Eladocagene exuparvovec for the treatment of patients with aromatic L-amino acid decarboxylase deficiency

19 April 2023 - NICE has published evidence based recommendations on eladocagene exuparvovec (Upstaza) for the treatment of patients 18 ...

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ICER releases draft evidence report on gene therapies for sickle cell disease

12 April 2023 - Public comment period now open until 9 May 2023; Requests to make oral comment during public ...

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RegenxBio receives FDA fast track designation for RGX-202, a novel gene therapy candidate for the treatment of Duchenne muscular dystrophy

11 April 2023 - RGX-202 is a potential one-time AAV therapeutic for the treatment of Duchenne and includes an optimised transgene ...

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Vertex and CRISPR Therapeutics complete submission of rolling biologics license applications to the US FDA for exa-cel for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia

3 April 2023 - EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review ...

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NICE recommends life-changing gene therapy for children with ultra rare genetic disorder in final draft guidance

23 March 2023 - The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended ...

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Top FDA official: agency needs to start using accelerated approval for gene therapies

20 March 2023 - A top FDA official said Monday that the agency needs to start using accelerated approval, a ...

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ICER to assess gene therapy for metachromatic leukodystrophy

9 March 2023 - Report will be subject of CTAF meeting in September 2023; draft scoping document open to public ...

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Use warranties, not outcomes-based agreements, to make payers whole when a gene therapy fails

9 March 2023 - A decade ago, I had pegged gene and cell therapies as the next frontier. As an ...

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Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar syndrome, a rare liver disease

7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...

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BioMarin provides update on FDA review of Roctavian (valoctocogene roxaparvovec) gene therapy for adults with severe haemophilia A

7 March 2023 - FDA extends PDUFA target action date to 30 June 2023. ...

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Will reimbursement criteria of SMA treatment Spinraza change?

6 March 2023 - The discussion will likely begin in earnest this month to expand reimbursement criteria for Spinraza (nusinersen ...

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Alnylam announces US FDA acceptance of supplemental new drug application for Onpattro (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis

21 February 2023 - PDUFA date set for 8 October 2023. ...

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First gene therapy for haemophilia B, CSL's Hemgenix, approved by the European Commission

20 February 2023 - Hemgenix underscores CSL's promise to deliver life-changing innovations that have the potential to help patients lead ...

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UK’s most expensive drug Libmeldy saved Teddi Shaw, but is too late for her sister

16 February 2023 - A toddler with a rare inherited condition has become the first child to be treated by ...

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FDA gene therapy head raises concern on accelerated approval use

14 February 2023 - More than 1,000 cell and gene therapy clinical trials registered. ...

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