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Cell therapy weekly: partnerships for advancing cell and gene therapies

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This week: Partnerships to advance cell and gene therapies have been announced, focusing on three key areas: developing gene therapies, enhancing cell and gene therapy manufacturing and developing treatments for difficult-to-treat conditions. The news highlights:Centers of excellence for gene therapy research Advancing cell and gene therapy manufacturing Developing therapies for difficult-to-treat conditions Centers of excellence for gene therapy research The Medical Research Centre (MRC), part of UK Research and Innovation (UKRI; both Swindon, UK) is establishing two Centers of Research Excellence (CoRE) to advance therapies for untreatable diseases, each receiving up to roughly US$64 million over 14 years aiming to transform treatments for diseases such as heart disease, immune disorders and neurodegenerative diseases like Huntington’s disease. Oxford University (UK) will lead the MRC/BHF CoRE in Advanced Cardiac Therapies, which will be co-funded with the British Heart Foundati...
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DBT, CSCR develop 1st human gene therapy for Haemophilia A in India

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Offering hope to around 136,000 Haemophilia A patients in India, the Department of Biotechnology (DBT), along with the Centre for Stem Cell Research (CSCR) at Christian Medical College (CMC), Vellore, announced that they have successfully executed a first-in-human gene therapy using lentiviral vectors for the disease. Haemophilia is a severe bleeding disorder caused by the deficiency of clotting Factor VIII, leading to spontaneous internal and external bleeding episodes. Although rare, India bears the world’s second-largest burden of haemophilia. Current treatments require frequent Factor VIII replacement therapy, which faces challenges such as high costs, intravenous (IV) access in children, and low patient acceptance.The single-centre study, which enrolled five participants aged between 22 and 41 years, showed transformational results, according to scientists involved in the research. “The therapy successfully produced zero annualised bleeding rates in all five enrolled participan...
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