Taysha Gene Therapies to present biodistribution data from AAV9 analysis

Taysha Gene Therapies (TSHA) will present biodistribution data from an analysis of 28 non-human primates evaluating the delivery of AAV9 gene therapy vectors across five different studies using intrathecal delivery by lumbar puncture or intra-cisterna magna injection. The data will be presented during a poster presentation at the upcoming 31st Annual Congress of the European Society of Gene & Cell Therapy, taking place in Rome, Italy from October 22-25, 2024. These findings reinforce Taysha’s clinical development approach utilizing IT administration to deliver AAV-based gene therapies designed to treat the genetic root cause of CNS diseases, including the TSHA-102 program in clinical evaluation for children, adolescents and adults living with Rett syndrome. “Findings from an analysis across five NHP studies showed that both IT and ICM administration led to comparable, consistent and widespread biodistribution of AAV9 vector throughout the brain and spinal cord regions,” said Sukumar Nagendran, President and Head of Research & Development. “Importantly, these findings further support the clinical potential of IT administration as an effective, safe and minimally invasive delivery approach for broad targeting of the CNS that has potential for outpatient use in both children and adults. We believe the NHP biodistribution data, together with our additional preclinical data, reaffirm our clinical development strategy utilizing IT administration and provide translational support for the broad clinical effect reported following treatment with TSHA-102 in both our REVEAL adolescent/adult trial and REVEAL pediatric trial.”