Reverse Rett

Making Rett History

In 2017, bio tech company AveXis have committed to advancing Gene Therapy for Rett into human clinical trials. Our focus is now to support the continued work of the GT Consortium as they work full speed ahead on improved second-generation GT techniques.

history Campaign has now closed

It ran from 12:00 PM, 28 November 2017 to 12:00 PM, 5 December 2017

open_in_new http://www.reverserett.org.uk

Registered Charity in England and Wales (1136809)

Check mark Match funded

Campaign target

£50,000

Amount raised

£85,205

Donations

159

    Category

    Helping

Location

  • When we started this consortium two years ago we knew that we faced significant challenges to developing a viable gene therapy approach for Rett Syndrome. Many of our initial approaches proved unsuitable for human application, but newer treatment designs appear to be overcoming those initial proble

    — Steven Gray

  • We‚Äôve learned that a single one-time administration of a gene therapeutic can have a clinically meaningful result in the workhorse rodent model of this disease, even when delivered later in life.

    — Brian Kaspar

  • While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett it is conceivable that gene therapy can correct the cause of Rett at its very source and thus provide a profound recovery of function.

    — Stuart Cobb

  • When we started this consortium two years ago we knew that we faced significant challenges to developing a viable gene therapy approach for Rett Syndrome. Many of our initial approaches proved unsuitable for human application, but newer treatment designs appear to be overcoming those initial proble

    — Steven Gray

  • We’ve learned that a single one-time administration of a gene therapeutic can have a clinically meaningful result in the workhorse rodent model of this disease, even when delivered later in life.

    — Brian Kaspar

  • While traditional drug approaches will likely be restricted to correcting specific aspects of what goes wrong in Rett it is conceivable that gene therapy can correct the cause of Rett at its very source and thus provide a profound recovery of function.

    — Stuart Cobb

Situation

Rett Syndrome strikes at random in early childhood, affecting little girls almost exclusively. Most girls lose the ability to walk, talk and use their hands. Most will have difficulty breathing and eating. All are at increased risk of sudden and unexplained death. Many girls live into adulthood, requiring total, 24-hour-a-day care. There is currently no effective treatment but the disease has already been reversed in a mouse model.

Solution

The concept behind gene therapy (GT) is simple: delivery healthy copies of the MECP2 gene to compensate for the mutated ones. Recent results from RSRT’s GT Consortium have exceeded expectations, suggesting that significant benefit may be achieved in people. In 2017, biotech company, AveXis, has committed to advancing a GT candidate into human trials. Our focus is now to support the continued work of the GT Consortium as they work full speed ahead on improved second-generation GT programs.