AVROBIO Announces the Appointment of Diana M. Escolar, M.D. as Chief Medical Officer

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 19, 2021-- 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.


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.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as “aims,” “anticipates,” “believes,” “could,” “designed to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the expected benefits from the appointment of Dr. Escolar to the position of chief medical officer, statements regarding our business strategy for and the potential therapeutic benefits of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including the planned clinical trial for our investigational gene therapy for Hunter syndrome, clinical trial results, product approvals and regulatory pathways, and anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success.

Any forward-looking statements in this press release are based on AVROBIO’s current expectations, estimates and projections about our industry as well as management’s current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIO’s product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned preclinical or clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIO’s product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIO’s actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled “Risk Factors” in AVROBIO’s most recent Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIO’s subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Christopher F. Brinzey
Westwicke, an ICR Company

Stephanie Simon
Ten Bridge Communications

Source: AVROBIO, Inc.

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