Taysha Gene Therapies R&D Day 2
10:00 am - 1:00 pm EST
Topics of discussion will include:
-TSHA-104 (SURF1-associated Leigh syndrome): AAV9-based gene therapy with a transgene encoding the human SURF1 protein to potentially treat SURF1-associated Leigh syndrome, a monogenic mitochondrial disorder with no approved treatments. This gene replacement therapy program is currently in IND/CTA-enabling studies, with an IND/CTA filing expected in the second half of 2021.
-TSHA-105 (SLC13A5 deficiency): AAV9-based gene therapy expressing human SLC13A5 protein to potentially treat SLC13A5 deficiency, a rare autosomal recessive epileptic encephalopathy characterized by the onset of seizures within the first few days of life. This gene replacement therapy program is currently in IND/CTA-enabling studies.
-TSHA-103 (SLC6A1 haploinsufficiency disorder): Gene replacement therapy constructed from a codon-optimized version of the human SLC6A1 gene packaged within an AAV9 viral vector for the treatment of SLC6A1 haploinsufficiency disorder. This program is currently in IND/CTA-enabling studies.
-TSHA-111-LAFORIN and TSHA-111-MALIN (Lafora disease): Recombinant AAV9 viral vectors with miRNA targeting the knockdown of GYS1 for the treatment of Lafora disease. These programs are currently in IND/CTA-enabling studies.
-TSHA-112 (APBD): Recombinant AAV9 viral vector with miRNA targeting the knockdown of GYS1 for the treatment of Adult Polyglucosan Body Disease (APBD). This program is currently in IND/CTA-enabling studies.
-Additional preclinical programs, including tauopathies (TSHA-113, an AAV9 viral vector that utilizes AAV-mediated gene silencing to deliver life-long reduction of tau protein levels in neurons following a single dose) and Angelman syndrome (TSHA-106, an AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS).