MeiraGTx Announces Upcoming Presentation on Achromatopsia Gene Therapy Candidate AAV-CNGA3 at ARVO 2019
April 25, 2019
Details of MeiraGTx’s poster are as follows:
Title: Development and efficacy assessment of AAV2/8-hG1.7p.coCNGA3, a CNGA3 gene therapy vector
Abstract Number: 3426 - A0197
Session Name: Gene therapy for ocular disorders
Location: West Exhibition Hall
Data Summary: AAV-CNGA3 utilizes a proprietary engineered promoter (hG1.7p) that was associated with strong, pan-cone gene expression in induced pluripotent stem cell (iPSC)-derived retinal organoids driving a codon-optimized CNGA3 cDNA. In pre-clinical models, treatment with AAV-CNGA3 resulted in long-term visual improvements and cone photoreceptor survival at titers planned for use in a Phase 1/2 clinical trial of AAV-CNGA3.
AAV-CNGA3 is a gene therapy treatment being developed as a potential treatment for patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene. Delivered via subretinal injection to a patient’s cone receptors at the back of the eye, AAV-CNGA3 is designed to restore cone function by delivering a normal copy of the CNGA3 gene. AAV-CNGA3 has been granted orphan drug designation and rare pediatric disease designation by the
AAV-CNGA3 is being developed in collaboration with
Achromatopsia is an inherited retinal disorder that specifically prevents cone photoreceptors from functioning. ACHM is characterized by severely reduced visual acuity of 20/200 or worse, disabling light sensitivity (photoaversion) and involuntary back and forth eye movements (nystagmus). ACHM occurs in approximately one in 30,000 people in
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Forward Looking Statement
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding our product candidate development and anticipated 2019 milestones regarding its pre-clinical and clinical data, as well as statements that include the words “expect,” “intend,” “plan,” “believe,” “project,” “forecast,” “estimate,” “may,” “should,” “anticipate” and similar statements of a future or forward-looking nature. These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, acquire additional capital, identify additional and develop existing product candidates, continue operating as a going concern, successfully execute strategic priorities, bring product candidates to market, build-out the manufacturing facility and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; failure of early data to predict eventual outcomes; failure to obtain