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- PMLiVE

Restructuring

- 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

- PMLiVE

FDA approves two cell-based gene therapies for sickle cell disease

Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

- PMLiVE

bluebird bio presents positive results for inherited blood disorder gene therapies

The company’s sickle cell disease therapy was accepted for FDA priority review earlier this year

- PMLiVE

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

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

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bluebird bio to sell FDA priority review voucher for $103m

The company previously sold its second voucher to Bristol Myers Squibb for $95m

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

regeneron headquarters

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

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

- PMLiVE

NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy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

- PMLiVE

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

- PMLiVE

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

- PMLiVE

bluebird bio’s sickle cell gene therapy accepted for FDA priority review

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

- PMLiVE

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

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