ABOUT GENE THERAPY

We believe our platform offers the potential for safer, targeted ocular gene therapy without some of the risks of surgery and subretinal administration. Suprachoroidal administration of gene therapy could ultimately enhance access to care because it does not require specialized gene therapy surgery treatment centers. The procedure for suprachoroidal injection is conducted in an office setting and is similar in terms of patient preparation and duration to the procedure for intravitreal injection.  Therefore, we believe our products could be incorporated into retina specialists’ standard medical practice.

We believe suprachoroidal administration may further enhance the value proposition of ocular gene therapy by potentially improving safety and expanding access.

The Suprachoroidal Space Reversibly Opens Posteriorly and Circumferentially Following DNA Nanoparticle Administration

Clearside Biomedical Preclinical Studies

POTENTIAL BENEFITS

  • Convert gene therapy into an office-based procedure

  • Avoid risks of vitrectomy (surgery), retinotomy, subretinal injection, and macular detachment

  • Deliver viral and non-viral vectors

  • Provide broader retinal coverage

  • Enhance patient access

TARGET INDICATIONS

Inherited retinal diseases, or IRDs, such as Stargardt disease and Usher syndrome, represent some of the most challenging diseases that ophthalmologists encounter. They cause progressive, relentless vision loss due to changes in genes critical to the survival of photoreceptors and RPE cells, yet delivery of therapeutics to these cells is challenging.

In preclinical animal studies from which data was presented at the American Academy of Ophthalmology 2019 Annual Meeting in October 2019, the suprachoroidal injection of luciferase DNA nanoparticles (DNPs), in rabbits produced activity comparable to that seen from subretinal injections of luciferase DNPs. In these studies, SCS injections of DNPs were generally well tolerated across both rabbits and non-human primates, and no significant abnormalities were observed on ophthalmic exams. DNPs can also transfer large genes at potentially higher doses without the risks of subretinal surgery, which may allow for gene therapy in some of the most common IRDs.

PIPELINE STATUS

PRECLINICAL STUDIES
In preclinical studies conducted independently and through collaborations with both an academic center and gene therapy companies, we have observed that suprachoroidal injection can administer both viral and non-viral gene therapy. Using marker genes like green fluorescent protein and luciferase in both rabbits and non-human primates, gene therapy was delivered with our suprachoroidal injection to achieve expression in the retina and choroid.

To expand our overall development pipeline, we are looking to selectively partner our proprietary technology for use with novel gene therapies. For further information on available indications, please contact Rick McElheny, VP, Business Development at rick.mcelheny@clearsidebio.com.