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

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

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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NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy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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FDA grants accelerated approval to Sarepta’s Duchenne muscular dystrophy gene therapy

The disease occurs in up to one in every 5,000 newborn males worldwide

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Decision date for Sarepta’s Duchenne muscular dystrophy gene therapy delayed by FDA

The disease occurs in up to one in every 5,000 newborn males worldwide

- PMLiVE

FDA advisory committee backs Sarepta’s Duchenne muscular dystrophy gene therapy

The disease occurs in up to one in every 5,000 newborn males worldwide

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bluebird bio submits US application for sickle cell gene therapy

The genetic disease affects approximately 100,000 people in the US alone

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EC approves first gene therapy for adults with haemophilia B

Typically, haemophilia B patients must adhere to strict, lifelong IV infusion schedules

- PMLiVE

Pfizer announces positive phase 3 results for haemophilia B gene therapy

A one-off, intravenous dose of the therapy candidate reduced bleeding by 71%

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