gene therapy

News

Roche removes tenecteplase from phase 3 for stroke

Roche said it has removed its thrombolytic drug tenecteplase from phase 3 for for acute ischaemic stroke, along with several earlier-stage projects.

News

Sensorion files to start trials of deafness gene therapy

French biotech Sensorion has filed its first regulatory application to start human testing of OTOF-GT, a gene therapy for one of the most common forms of congenital deafne

News

BioMarin’s haemophilia A gene therapy gets US approval

The FDA has approved BioMarin Pharma’s Roctavian as the first gene therapy for haemophilia A, giving patients an alternative to regular injections of blood clotting factor

News

Big day for Duchenne as Sarepta gets first gene therapy nod

Almost against the odds, Sarepta’s gene therapy for muscle-wasting disease Duchenne muscular dystrophy (DMD) has been given accelerated approval by the FDA, albeit in a fa

News

uniQure craters on Huntington’s gene therapy data

uniQure has presented new results from its phase 1/2 study of its gene therapy for Huntington’s disease that

News

Beacon debut sheds light on Syncona’s gene therapy plans

UK life sciences investor Syncona is bankrolling a new gene therapy company, Beacon Therapeutics, that emerged this morning with a pipeline headed by a therapy acquired fr

Roche removes tenecteplase from phase 3 for stroke

Sensorion files to start trials of deafness gene therapy

BioMarin’s haemophilia A gene therapy gets US approval

Big day for Duchenne as Sarepta gets first gene therapy nod

uniQure craters on Huntington’s gene therapy data

Beacon debut sheds light on Syncona’s gene therapy plans

Lilly bags FDA okay for Wegovy pill rival orforglipron

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

gene therapy

Editor's Picks