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Sarepta

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FDA approves Italfarmaco’s Duvyzat as first nonsteroidal Duchenne muscular dystrophy drug

The neuromuscular genetic disease is estimated to affect one in every 3,500 to 6,000 male births worldwide

- PMLiVE

Santhera’s Duchenne muscular dystrophy drug shows promise as long-term treatment

The muscle-wasting disorder is estimated to affect one in every 3,500 male births worldwide

- PMLiVE

FDA publishes final industry guidance for CAR-T and gene therapies

The new guidance can also be applied to other genetically modified lymphocyte products, including  CAR NK cells

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MHRA approves Santhera’s Duchenne muscular dystrophy drug Agamree

The rare muscle-wasting disorder is estimated to affect one in 3,500 male births worldwide

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EC approves Santhera’s Duchenne muscular dystrophy drug Agamree

The rare muscle-wasting disorder is estimated to affect one in 3,500 male births worldwide

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AstraZeneca and Cellectis enter gene therapy partnership worth over $2.2bn

The deal gives AZ access to Cellectis’ gene editing technologies and manufacturing capabilities

- PMLiVE

FDA approves Santhera’s Duchenne muscular dystrophy drug Agamree

The rare muscle-wasting disorder is estimated to affect one in 3,500 male births worldwide

Biomarin

BioMarin announces first patient dosed with haemophilia A gene therapy in Europe

The therapy was granted conditional marketing authorisation by the EC in August 2022

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Regeneron to acquire Decibel Therapeutics in deal worth up to $213m

The transaction expands the company’s gene therapy and hearing loss pipeline

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EMA endorses digital measure as primary endpoint in Duchenne muscular dystrophy trials

The decision qualifies SV95C as an alternative to the widely used six-minute walking test

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NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy

The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors

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FDA approves BioMarin’s Roctavian as first gene therapy for severe haemophilia A

Approximately 2,500 patients are expected to be eligible to receive the one-time therapy

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