The Rett Syndrome Research Trust (RSRT) has launched an initiative to advance three gene medicines for Rett syndrome into clinical trials by 2028.
Called the Cures Roadmap, it seeks to raise $40 million over the next four years to help select and develop therapies and is based on the new MECP2 Editing Consortium, a collaboration between six labs working on gene-editing therapies. and next-generation RNA. for Rett syndrome.
RNA molecules include messenger RNA that carries instructions from genes and functions as a template to make proteins, and other regulatory RNA molecules that regulate protein production.
“Next-generation drugs have the potential to provide even more benefits and extend the reach of a cure to more patients,” Monica Coenraads, CEO of RSRT and a parent of a child with Rett, said in an organization press release.
Rett syndrome is mainly caused by mutations in MECP2 gene that impairs the function of MeCP2, a protein that regulates the activity of other genes. MeCP2 plays an important role in the brain, helping to maintain the connection between nerve cells, and loss of the protein in Rett impairs the growth and connection of nerve cells, leading to symptoms of the disease.
However, too much MeCP2 can be toxic to cells, so its levels must be fine-tuned. Both gene and RNA editing do not interfere with cells’ ability to modulate MeC2 protein levels, which makes these approaches attractive, according to the press release.
The goal is to accelerate the development of therapies using CRISPR-based technology
The RSRT initiative aims to accelerate the development of therapies, using editing systems such as base editing and prime editing, both based on CRISPR technology. While base editing corrects single mutations in a single building block of the genetic code, master editing can correct multiple mutations.
A team including RSRT staff, consultants and experts in genomic medicine, clinical trial design and regulation are working towards the goal of accelerating treatment development. The Rett community is encouraged to contribute through fundraising events and fundraising campaigns.
“RSRT is uniquely positioned in the Rett world with staff and advisors who have deep drug development and genetic medicine experience to effectively guide translational research and advance cures,” said Robert Deans, PhD, chief executive officer of technology of RSRT.
According to the organization’s plan, three gene drugs should be selected by 2025, and safety and dosing studies will be completed by 2026. Then, after approval by the US Food and Drug Administration, clinical trials could begin in 2028. .
The RSRT launched the Roadmap for Cures in 2017 to advance genetic medicine approaches that target the root cause of Rett. The organization has invested more than $11 million in two gene therapy programs for Rett that are now in clinical trials: Neurogene’s NGN-401 and Taysha Gene Therapy’s TSHA-102. Both are designed to provide working versions of MECP2 gene in patients’ cells.
“Given the dramatic nature of symptom change in animal models of Rett syndrome, we are optimistic that targeting the underlying genetic cause will be transformative,” said Coenraads.
“Time is of the essence, and the best chance of achieving a cure in Rett is to advance, in parallel, as many shots on goal as possible. That’s the goal of the Roadmap for Cures,” she added.
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