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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.
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).
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.
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.
A reduced sampling plan was recommended in a Biophorum report for reducing required bioburden and sterility testing volumes for gene therapy batch yield.
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
Researchers have revealed positive preliminary data from a CRISPR gene therapy trial for sickle cell disease.
Cell and Gene Therapy Catapult's new laboratories in Scotland aims to help cell therapy developers improve their manufacturing processes and navigate regulatory requirements.
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.
A microbial gene therapy can “eliminate antibiotic-resistant E. coli strains in the gut”, according to first-in-human study interim results.
The “landmark approval” of a redosable gene therapy for dystrophic epidermolysis bullosa “ushers in a whole new paradigm to treat genetic diseases".
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.
A new gene therapy developed by UC San Francisco has enabled ten young Artemis-SCID patients to achieve full T-cell immunity.
The first gene therapy for high-risk non-muscle-invasive bladder cancer has been approved by The US Food and Drug Administration (FDA).
What’s new in the cell and gene therapy landscape? What is the role of quality control in efforts to scale up manufacturing of CGT products and alleviate talent constraints? Find out more in this podcast with bioMérieux…
