Project information

NLX-101: Change is Possible

NLX-101 is a new drug being developed to treat breathing abnormalities in people with Rett Syndrome. It's possible that beyond breathing, the drug may also improve other core symptoms such as anxiety and movement disorders.

January 2016 - January 2017

Charity information: Reverse Rett

Reverse Rett logo
  • Need

    Need

    Many people with Rett Syndrome struggle daily with breathing irregularities, anxiety and movement disorders. There is currently no established effective treatment for any of these problems.

    Solution

    NLX-101 is a new drug under development by the pharmaceutical company, Neurolixis.

    NLX-101 has already been shown to normalise the irregular breathing patterns in Rett mice and has been given Orphan Drug status both in Europe and the US.

    Through this project, Reverse Rett will help Neurolixis, bring the new drug to the market, by funding the manufacturing and characterisation of clinical supplies of NLX-101 and supporting preparation for submission to the regulatory authorities.

  • Aims

    Aim 1

    Funding Neurolixis to develop NLX-101 to treat breathing abnormalities in people with Rett Syndrome.

    Activities

    » Deliver funds raised through this project to Neurolixis

    Every quarter we publish information which shows which research projects we have allocated funds to via website, email newsletter and other relevant media.


    Aim 2

    For Neurolixis to have submitted the Investigational New Drug application to the FDA within one year

    Activities

    » Deliver funds raised through this project to Neurolixis.

    As soon as Neurolixis have been able to submit the IND application we will report to our supporters via website, email newsletter and other relevant media.


  • Impact

    Impact

    NLX-101 could be one of the first treatments to make a significant impact on the daily lives of girls and women with Rett Syndrome.

    Long term success will be defined when NLX-101 is able to be tested in people with Rett Syndrome and if it is demonstrated to be effective in treating breathing irregularities and other core symptoms of Rett.

    For many girls and women with Rett, who struggle to breathe properly every waking minute of their day, an effective treatment would be life-changing.

    Risk

    The focus of this project is about 'de-risking' the development of a new drug to treat a devastating disorder to make the drug more attractive to investors who will then support the next stage of development and expedite the process of bringing the drug to market.



    Reporting

    Donors to this project will receive regular updates on the progress of this and other Reverse Rett supported work, via our website, email newsletter and social media.

  • Budget

    Budget - Project Cost: £140,000

    Loading graph....
      Amount Heading Description
      £140,000 NLX-101: Change is Possible Funding to Neurolixis to help move NLX-101 from research to reality

    Current Funding / Pledges

    Source Amount
    Mr & Mrs John and Maureen Sharpe £35,000 Guaranteed
  • Background

    Location

    NLX-101 has already obtained Orphan Drug status in the US and Europe.

    Neurolixis, the company who are working to develop the drug, is a small biotech company based in Southern California.

    Beneficiaries

    People with Rett Syndrome and related MECP2 disorders and their families, friends and communities will benefit from treatment for breathing irregularities and other core symptoms of Rett Syndrome.

  • Why Us?

    Why Us?

    Reverse Rett is the only UK charity working exclusively to speed treatments and a cure for Rett Syndrome and related MECP2 disorders.

    We fund more Rett Syndrome research than any other UK charity having delivered over £3 million to international research in the last five years.

    Read more about the Charity running this project.

    People

    Rachael Bloom

    Rachael is Executive Director of Reverse Rett. She has successfully executed 5 Big Give campaigns with a total of £269,617.24 raised!

    Andy Stevenson

    Andy is our Fundraising and Communications Manager. He will work with donors and run social media/website communications around the challenge.

    Thomas Johnston

    Thomas will be first poc with donors on the day and will run email newsletter campaigns in advance and during the challenge.

    Beth Johnsson

    Beth will work with supporters in the field to promote the challenge in the months leading up to December and during challenge week.

Amber aged 19

Amber aged 19