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NIH Technology Opportunity Webinar: Novel therapy for Retinitis Pigmentosa
Register to attend a free NIH webinar and learn about a potential novel treatment for Retinitis Pigmentosa (RP). Dr. Patricia Becerra and her team at the National Eye Institute developed a novel therapeutic pigment epithelium-derived factor (PEDF) peptide that exhibits a highly potent effect on photoreceptor protection in in vivo mouse models.
Over 100,000 people in the U.S. and 1.5 million people worldwide suffer from RP, which leads to progressive photoreceptor cell degeneration and ultimately vision loss. This new therapeutic PEDF peptide has the potential to be a treatment option for the worldwide RP patient population and may also protect retinal cells from damage associated with glaucoma and age-related macular degeneration (AMD).
This novel therapeutic PEDF peptide contains a 17 amino acid residue section of the human PEDF protein that incorporates an amino acid substitution at position 105 (PEDF 17-mer[H105A]) which exhibits a highly potent effect on photoreceptor protection in in vivo mouse models of RP. The treatment method contemplates an eyedrop formulation to deliver the novel PEDF peptide to a patient’s eye, in addition to an AAV2-based delivery system, to deliver a nucleic acid encoding the novel PEDF peptide or the PEDF 17-mer[H105A] peptide.
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