TS-005572 — This novel gene replacement strategy uses an adeno-associated viral vector to treat Cockayne syndrome type B (CSB). CSB is a rare neurodegenerative disorder caused by mutations in the ERCC6 or ERCC8 genes, both of which are responsible for DNA repair, causing tremors, ataxia, seizures, and cachectic dwarfism, significantly impacting their quality of life. An AAV-based gene replacement therapy will effectively combat this disorder by delivering a functional copy of the defective gene into target cells, thereby restoring normal gene expression.

TS-004704 — This IP automates the miniaturization of large gene targets through a structure-based protein engineering AI algorithm.

TS-004616 — A method for targeted delivery and constitutive expression of large genes using adeno-associated viral (AAVs) vectors that contain ubiquitous mini-promoters. Incorporation of the compact mini-promoters will increase the cargo space of the AAVs such that larger genes can be packaged into the vectors.