September 21, 2016
Abeona Therapeutics Inc., a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, announced today the addition of an AAV-based infantile Batten disease (CLN1) program to its Batten pipeline. The gene therapy treatment, developed at the University of North Carolina at Chapel Hill by Steven Gray, Ph.D. with the support of Taylor’s Tale, The Saoirse Foundation, Hayden’s Batten Disease Foundation and the Batten Disease Support and Research Association, is anticipated to enter clinical trials as early as 2018.
Dr. Gray’s work has shown promising preclinical efficacy in mice with infantile Batten disease. Because the treatment works best when administered early in the disease course, it is important to screen children early, before symptoms appear. In hopes that this will someday become a clinically meaningful treatment for children like Taylor, Taylor’s Tale must now work to ensure that babies born in our state of North Carolina will be screened for devastating diseases within two years of their addition to the federal Recommended Uniform Screening Panel.
Thank you to Dr. Gray, Abeona Therapeutics and all of our donors who helped make this incredible milestone possible. We look forward to working with our partners and donors to bring this revolutionary gene therapy treatment to patients who need it.