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An Unexpected Finding

In 1992, Sir Adrian Bird discovered a new protein and named it MECP2. Seven years later, Professor Huda Zoghbi discovered that mutations in MECP2 cause Rett. In 2007, Professor Bird shocked the scientific and Rett communities by showing that Rett symptoms in mice models disappeared upon restoration of MECP2.

This finding changed everything as it suggested a cure was possible. Monica Coenraads launched the Rett Syndrome Research Trust in 2008 to focus exclusively on cures. Adrian Bird joined Monica as a founding trustee, a position he holds to this day.

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Gene Therapy Consortium

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Since the moment RSRT was created, we’ve been optimistic about curing Rett. Fueling our optimism are three key facts: a single gene means we have a clear target, Rett brain cells don’t die, and restoring levels of MECP2 reverses symptoms in mice models.

When we began there was only one option to boost levels of MECP2: gene therapy. Many believed gene therapy for the brain was science fiction. We recognized that it was the wave of the future. Determined to make it a priority, we provided generous funding to drive this approach forward. In 2013, an RSRT-funded study by Gail Mandel, Brian Kaspar, and Adrian Bird showed that gene therapy had reversed symptoms in female Rett mice.

Spurred by the belief that labs with synergistic but varied skill sets would accelerate progress, we conceived the Gene Therapy Consortium in 2014. After scouring the gene therapy landscape we invited gene therapists Brian Kaspar and Steve Gray, and MECP2 experts Stuart Cobb and Gail Mandel to join the Consortium.

The Consortium is one of the most valuable investments we’ve made as it formed the foundation for the Rett programs at Neurogene, Taysha, and Vico.

Gene therapy is one approach to target Rett but it’s not the only approach. We believe the greatest likelihood of achieving cures is to take as many shots on goal as possible.

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Roadmap to a Cure 1.0

In 2017 we launched a three-year, $33 million research plan, with the goal of identifying and advancing all possible genetic medicine avenues that address the root cause of Rett.

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Yearly Awards

A list of our research awards from 2008 to 2023.

Publications

A list of scientific publications made possible through our funding.

Our Collaborators

A History of Rett & RSRT

1966-andreas-rett

1966

1st article published on Rett in German medical journal

1983-bengt-hagberg

1983

2nd article on Rett published in high-profile neurology journal

1992-adrian-bird

1992

MECP2 protein discovered by Adrian Bird

1999-huda-zogbhi

1999

MECP2 mutations identified by Huda Zogbhi as cause of Rett

2007-adrian-bird

2007

Adrian Bird discovers Rett symptoms in mice are reversible

2008-rsrt-launches

2008

Rett Syndrome Research Trust launches

2011-mecp2-consortium

2011

MECP2 Consortium launches

2013-rett-mice

2013

Gail Mandel and Brian Kaspar publish gene therapy results

2014-gene-therapy-consortium

2014

RSRT Gene Therapy Consortium launches

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2017

Roadmap to a Cure launches

2019-beam

2019

Beam joins DNA editing effort

2020-cure-360

2020

CURE 360 launches

2020-taysha

2020

Taysha Gene Therapies announces gene therapy program

2020-vico-therapeutics

2020

Vico Therapeutics announces RNA editing program

2020-shape-therapeutics

2020

Shape Therapeutics announces RNA editing program

2021-alcyone-therapeutics

2021

Alcyone Therapeutics announces lead MECP2 reactivation program

2021-herophilus

2021

Herophilus announces lead MECP2 reactivation program

2020-taysha

2022

Taysha Gene Therapies announces first gene replacement trial

2022-neurogene

2022

Neurogene announces gene replacement program

2020-taysha

2023

Taysha Gene Therapies doses patient #1 in gene replacement trial

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2023

Neurogene doses first two patients

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2023

Rett Syndrome Global Registry launches

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2024

MECP2 Editing Consortium created

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2024

Roadmap to Cures 2.0 announced

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Roadmap to Cures 2.0

Built on decades of scientific advances Roadmap to Cures will select and drive three genetic medicines that attack the root cause of Rett syndrome to clinical trials by 2028.

$40M