Apic Bio, a gene therapy company developing treatment options for patients with rare genetic diseases and co-founded by UMass Medical School’s Robert H. Brown Jr., DPhil, MD, announced that the FDA has cleared its investigational new drug (IND) application for APB-102, a gene therapy candidate designed to treat a common cause of familial amyotrophic lateral sclerosis. “The clinical development of APB-102 is rooted in nearly 30 years of gene therapy research demonstrating the link between the SOD1 gene mutation and ALS and the strong potential of AAV-delivered SOD1 targeting miRNA to slow down or reverse the progression of ALS in patients with SOD1 mutations,” said Dr. Brown, the Leo P. and Theresa M. LaChance Chair in Medical Research, professor of neurology and scientific co-founder of Apic Bio.
