On September 22, 2021, Taysha Gene Therapies hosted a virtual educational event on Rett syndrome for the investment community. As you may know Taysha is pursuing a gene replacement program called TSHA-102 for Rett syndrome. This key opinion leader webinar provided investors with an opportunity to learn about the disorder from both a clinician and parent perspective, as well as get updates on TSHA-102 and the plan for the first clinical trial.
Our own Monica Coenraads, CEO of RSRT, gave a poignant presentation on her experiences parenting a child, now an adult, with Rett syndrome. Dr. Jeff Neul, MD, PhD, gave an overview of the disease, Dr. Steve Gray, PhD, Taysha’s chief scientific advisor, provided details on the gene replacement program and Dr. Suyash Prasad, Taysha’s chief medical officer, described plans for the first clinical trial.
We learned from Dr. Suyash that, pending FDA approval, Taysha plans to enroll individuals with Rett over the age of 18 for their first trial. The plan is to then move rapidly to younger age groups.
Included in the symptom assessments that Taysha will conduct is the ORCA communication tool originally developed for Angelman syndrome and now being adapted for Rett with funding from RSRT.
TSHA-102 is a gene replacement therapeutic that delivers a mini-gene generated by RSRT trustee Sir Professor Adrian Bird; Professor Bird received funding from the RSRT MECP2 Consortium. The therapeutic incorporates a regulatory mechanism called miRARE that controls the amount of MECP2 protein made. miRARE was developed by Dr. Steve Gray with funding from the RSRT Gene Therapy Consortium.
Dr. Prasad announced during yesterday’s presentation that TSHA-102 received Orphan Drug Designation from the European Commission. An orphan designation allows a pharmaceutical company to benefit from incentives from the EU, such as reduced fees and protection from competition once the medicine is placed on the market.
A recording of the key opinion leader webinar as well as the presentation deck is available on the Taysha website.