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Rett Syndrome Research Trust Launches $40M Fundraising Effort to Fund New Genetic Medicines

NEW YORK – The Rett Syndrome Research Trust on Wednesday launched a new initiative to develop three genetic medicines for Rett syndrome.

As part of the new initiative, dubbed Roadmap to Cures, the patient advocacy group is aiming to raise $40 million over the next four years, which it will put toward selecting and developing three disease-modifying therapeutic candidates. The Rett Syndrome Research Trust's MECP2 Editing Consortium, a collaboration between six laboratories, will play a role in this work, as the consortium seeks to develop DNA- and RNA-editing therapies for Rett syndrome.

Rett syndrome is a rare neurodevelopmental disorder primarily caused by mutations in the MECP2 gene and mostly occurs in girls.

"Time is of the essence, and the greatest likelihood of achieving cures in Rett is to advance, in parallel, as many shots on goal as possible," Monica Coenraads, the organization's CEO and a parent of a patient with Rett syndrome, said in a statement. "That is the goal of Roadmap to Cures."

The Rett Syndrome Research Trust aims to advance the three candidates into clinical trials by 2028.