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- PMLiVE

Chiesi Global Rare Diseases

- PMLiVE

Pfizer’s sickle cell disease therapy Oxbryta recommended by NICE for NHS use

About 4,000 people in England with the genetic blood disorder are expected to benefit from the decision

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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

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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

Sanofi and Scribe announce in vivo genetic therapy partnership worth over $1.2bn

The agreement expands the companies’ existing collaboration focused on ex vivo editing

- PMLiVE

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

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

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EMA recommends revoking marketing authorisation for Novartis’ sickle cell disease drug

The EU regulator concluded that the benefits of Adakveo did not outweigh its risks

- PMLiVE

NHS to introduce world-first genetic blood-matching test for transfusion treatments

NHS Blood and Transplant will receive almost £1m in funding from NHS England

- PMLiVE

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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