gene therapies

News

Multiple studies of Sarepta's DMD therapy halted after death

Three studies of Sarepta's Elevidys Duchenne gene therapy have been placed on hold as the death of a 16-year-old boy is investigated.

News

Trial of Boehringer's cystic fibrosis gene therapy imminent

Boehringer's cystic fibrosis gene therapy is poised to start clinical testing, hoping to offer a one-shot alternative to costly CFTR modulator drugs.

News

New data could widen use of Novartis' SMA therapy Zolgensma

Phase 3 results with an intrathecal version of Novartis spinal muscular atrophy (SMA) gene therapy could make older children eligible for treatment.

R&D

From promise to reality: Unlocking the true business potenti...

The genomic medicine space has seen incredible success, but investors are overlooking it.

R&D

science laboratory research and development

The role of quality assurance in accelerating drug developme...

The pharmaceutical landscape is experiencing a paradigm shift with the rise of gene therapies, cell therapies, and personalised medicine.

News

uniQure wields axe again as Huntington's therapy progresses

uniQure is shrinking again, saying it will shed two-thirds of its staff by the end of the year – about 300 jobs – as it tries to trim $75 million off its annual cash burn.

Multiple studies of Sarepta's DMD therapy halted after death

Trial of Boehringer's cystic fibrosis gene therapy imminent

New data could widen use of Novartis' SMA therapy Zolgensma

From promise to reality: Unlocking the true business potenti...

The role of quality assurance in accelerating drug developme...

uniQure wields axe again as Huntington's therapy progresses

LSX2026: On promise and patience, with Hans Schambye

Using AI-driven synthetic personas to take your insights fur...

gene therapies

Editor's Picks