CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced the appointment of Diana M. Escolar, M.D., FAAN, as chief medical officer. Dr. Escolar brings deep expertise across rare diseases, with a focus on lysosomal and neurogenetic disorders. She has been heavily involved in guiding the development of new therapies for rare diseases, including Pompe disease.
“We intend to rapidly expand enrollment across our global clinical programs this year, following our multiple strong data readouts in late 2020, as well as continue to plan our regulatory strategy to potentially bring our most advanced program in Fabry disease closer to approval in one or more major markets, and we’re delighted to have Diana on board to lead this work,” said Geoff MacKay, president and CEO of AVROBIO. “We’re also looking ahead to our next wave of indications, which target disorders that can cause rapid and devastating neurological damage. We believe Diana’s proven track record in pediatric neurogenetic diseases will be critical as we advance these programs, including initiating a planned clinical trial for our investigational gene therapy for Hunter syndrome later this year.”
“AVROBIO’s vision for and support of the lysosomal disorder community speak directly to my personal commitment to improving patient care globally and developing new treatment options to those who need them most,” said Dr. Escolar. “As a neurologist, I am especially excited for the potential of ex vivo lentiviral gene therapy to treat the brain as well as the rest of the affected organs – an important advance over the current standard of care for patients living with lysosomal disorders. I am proud to join this impressive team in their pursuit of bringing personalized gene therapy to the world.”
A board-certified neurologist and fellow of the American Academy of Neurology, Dr. Escolar previously served as chief medical officer at miRagen Therapeutics, a clinical-stage company developing microRNA therapeutics for leading indications in rare diseases. She also served as the chief medical officer of Akashi Therapeutics, Inc., the principal medical consultant for Acceleron Pharma’s muscular dystrophy program and a senior medical consultant and advisory board member for the muscular dystrophy programs at Shire, Plc. Dr. Escolar was the principal medical consultant for Genzyme’s pivotal trial for Myozyme, an enzyme replacement therapy approved to treat adult-onset Pompe disease.
In addition to her industry leadership, Dr. Escolar has served as associate professor of neurology at both Johns Hopkins University and George Washington University School of Medicine and Health Sciences, and was on the faculty at the Kennedy Krieger Institute. She has served as the co-founder and director of the Cooperative International Neuromuscular Research Group (CINRG) and as a steering committee member of the Spinal Muscular Atrophy Pilot Program funded by the National Institutes of Health (NIH). She served as director of the amyotrophic lateral sclerosis (ALS) clinic at George Washington University. As a faculty member at the Children’s National Hospital and Children’s Research Institute in Washington, D.C., she initiated and developed an international pediatric neuromuscular program, serving as director of the muscular dystrophy association (MDA) clinic and leading an international network conducting translational and clinical research in pediatric neurogenetic disorders.
About AVROBIO
Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.
