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- PMLiVE

Spark Therapeutics

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EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders

There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy

- 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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CHMP recommends gene therapy for beta thalassemia and sickle cell disease

If approved, Casgevy will be the only genetic therapy in the EU to treat these conditions

- 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

MHRA authorises world-first gene therapy for two inherited blood disorders

Casgevy is now the first licensed treatment that uses the gene-editing tool CRISPR

- 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

FDA completes meeting for potential genetic therapy to treat sickle cell disease

If approved, exa-cel will be the first genetic therapy available to treat patients with severe sickle cell disease

- 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

- PMLiVE

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

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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

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NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy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

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