, /PRNewswire/ -- ("Myrtelle" or the "Company"), a clinical stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) has selected the Company's gene therapy, rAAV-Olig001-ASPA for the treatment of Canavan disease (CD), as one of a few programs for participation in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.
The objective of the program is to accelerate development of gene therapies for rare diseases that lead to significant disability or death within the first decade of life by facilitating more frequent advice and regular communication with FDA staff. These communications go beyond available formal meetings and are meant to expedite the review process for approval. As a pilot program, this ad-hoc communication mechanism will test if reducing wait times associated with the formal FDA meeting process quickens the pace of development for products intended to address unmet medical needs.
"Acceptance into the START pilot program is an honor in that it recognizes rAAV-Olig001-ASPA as a candidate for accelerated development as a potential treatment for Canavan disease. Opening the lines of communications beyond traditional meeting pathways provides the opportunity to quickly address development issues that would otherwise delay progression to market application. We are en.
