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Gene therapy approved for Duchenne muscular dystrophy
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
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Gene therapy
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Could novel gene therapy treat sickle cell disease?
Researchers have revealed positive preliminary data from a CRISPR gene therapy trial for sickle cell disease.
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Cell and Gene Therapy Catapult opens new laboratories
Cell and Gene Therapy Catapult's new laboratories in Scotland aims to help cell therapy developers improve their manufacturing processes and navigate regulatory requirements.
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CRISPR-based microbial gene therapy delivers promise
A microbial gene therapy can “eliminate antibiotic-resistant E. coli strains in the gut”, according to first-in-human study interim results.
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First redosable gene therapy approved
The “landmark approval” of a redosable gene therapy for dystrophic epidermolysis bullosa “ushers in a whole new paradigm to treat genetic diseases".
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Realising milestones with gene therapy for SMA
Novartis Gene Therapies' Vice President for Clinical Development & Analytics shares what shaped the success of one of the few commercially-available gene therapies.
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Press release: SGS wins business leadership award 2023
SGS’s biosafety centre of excellence in Glasgow has received the Business Leadership Award at Scotland's Life Sciences Annual Awards 2023.
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What gene therapy manufacturers can gain from collaboration
In this article, Neil Almstead, Chief Technical Operations Officer at PTC Therapeutics, shares his view on why collaboration is key to streamlining manufacture of gene therapies.
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Realising the potential of AAV gene therapies
In this article, Dr Rajiv Vaidya, Head of Manufacturing Science & Technology at Andelyn Biosciences, explores strategies for improving the scalability and cost‑effectiveness of AAV production while maintaining regulatory compliance.
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Bioprocessing/Bioproduction In-Depth Focus 2023
In this In-Depth Focus are articles on the potential of AAV gene therapies and what gene therapy manufacturers can gain from collaboration.
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European Pharmaceutical Review Issue 2 2023
EPR Issue 2 includes articles on the future of cell and gene therapy, the potential of AAV gene therapies, NMR relaxometry and more…
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First gene therapy recommended for AADC
NICE's final draft guidance has recommended the first and only gene therapy for children with aromatic L-amino acid decarboxylase (AADC) deficiency.
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Novartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
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GOSH and Leucid to manufacture novel CAR T therapy
Great Ormond Street Hospital and Leucid Bio will manufacture a novel lateral CAR targeting NKG2D ligands, under a first-of-its-kind manufacturing agreement.
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New NHS cell and gene therapy centre opens
The new Clinical Biotechnology Centre (CBC) in Bristol will manufacture cell and gene therapies to help boost the UK life sciences sector.
