CSL Behring announces positive reimbursement decision in Canada for Hemgenix (etranacogene dezaparvovec), the first gene therapy for haemophilia B

16 July 2024 - The positive decision from CADTH recognises the importance of this one-time, single dose innovative treatment for ...

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Innorna announces US FDA rare paediatric disease designation granted to IN016 for the treatment of progressive familial intrahepatic cholestasis

11 July 2024 - Innorna today announced that the US FDA has granted rare paediatric disease designation to IN016, one of ...

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A new gene therapy, transformative cystic fibrosis medicines and an obesity jab included in guidance recommended during pre-election period

8 July 2024 - More than 20 pieces of draft and final guidance on a range of medicines and medical conditions ...

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CSL Behring announces first two patients treated with Hemgenix (etranacogene dezaparvovec) gene therapy for haemophilia B in Europe

4 July 2024 - CSL Behring today announced that two haemophilia B patients were treated with the gene therapy Hemgenix ...

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NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix

28 June 2024 - NICE has recommended CSL Behring’s haemophilia B gene therapy Hemgenix (etranacogene dezaparvovec) for immediate reimbursement by ...

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Rocket Pharmaceuticals provides regulatory update on Kresladi (marnetegragene autotemcel)

28 June 2024 - Rocket Pharmaceuticals today announced a regulatory update for Kresladi (marnetegragene autotemcel), a lentiviral vector-based gene therapy ...

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Top FDA official Peter Marks overruled staff, review team to approve Sarepta gene therapy

20 June 2024 - For a third time, Sarepta Therapeutics has convinced a top FDA official to overrule the prevailing ...

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FDA expands approval of gene therapy for patients with Duchenne muscular systrophy

20 June 2024 - Today, the FDA expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment ...

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SpliSense receives FDA fast track designation for SPL84 for the treatment of cystic fibrosis

29 May 2024 - SpliSense today announced that the US FDA has granted fast track designation to SPL84 for cystic ...

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Neurogene announces NGN-401 gene therapy for Rett syndrome selected by FDA for START pilot program

3 June 2024 - NGN-401 is one of only three CBER programs chosen by FDA. ...

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uniQure receives FDA regenerative medicine advanced therapy designation for investigational gene therapy AMT-130 in Huntington’s disease

3 June 2024 - Designation based on 24 month interim Phase 1/2 clinical data for AMT-130 announced in December 2023. ...

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Biogen receives European Commission approval for Qalsody (tofersen), the first therapy to treat a rare, genetic form of ALS

30 May 2024 - Qalsody is Biogen’s third rare disease therapy to be approved in the EU, demonstrating the company’s commitment ...

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New gene therapy treatment for haemophilia B

31 May 2024 - The EMA has recommended granting a conditional marketing authorisation in the European Union for Durveqtix (fidanacogene ...

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Highlights from the 27-30 May 2024 CHMP meeting

31 May 2024 - The EMA’s CHMP recommended 14 medicines for approval at its May 2024 meeting. ...

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Advancing access to cell and gene therapies in Medicaid

22 May 2024 - The Biden administration’s stated priority to increase access to novel cell and gene therapeutics while controlling their ...

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