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Gene Therapy for Macular Degeneration

Description:

Gene therapy targeting disposal of toxic intracellular lipid debris and lipofuscin in wet age related macular degeneration.

 

Inventor

Kathleen Boesze-Battaglia

 

Problem

Macular degeneration is a genetic eye disorder that affects the retina resulting in progressive vision loss. Macular degeneration has two main forms, Stargardt’s macular degeneration (STGD) which affects juveniles, and age related macular degeneration (AMD) which affects older individuals. While a fairly small proportion of the population has STGD, with an estimated prevalence of 1 in 8,000 to 10,000, AMD affects a significantly larger segment at 2 million people in 2010 in the U.S. alone. There are currently few treatment options for macular degeneration disorders and therefore a serious need for innovative solutions.   

 

Solution

Work in the lab of Dr. Kathleen Boesze-Battaglia has resulted in a novel therapeutic approach which targets the underlying cause of macular degeneration vision loss, the accumulation of toxic lipid debris including lipofuscin. Lipofuscin is a fatty yellow pigment which builds up in cells underlying the macula, eventually causing cell death and vision loss. Utilizing a unique gene therapy method, Dr. Boesze-Battaglia has achieved a significant reduction in the amount of lipofuscin accumulation in a well-established in vivo model system. Taken together, this technology could be used to potentially delay or prevent the onset of vision loss in patients suffering from macular degeneration disorders.

 

 

Advantages

•       Potential treatment for wet AMD – accounts for 80% of cases.

•       Target is gene defect independent

•       Limits lipid debris accumulation

•       May delay serious vison loss if used early

 

 

Stage of Development

•       In vitro and In vivo proof of concept

 

Intellectual Property

Provisional Pending 

 

 

Desired partnerships

• License

Co-development

 

 

Download PDF

 

Docket #  15-7361 


Patent Information:
For Information, Contact:
Melissa Kelly
Associate Director, SEAS/SAS Licensing Group
University of Pennsylvania
215-898-9877
kellymel@upenn.edu
Inventors:
Kathleen Boesze-Battaglia
Keywords: