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

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

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

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

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

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

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

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

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

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

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

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