In the last year, some drugmakers have pulled back from the sector, including Pfizer, which recently stopped selling its gene ...

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

This collection of health news showcases a range of significant developments: measles cases are surging in Texas and New ...

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

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

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

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

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

ECURE previously announced that the first patient dosed with ECUR-506 for OTC deficiency achieved a complete clinical ...

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

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: ...