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-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-004703 — For AAVs that harbor gene knockdown machinery, this IP is a novel potency assay development for release testing that creates a HEK293T stable cell line to integrate two genetic components.

TS-004612 — This IP is a therapeutic strategy for facioscapulohumeral dystrophy (FSHD), which is associated with an inappropriate expression of DUX4. Using dual AAV split-intein vectors, it expresses full-length SMCHD1, enhances epigenetic silencing and reduces DUX4 toxicity.

TS-004598 — This system may help improve the safety of AAV vector products by decreasing cross-packaged bacterial sequences, increasing correctly packaged AAV payloads and blunting ITR-driven transcription of cross-packaged material to avoid expressing potentially toxic bacterial sequences.

TS-004596 — There's a signficiant unmet need among thousands of Americans who struggle with genetic neuromuscular disease; this one-time AAV-delivered proprietary engineered miRNA targets core disease biology, specifically in Facioscapulohumeral Muscular Dystrophy (FSHD). This durable gene therapy reduces immunogenicity by taking advantage of nature’s highly active gene regulation biology.

TS-002282 — Rare variants in genes GRIN2A, GRIN2B and GRIN2D are associated with severe childhood-onset neurological diseases like epileptic encephalopathies and autism. There are no treatments for these chronic disorders that cause developmental delays or developmental skill loss. Researcher and principal investigator, Scott Harper, at Nationwide Children’s Hospital developed a genetic therapy for GRIN2 disease through a knockdown-and-replace strategy by using artificial microRNAs that non-selectively knockdown both mutant and wildtype GRIN2A alleles using RNAi and adding back an RNAi-resistant, wildtype GRIN2A cDNA. The therapy will express functional GRIN2 exogenously while eliminating endogenous GRIN2 mRNA entirely through AAV delivery.

TS-002175 — The third most common type of muscular dystrophy, Facioscapulohumeral Muscular Dystrophy (FSHD), affects over 870,000 individuals worldwide by causing debilitating pain, muscle weakness, fatigue along with many other symptoms in their face, shoulders, upper arms and lower legs. Researchers at Nationwide Children’s Hospital created a treatment using Salvianolic Acid (SAA) as a drug therapy for neuromuscular disorders including FSHD.

TS-002174 — Currently, no cure exists for Charcot-Marie tooth type 1B (CMT1B). Inventors and specialists in Gene Therapy at Nationwide Children’s Hospital invented a methodology along with sequences for using microRNAs (miRNA) to inhibit and replace abnormal expressions of the myelin protein zero (MPZ) gene. Affecting 1 in 30,000 people, CMT1B is caused by more than 200 mutations of the MPZ, the essential protein needed for a healthy and efficient peripheral nervous system. The accumulation of mutant MPZ genes will result in, but not limited to, muscle weakness, atrophy, lost of sensation in the lower legs and feet and sensory loss.