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- PMLiVE

Sarepta Therapeutics

- PMLiVE

Duchenne UK and pharma partner pilot data-sharing HTA approach

Pfizer, Roche and PTC Therapeutics are among those participating in Project Hercules

- PMLiVE

Pfizer buys into Sangamo’s motor neuron disease gene therapy

The pharma giant will pay $12m upfront and $150m in potential milestones

- PMLiVE

NICE backs GSK’s €594k gene therapy for rare disease

The treatment offers 'bubble baby' patients an alternative to a stem cell transplant

- PMLiVE

Spark sets Luxturna price, igniting US affordability debate

The IRD treatment is set to cost patients $425,000 per eye

- PMLiVE

Spark’s Luxturna becomes US’s first approved gene therapy

FDA says its move 'reinforces the potential of this breakthrough'

- PMLiVE

Gene therapy

When will it deliver?

- PMLiVE

Shire’s gene therapy candidate receives orphan drug status in the US

Investigational factor VIII treatment SHP654 is being developed for haemophilia

- PMLiVE

Sarepta eyes FDA filing for DMD drug golodirsen after positive trial

The exon-skipping drug raised functional dystrophin levels

Will gene therapies finally be commercially viable to pharma?

Our latest Free Thinking white paper explores the science behind the idea of gene therapy and takes a look at some of the core challenges medical researchers have faced when...

Research Partnership

- PMLiVE

Spark closes on first US gene therapy approval

FDA starts review of Luxturna for patients with inherited retinal disease

- PMLiVE

Marathon pauses Emflaza launch amid pricing furore

US Senator Bernie Sanders calls Duchenne muscular dystrophy drug’s pricing “unconscionable”

- PMLiVE

FDA approves first steroid for Duchenne muscular dystrophy

Marathon Pharma’s Emflaza is first approved to treat all genetic forms of the condition

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