CSL Behring announces the first patient has received FDA approved Hemgenix (etranacogene dezaparvovec-drlb) for haemophilia B

20 June 2023 - Hemgenix, the first and only FDA approved gene therapy for haemophilia B, has been proven to ...

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FDA should reject accelerated approval of gene therapy SRP-9001

15 June 2023 - The US FDA should reject the accelerated approval of the gene therapy SRP-9001 (Sarepta Therapeutics) for ...

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FDA accepts biologics license applications for exagamglogene autotemcel (exa-cel) for severe sickle cell disease and transfusion-dependent beta thalassaemia

8 June 2023 - FDA grants priority review for severe sickle cell disease and standard review for transfusion-dependent beta thalassemia. ...

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Rocket Pharmaceuticals receives FDA fast track and orphan drug designations for RP-A601 gene therapy for PKP2 arrhythmogenic cardiomyopathy

8 June 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted fast track and orphan drug designations ...

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Arcturus Therapeutics receives US FDA fast track designation for ARCT-810, mRNA therapeutic candidate for ornithine transcarbamylase deficiency

1 June 2023 - Arcturus Therapeutics today announced that the US FDA has granted fast track designation to ARCT-810, the Company’s ...

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Sarepta Therapeutics announces update on regulatory review of SRP-9001

24 May 2023 - New regulatory action date is 22 June 2023. ...

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RegenxBio receives FDA regenerative medicine advanced therapy designation for RGX-121 gene therapy for Hunter syndrome

23 May 2023 - CAMPSIITE trial is enrolling mucopolysaccharidosis type II patients as part of a pivotal program that incorporates ...

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Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-L301 gene therapy for pyruvate kinase deficiency

23 May 2023 - Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...

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Sangamo Therapeutics receives US FDA fast track designation for isaralgagene civaparvovec for the treatment of Fabry disease

22 May 2023 - Sangamo Therapeutics today announced that the US FDA has granted fast track designation to isaralgagene civaparvovec, or ...

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FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa

19 May 2023 - Today, the US FDA approved Vyjuvek, a herpes-simplex virus type 1 vector-based gene therapy, for the treatment ...

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FDA grants SiSaf’s innovative siRNA therapy SIS-101-ADO orphan drug designation and rare paediatric disease designation for the treatment of autosomal dominant osteopetrosis

15 May 2023 - SiSaf announces that SIS-101-ADO, its siRNA therapeutic, has been granted orphan drug designation and rare paediatric disease ...

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FDA grants accelerated approval for Qalsody (tofersen) for SOD1-ALS, a major scientific advancement as the first treatment to target a genetic cause of ALS

25 April 2023 - FDA granted accelerated approval of Qalsody based on a reduction of neurofilament, a marker of neurodegeneration. ...

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bluebird bio submits biologics license application to FDA for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease 12 years and older with a history of vaso-occlusive events

24 April 2023 - BLA submission based on data from the largest and most mature clinical development program for any ...

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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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