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-004707 — This IP is a gene therapy designed to treat cystic fybrosis using variants to improve stability and channel conductivity.

TS-004610 — This is a new approach to gene therapies in cells that are longer than 4.5 kb. By inhibiting DNA-PKcs and 53BP1, it allows the enrichment cassette in the second AAV to be replaced with genetic cargo, which consequently allows insertion for genes up to 6.5-7 kb.

TS-004608 —