gene therapies

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.

R&D

Opportunity costs of gene therapies. Where do we go from her...

Over 30 gene therapies have received regulatory approval.

News

Future of Pfizer’s Duchenne gene therapy in doubt

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial.

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

Opportunity costs of gene therapies. Where do we go from her...

Future of Pfizer’s Duchenne gene therapy in doubt

Sun Pharma unit poised to buy Canadian biotech Antibe

Health Trends 2025: Reimagining What's Possible

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gene therapies

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