Abeona is eager to continue its commitment to the Batten community as they focus on bringing the potential CLN3 gene therapy, developed by Tammy Kielian, Ph.D. and her team at the University of Nebraska Medical Center, into clinical trial. Their aim remains to initiate enrollment at the University of Rochester during the latter half of 2017 and the team is working diligently to complete the numerous and critical steps to be able to do so. As they progress, they will surely seek insight from families that have had or have children impacted by CLN3 to better understand how to best serve participants in clinical trial and other important considerations.
Abeona is further pleased to extend our gene therapy programs for Batten disease to include CLN1 through a recently announced collaboration with Steven J. Gray, Ph.D. and his team from the University of North Carolina Chapel Hill. In pre-clinical gene therapy studies that deliver a functioning copy of the CLN1 gene to cells in the central nervous system of INCL mice, Gray’s team demonstrated extended survival and preservation of strength. The pre-clinical work was supported by Batten Disease Support and Research Association along with Saoirse Foundation, Taylor’s Tale, and Hayden’s Batten Disease Foundation.
As 2016 draws to a close, there is certainly much to look forward to in 2017 and beyond. Abeona is looking forward to providing information and updates along the way and hearing more from the community.