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NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease
About 15,000 people in England are living with the inherited blood disorder
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
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
FDA approves Vertex/CRISPR’s Casgevy gene therapy for beta thalassaemia
The companies estimate that approximately 1,000 patients in the US will be eligible for the one-time treatment
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
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
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
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
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
bluebird bio to sell FDA priority review voucher for $103m
The company previously sold its second voucher to Bristol Myers Squibb for $95m
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
bluebird bio’s sickle cell gene therapy accepted for FDA priority review
The genetic disease affects approximately 100,000 people in the US