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-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.

TS-005640 — This new gene editing system is based on prime editing, connecting N-terminal mutations in the DMD gene to enable expression of full-length or near-full-length dystrophin. This system also has the advantage of stably incorporating edits into the genome. Current treatments such as exon skipping and microdystrophin gene replacement have not successfully halted disease progression in clinical trials, leaving a critical need for therapies that restore full-length or near-full-length dystrophin, especially for patients with mutations in the N-terminal region of the gene, which have been under-addressed.

TS-005619 — This new gene-delivery platform optimization for safe and efficient transduction of Schwann cells in the peripheral nervous system addresses the barriers in treating demyelinating neuropathies such as Charcot‑Marie‑Tooth disease (CMT1A, CMT1B, CMT4, CMT‑X), neurofibromatosis, and Multiple Sclerosis (MS). Through the combination of several capsid engineering strategies to enhance the tropism of adeno-associated virus (AAV) vectors for Schwann cells. Using barcodes ancestral AAV libraries that enable systematic identification of structural capsid motifs associated with Schwann‑cell targeting, in combination with peptide, DARPin, or nanobody insertions at key capsid regions, VRVIII, VRIV, and the VP2 N‑terminus, to enhance Schwann‑cell receptor binding and blood‑nerve‑barrier penetration. This platform produces novel capsid variants that achieve dramatically improved Schwann‑cell tropism at lower vector doses, reducing systemic and liver exposure.