Novel Therapeutic Applications of Gene Editing Technology

This inventive concept expands the application of gene editing technology to new therapeutic areas, including hearing loss, age-related macular degeneration, peripheral neuropathy, sickle cell disease, and muscular dystrophy, providing new hope for patients with these debilitating conditions.

The original patent focused on treating mucopolysaccharidosis type I (MPS I) using a combination of AAV vectors. However, the core technology has broader applicability, and this inventive concept addresses the limitations of the original patent by exploring new therapeutic areas where gene editing can make a significant impact.

The new claims describe systems and methods for treating various diseases using gene editing technology, including engineered zinc finger nucleases (ZFNs) and AAV vectors. For example, the composition of ZFNs can be used to site-specifically integrate a corrective copy of a hearing-related gene into inner ear cells to restore hearing. Similarly, AAV vectors encoding an enzyme that degrades lipofuscin can be administered to prevent and/or treat age-related macular degeneration. These new applications leverage the core technology to address unmet medical needs in diverse fields.

The new claims introduce novel and non-obvious applications of gene editing technology, expanding its therapeutic reach beyond MPS I. The inventive step lies in the recognition of the technology's broader potential and the development of new systems and methods tailored to specific diseases.

Alternative embodiments may include the use of CRISPR-Cas9 or other gene editing tools, different vector systems, or various routes of administration. Variations may involve targeting different genes or using combination therapies to enhance treatment outcomes.

This inventive concept has significant commercial potential, as it opens up new markets in hearing loss, ophthalmology, neurology, and hematology. The addressable market is substantial, with millions of patients worldwide suffering from these diseases, and the technology has the potential to transform treatment paradigms and improve patient outcomes.