Unmet Need
Inherited retinal diseases (IRDs) are a group of disorders with genetic and clinical heterogeneity and are the leading cause of vision loss. [1, 20] ] Collectively, IRDs have an estimated incidence of 1:2000. [1] Gene therapies targeting IRD, including FDA approved Luxturna, are injected directly into the retina to correct the underlying genetic defect. Viral gene therapy strategies have several limitations, including high cost, risk of cancer and immunological side effects, limited payload packaging capacity and suboptimal tissue-specific delivery [6]. Therefore, there is a strong need for a non-viral gene therapy delivery vector to overcome the barriers associated with viral vectors and improve health outcomes for patients with IRD.
Technology Overview
Researchers at Johns Hopkins have developed a synthetic polymer for non-viral vector for delivery of gene modification therapies to the eye. The delivery platform is applicable to various nucleic acid payloads (plasmid DNA, mRNA, siRNA, CRISPR/Cas9, etc.) and target tissues, such as ocular tissues. This methodology provides a standardized, scalable, and low-risk platform for gene modification therapy delivery and can be expanded for use across the human body.
Stage of Development
The inventors have established efficacy of gene transfer using the technology in small animals. Upcoming studies to validate efficacy and toxicology in animal models of IRD.
Publication
N/A
