What is the scAAV9/GAN Investigational New Drug?
Hannah’s Hope Fund had monies available to fund everything except the actual clinical trials. Lori approached the National Institutes of Health (NIH) and Dr. Carsten Bonnemann agreed to run a clinical study out of the NIH Clinical Center in Bethesda, MD.
In May of 2015, the first patient in the world to receive a therapeutic gene to the spinal cord via spinal fluid was a patient with Giant Axonal Neuropathy! Yes, this was a first-in-human made possible by the thousands of people supporting Hannah’s Hope Fund since Hannah’s diagnosis in 2008!
In February of 2021, Hannah’s Hope Fund sold the rights to the human data and the supporting pre-clinical data to Taysha Gene Therapies. As of the end of 2021, Taysha has amassed a team of 120 experienced professionals who are dedicated to pressing AAV9/GAN on through the research phase to commercialization so it is available for generations to come.
The scAAV9/GAN clinical study is still underway. Taysha is looking to add one or two additional study sites to speed up the pace of this critical study, which will hopefully be completed in 2023.
AAV9/GAN gene therapy clinical trial identifier: NCT02362438