Gene Therapy for Optic Neuropathy

Problem: 
Optic neuritis is a condition commonly observed in MS patients that leads to temporary or permanent visual decline following demyelination of the optic nerve and loss of retinal ganglion cells. Current therapies for MS and ON include immunosuppressive agents that mitigate the inflammatory component of disease. Unfortunately, these treatments provide temporary symptomatic relief and, moreover, do not attenuate further neuronal loss.

Solution: 
Jean Bennett, one of the first investigators to develop a gene therapy for a rare inherited form of retinal blindness, has developed a novel Adeno Associated Viral Vector that drives constitutive expression of human SIRT1 in vivo in the mouse retina of an experimental autoimmune murine model of Multiple Sclerosis (MS).

Bennett and her team have demonstrated SIRT1’s neuroprotective augmentation in suppressing retinal ganglion cell death, optic nerve inflammation and demyelination, and vision loss.

Stage of Development:

• Penn has intellectual property claiming these novel vectors encoding the SIRT1 gene for treatment for treatment of retinal disorders resulting from optic nerve damage.

Intellectual Property:

• US 11,879,133
• EP Patent Pending
• US Patent Pending

Desired Partnerships: 

• Licensing
• Co-Development.

As discussions progress, we would happy to put you in touch with Penn’s world class gene therapy investigator who led this important new initiative

Docket # 17-8143