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-005789 — This novel vitro, cell‑based potency assay for evaluating adeno‑associated virus (AAV)–mediated gene therapies targeting muscle disorders, particularly those utilizing AAV9 vectors. This represents a significant improvement in potency determination workflows, providing a reproducible, scalable, and regulatory‑relevant alternative to animal‑based assays for muscle‑directed gene therapies.

TS-005788 — This improved AAV vector–design strategy significantly reduces genome truncations caused by micro‑RNA–derived DNA hairpins during viral replication, introducing targeted point mutations within the DNA sequence encoding the micro‑RNA passenger strand. This innovation improves AAV batch quality, enhances efficacy and safety of micro‑RNA–based gene therapies, and reduces manufacturing cost.

TS-005769 — This novel gene‑therapy strategy is the first known anti-DUX4 gene-therapy targeting DUX4 as a potential universal driver of cancer progression. The proposed approach repurposes an existing AAV‑based platform to deliver a therapeutic microRNA (mi405) that specifically knocks down DUX4 expression in cancer cells.

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-005674 — This IP is a novel RNA‑targeted therapeutic strategy for alveolar rhabdomyosarcoma (ARMS) using CRISPR‑Cas13d to enable the highly specific degradation of the oncogenic mRNA while preserving the wild‑type PAX7 and FOXO1 genes, avoiding the genotoxicity risks associated with DNA‑targeting CRISPR systems.

TS-005663 — This new method builds upon previous HR templates for correcting CFTR mutations. This invention combines HMEJ templates with DNA-repair inhibitors to improve gene insertion further. Combining HMEJ templates and DNA repair inhibitors during gene editing has shown more efficient CFTR cDNA replacement, which could be utilized in both in vivo and ex vivo therapies for people with cystic fibrosis (CF).

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.