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-005787 — This custom DNA methylation microarray is designed to assess long‑term health risks and recovery potential associated with early‑life nutritional stress and starvation, based on the discovery of unique DNA methylation signatures that distinguish individuals who survived severe childhood starvation and achieved advanced age (70–80 years).

TS-005785 — This hands‑free dispensing device generates a refillable air reservoir to enable precise, controlled dispensing or withdrawal of small volumes of material by a single operator. This technology would have multiple applications in embryology where mouth pipettes are still quite common, perfusions of animal tissue with fixatives, dyes, etc.

TS-005784 — This novel, personalized hormone‑replacement therapy for anorexia nervosa (AN) simultaneously targets dysregulated ghrelin and leptin signaling using a transdermal skin‑patch delivery system. This represents a new device and treatment paradigm for AN, introducing both a previously undescribed hormone combination therapy and a precision‑medicine framework that tailors treatment based on epigenetic biomarkers, with strong translational potential supported by an existing, genomically characterized patient cohort.

TS-005782 — This new IP introduces a modular, cell‑derived lipid nanoparticle (LNP) delivery platform engineered for highly efficient, biocompatible, and customizable delivery of nucleic acids and therapeutic agents, which is a significant improvement over existing synthetic LNP technologies.

TS-005781 — This invention introduces an advanced mRNA‑based gene‑therapy platform designed to treat both genetic and acquired elastin‑deficiency conditions, including autosomal dominant cutis laxa (ADCL), supravalvular aortic stenosis (SVAS), emphysema, aneurysms, and age‑related tissue degeneration. This IP represents a significant advancement over both plasmid‑based and existing mRNA therapies, establishing a safer, more effective strategy to address the root cause of elastin‑related diseases.

TS-005780 — A novel mouse model engineered to recapitulate a human disease–associated variant in AIFM1, in which a valine at position 67 is replaced by a premature stop codon. This model provides a valuable in vivo platform for studying the mechanisms underlying AIFM1‑associated disease and for evaluating potential therapeutic strategies.