New video explainer series - neurodevelopmental disorders and gene therapy
New gene therapy video series
The CoRE is proud to support the launch of Genes, Brains, and Breakthroughs, a new educational campaign designed to raise public awareness and understanding of neurodevelopmental disorders through a series of accessible, family-informed video resources.
YouTube: https://www.youtube.com/@MRCCoRETG
TikTok: https://www.tiktok.com/@genebrainbreakthrough
Instagram: https://www.instagram.com/genebrainbreakthrough/
Click through for more info!
Consultation on UK rare disease therapy regulation
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has opened its consultation on the Draft rare disease therapies regulatory framework. The CoRE will be sending in a response, and notes that patients and the general public are also invited to respond.
For more, see https://www.gov.uk/government/consultations/draft-rare-disease-therapies-regulatory-framework
Early Career Investigator Award for Emilie Wigdor
Congratulations to Emilie Wigdor on receiving the Early Career Investigator Award from the International Society for Autism Research at INSAR 2026. This award recognises her impactful work on the role of common variants in rare neurodevelopmental conditions.
MRC CoRE part of England Rare Diseases Action Plan 2026
MRC CoRE TG namechecked as part England's national strategy for rare diseases in 2026
Nicky Whiffin in Alternative Honours List
Nicky Whiffin was named in The Sunday Times Alternative Honours List of 2025, for her role in helping to discover the genetic basis of ReNU syndrome - a previously unexplained neurodevelopmental disorder.
Baby KJ in Nature Top 10
Kyle Muldoon Jr has been named in the 2025 Nature's 10 list, as the first recipient of personalised CRISPR therapy.
Tabrizi in Nature Top 10
Professor Sarah Tabrizi, UCL Site Lead for the MRC CoRE and member of the CoRE Brain Theme, has been named in Nature's 10 list.
Good news in blood cancer trial
Gene therapy has been used to treat a previously untreatable type of blood cancer, using base-editing technology developed at UCL and Great Ormond Street Hospital.
Child successfully treated for Hunter Syndrome
In a world first, a boy named Oliver Chu has been successfully treated for Hunter syndrome. The BBC website has an excellent explanation of the technology, which is similar to the approaches taken as part of the MRC CoRE Blood Theme projects.
University of Manchester website
Mouse study details produced by the team in Manchester
Tabrizi wins top prize from British Neuroscience Association
Professor Sarah Tabrizi has been awarded the British Neuroscience Association (BNA) Outstanding Contribution to Neuroscience Award for 2025, the association’s top annual prize.
Read more on the UCL website.
FDA's new Plausible Mechanism Pathway
The US FDA has published an initial "Sounding Board" piece in the New England Journal of Medicine, outlining a regulatory path to market entry for products where a randomized trial is not feasible.
"Once a manufacturer has demonstrated success with several consecutive patients with different bespoke therapies, the FDA will move toward granting marketing authorization for the product. Manufacturers will then be able to leverage platform data from such personalized products to gain marketing approval for similar products in additional conditions. Depending on the strength of evidence, either the accelerated or the regular pathway may be utilized."
Read more here; original NEJM article here.
MHRA signals change for rare disease treatment regulation
The UK Medicines and Healthcare products Regulatory Agency has announced plans to update regulation for genomic therapies.