AviadoBio and Taysha Gene Therapy announcements provide hope for Rett Syndrome.
Readers of the RSAA Newsletter will be aware of an upcoming gene therapy clinical trial for Rett in Canada performed by Taysha, a company based in the USA. Taysha will also use AAV to deliver a modified form of the MeCP2 gene, as reported in a previous article on the RSAA website (https://rettaustralia.org.au/blog/a-game-changer-for-gene-therapy-for-rett-syndrome/). As this trial will focus on adults with Rett Syndrome, a recent announcement from another US company, AviadoBio, that they plan to develop gene therapy for early-onset (adult) dementia is good news for the Rett community, because the more we learn about the delivery of AAV to the human brain the more likely we are to develop a cure for Rett Syndrome.
This is an exciting time for AAV-based gene therapy projects, not least the upcoming Taysha trial to treat adults with Rett Syndrome. Our fervent hope is that this trial will provide sufficiently encouraging results to ensure that research continues with the ultimate goal of a cure for Rett.
