Dear GP2C & Rett University Communities,

Keeping you informed! Here are some recent articles regarding work funded by our friends at Rett Syndrome Research Trust, and other companies investigating Rett-related research:


In Editing RNA, Researchers See Endless Possibilities

“…(Gail) Mandel sees so much potential in RNA editing that she cofounded VICO Therapeutics, a biotech startup centered around the technology. In 2018, the Rett Syndrome Research Trust awarded nearly $6 million in grants to researchers, including Mandel, pursuing RNA editing–based therapeutics for the condition.”

Read more here:


Rett Syndrome Research Trust Joins the Bespoke Gene Therapy Consortium

“The Rett Syndrome Research Trust (RSRT) has joined the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP®BGTC), a public-private collaboration led by the Foundation for the National Institutes of Health (FNIH) and the National Institutes of Health (NIH), is intended to accelerate new gene therapies for patients with rare diseases. RSRT is one of two disease-specific advocacy groups to join the Consortium since it was launched in October 2021…”

“Having a seat at the Bespoke Gene Therapy Consortium table to represent Rett syndrome and the unique gene therapy challenges around neurological disorders is an honor and special privilege,” said Randall Carpenter, MD, RSRT’s Chief Medical Officer. “Collaborating with this distinguished group of scientists, industry representatives, and regulators allows RSRT to more effectively navigate the gene therapy landscape, and shape the considerations and decisions that will affect the Rett syndrome community…”

Read more here:


Rett Syndrome Global Registry

“…The Rett Syndrome Global Registry will take a new approach to understanding Rett syndrome by unifying patient data captured from a variety of sources into a single, clinical trial-grade registry.”

“…The Rett Syndrome Global Registry is a key component of RSRT’s plan to cure Rett syndrome by incubating and expediting curative treatment development programs for biopharmaceutical companies, and facilitating the drug approval process.”

Read more here:


To learn more about the comprehensive RSRT “CURE 360” plan, visit: