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UK ATMP facility granted cGMP approval
A UK CDMO is now permitted through MHRA certification to produce clinical supply of gene therapy AAV, including bioprocess through to cGMP manufacturing.
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Gene therapy
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Stem cell therapy delivers potential for sickle cell disease
A Novartis’ Phase I/II study for sickle cell disease trialling the gene therapy OTQ92 is the first treatment to target a new genetic area and use cryopreserved stem cells, according to new paper published in NEMJ.
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Finnish manufacturing facility to get €50m expansion
The planned €50 million investment at the Finland-based CDMO facility will enable advanced technologies to be used for development and manufacture of advanced therapy medicinal products (ATMPs).
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The evolution of AAVs in cell and gene therapy
AAV development for cell and gene therapy in 2023 is being impacted by manufacturing and regulation challenges, however advancing technologies offer opportunity, according to leaders in the field.
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First gene therapy approved for Haemophilia A
The first approved gene therapy for haemophilia A reduced the mean annualised bleeding rate from 5.4 bleeds to 2.6 bleeds per year in a clinical study.
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Reducing bioburden testing and sterility testing volumes for gene therapies
A reduced sampling plan was recommended in a Biophorum report for reducing required bioburden and sterility testing volumes for gene therapy batch yield.
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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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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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The cell and gene therapy CDMO bottleneck isn’t capacity – it’s capability
CDMOs have been squeezed by the sharp increase in companies preparing to scale their cell and gene therapy manufacturing for Phase III trials and commercialisation, and physical capacity has been a common scapegoat. But eXmoor CEO Angela Osborne says the real limiting factor is experience.
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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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New UK point of care manufacturing regulatory framework
A first-of-its-kind regulatory framework for point of care manufacturing of innovative medicines is set to be introduced in the UK, ensuring these products are as safe as conventional medicines.
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Breakthrough gene therapy gives hope for Artemis-SCID
A new gene therapy developed by UC San Francisco has enabled ten young Artemis-SCID patients to achieve full T-cell immunity.
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FDA approves first gene therapy for high-risk early bladder cancer
The first gene therapy for high-risk non-muscle-invasive bladder cancer has been approved by The US Food and Drug Administration (FDA).
