Delivery of NAV AAV8-based gene therapy through the suprachoroidal space can potentially provide a targeted, in-office, non-surgical approach to obtaining widespread transgene expression in the retina without exposing the vitreous and the anterior segment of the eye to the injected drug. Clearside's patented SCS Microinjector is specifically designed to allow for consistent injection into the suprachoroidal space and has been tested in over 1,000 injections in clinical trials to date.
"We are pleased to partner with Clearside in evaluating the use of the SCS Microinjector to deliver RGX-314 to the suprachoroidal space in the eye. We believe this approach can potentially allow for the treatment of an expanded population of patients with wet AMD and DR by providing access to gene therapy treatment in all settings of patient care," said
A recent research collaboration with
"Intravitreal delivery of AAV gene therapy can be limited by ocular inflammation, immune responses in the vitreous and restrictive physical transduction barriers due to the inner limiting membrane on the retina. These hurdles can potentially be overcome with alternative in-office delivery approaches such as delivery to the suprachoroidal space," said
"Following on the positive long-term clinical results from RGX-314 delivered subretinally, it is exciting to see that preliminary preclinical studies with suprachoroidal delivery of RGX-314 achieved widespread transgene expression in the retina, similar to what was observed in RGX-314 preclinical studies using subretinal delivery. Suprachoroidal delivery of gene therapy has the opportunity for broad transduction of the retina and has the potential to be the preferred choice for in-office treatment of wet AMD, DR and other chronic retinal diseases," said
Under the terms of the agreement, Clearside has granted
RGX-314 is being developed as a potential one-time treatment for wet AMD, DR and other additional chronic retinal conditions treated with anti-VEGF. RGX-314 consists of the NAV AAV8 vector encoding an antibody fragment which inhibits VEGF, modifying the pathway for formation of new leaky blood vessels which lead to retinal fluid accumulation and vision loss. In
About REGENXBIO Inc.
Clearside Biomedical, Inc.
REGENXBIO Forward-Looking Statements
This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things,
Clearside Forward-Looking Statements
Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as "believe", "expect", "may", "plan", "potential", "will", and similar expressions, and are based on Clearside's current beliefs and expectations. These forward-looking statements include statements regarding the potential application of the SCS Microinjector for AAV gene therapy and the potential benefits of the SCS injection platform. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside's reliance on third parties over which it may not always have full control, and other risks and uncertainties that are described in Clearside's Annual Report on Form 10-K for the year ended
- Ding, K., Shen, J.,
Hafiz, Z., Hackett, S. F., Silva, R. L. E., Khan, M., … Campochiaro, P. A. (2019). AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression. Journal of Clinical Investigation. doi: 10.1172/jci129085