Sir Professor Adrian Bird shows Rett Syndrome can be reversed in mice, a huge moment that paves the way for Rett Syndrome gene therapy research in humans
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Reverse Rett was founded in 2010 by a small group of UK parents who wanted to accelerate treatments for Rett syndrome.
Since then, we’ve raised over £10 million, investing in high-quality research projects such as the RSRT Gene Therapy and MECP2 Consortia, as well as in the work of individual labs at the University of Edinburgh. Progress in these research programs has led to the gene therapy programs in clinical trials today.
Now that potential treatments for Rett syndrome are on the horizon, there is even more for our small team to do.
We run the UK’s patient registry for Rett syndrome-essential to delivering clinical trials, campaigning for treatments to be approved and for managing delivery.
We work with clinicians to develop their Rett syndrome provision, ready for delivery.
We provide families with health information and resources and help connect them to UK Rett syndrome clinics.
We host an annual educational conference for families, caregivers, researchers and health care professionals.
We provide online workshops and up-to-date information via our website, social media platforms and one to one contact with families.
We advocate for treatment for Rett syndrome nationally and internationally.
Two clinical trials of gene therapy are now in pivotal/registrational gene therapy trials.
We need your help to continue our work.
Get in touch, we’d love to hear from you. mailto: [email protected]
Sir Professor Adrian Bird shows Rett Syndrome can be reversed in mice, a huge moment that paves the way for Rett Syndrome gene therapy research in humans
Reverse Rett is founded by five parents from the home of now CEO, Rachael Stevenson
Reverse Rett support the launch of RSRT’s international MECP2 Consortium (Bird, Greenberg and Mandel Labs) to combine global efforts to find a tangible way to reverse Rett Syndrome in humans.
Reverse Rett support RSRT launch of Gene Therapy Consortium (Cobb and Gray Labs) to compound efforts to drive Gene Therapy for Rett Syndrome into human trials.
Gene therapy shown to reverse the symptoms of Rett Syndrome in mice at the lab of MECP2 Consortium member, Gail Mandel.
Reverse Rett start Rett Registry UK in anticipation of human clinical trials of potential treatments
Reverse Rett begin seed-funding Rett-focused clinical team at King’s College London under Professor Santosh.
UK’s first Rett syndrome clinical trial starts at King’s College Hospital under Professor Santosh with Clinical Trial Co-ordinator funding and logistical support from Reverse Rett
2nd UK clinical trial for a potential treatment for Rett Syndrome launched at Alder Hey Children’s Hospital with recruitment and retention support by Reverse Rett.
Reverse Rett launches Rett Syndrome clinical treatment and research centre, the CIPP Rett Centre at King’s College Hospital.
3rd UK clinical trial for Rett Syndrome is launched at trial sites in Manchester and London with recruitment and retention support from Reverse Rett.
Neurogene announce Rett gene therapy program, NGN-401, based on the work of Cobb Lab.
4th UK clinical trial (Anavex 2-73 paediatric) starts at five trial sites: CIPP Rett Centre, Evelina Children’s Hospital, Manchester Rare Conditions Centre, Nottingham Children’s Hospital, Royal Hospital for Children, Edinburgh with recruitment and retention support from Reverse Rett.
Taysha Gene Therapies receives Health Canada approval for clinical trial in Rett gene therapy, TSHA-102 in Canada
Neurogene receives FDA approval for clinical trial in Rett gene therapy, NGN-401 in the USA
Taysha Gene Therapies announce that the first adult patient has been dosed with their gene therapy product, TSHA-102
UK regulator, MHRA approves UK Rett syndrome gene therapy clinical trial of NGN-401
UK regulator, MHRA approves UK Rett syndrome gene therapy clinical trial of TSHA-102
Taysha Gene Therapies announce that dosing is complete in phase 1/2 trial and that the FDA are aligned on pivotal trial plans due to start in Q3 2025
Neurogene announce that dosing is complete for 10 of 11 patients in phase 1/2 trial and pivotal trial plans are due to be announced imminently
Our UK clinical and research projects are subject to a rigorous review process set out by our membership of the Association of Medical Research Charities.
International research is funded primarily through our partnership with the US based Rett Syndrome Research Trust who provide peer review and monitoring for those projects.
Reverse Rett is a full member of the Association of Medical Research Charities (AMRC), a network of 150 medical and health research charities who deliver high-quality research that saves and improves lives. Membership of AMRC has to be earned through adherence to stringent peer review criteria.
I was first introduced to Rett Syndrome when I received an out of the blue phone call from Adrian Bird, who first discovered MeCP2 protein, requesting some help with a set of specific experiments. Following this successful collaboration, Rett Syndrome research has become a primary focus of my lab. While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett Syndrome, it is conceivable that Gene Therapy can correct the cause of Rett Syndrome at it’s very source and thus provide a profound recovery of function.
