Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...

By replacing the defective gene associated with Dravet syndrome in mice, scientists successfully alleviated symptoms without ...

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...

OXB honoured at 2025 CDMO Leadership Awards in 'Cell & Gene Therapy – Global' category Oxford, UK – 21 March 2025: OXB (LSE: ...