Taysha's gene replacement trial for Rett, TSHA-102, is now recruiting. The first site is in Montreal, Canada. This program got its start through RSRT's Gene Therapy Consortium and MECP2 Consortium.
Unravel Biosciences plans to take the first drug validated in their new platform, a liquid reformulation of vorinostat for Rett syndrome, into the clinic in a one-patient trial outside of the US by the end of this year.
Neurogene Inc., a company founded on the vision to push the boundaries of genetic medicine to address complex and devastating neurological diseases, announced the addition of NGN-401 for Rett syndrome to the Company’s development pipeline.
Alcyone Therapeutics, a biotechnology company pioneering next-gen precision gene-based therapies for complex neurological conditions, today presents preclinical data showing the Company’s partnered novel AAV9 gene therapy vector ACTX-101 safely and effectively reactivates the inactive X ...
As it stands there is no nationally or internationally defined registry for Rett syndrome. With the valuable data and collective knowledge that parents have related to their children with Rett Syndrome, it is critical to have a research-ready, clinical-trial-grade database that combines ...
In a fourth-quarter corporate update, Taysha Gene Therapies announced that it will focus its efforts on clinical programs targeting Rett Syndrome and giant axonal neuropathy (GAN). Homing in on these programs will result in the pausing of other clinical research and development and a 35% ...
Spectrum, a leading source of news on autism research, spoke to Dr. von Hehn, RSRT's senior director of research and clinical strategy, about the Rett Syndrome Global Registry which will be launching soon.
Technology Networks speaks with Saul Kato, the CEO of Herophilus, about how the company plans to reinvent neuro drug discovery, by combining human brain models, scaled biology and machine learning to decode brain disease, including Rett syndrome.
RSRT has invested $8 million in RNA editing and the investment is paying off! Four of the seven companies mentioned in this article have Rett programs. Read on....