TS-005791 — This invention represents an improved, precision‑based AAV.U7snRNA‑mediated exon‑skipping gene‑therapy platform targeting exon 19 of the DMD gene to treat Duchenne and Becker muscular dystrophies.

TS-005790 — This novel AAV.U7snRNA‑mediated exon‑skipping gene‑therapy platform targets exon 18 of the DMD gene to treat Duchenne and Becker muscular dystrophies. Unlike micro‑dystrophin approaches currently in clinical development, this strategy leverages antisense sequences embedded within optimized U7snRNA cassettes to modulate endogenous DMD splicing and restore native dystrophin expression.

TS-005705 — This dual‑AAV vector system is engineered to produce a highly functional dystrophin protein that mirrors the isoform observed in asymptomatic or minimally symptomatic individuals with naturally occurring deletions across exons 19–44. This approach represents a significant advance over current microdystrophin therapies, such as Elevidys, which have shown limited efficacy and safety issues in certain patient groups.

TS-005704 — This dual‑AAV vector system is engineered to produce highly functional dystrophin protein equivalents found in asymptomatic or minimally symptomatic individuals with naturally occurring dystrophin exon deletions.

TS-005662 — This new application to treat Desminopathy, a progressive muscle disease caused by mutations in the DES gene, uses a dual knockdown and replace approach with microRNA‑based tools to selectively suppress mutant DES mRNA while simultaneously delivering a healthy, codon‑optimized DES sequence via an AAV vector.