AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a vision to free people from a lifetime of genetic disease, today announced the appointment of Essra Ridha, M.D., MRCP, FFPM, as chief medical officer. Dr. Ridha brings wide-ranging expertise in early- and late-stage clinical development of ex vivo lentiviral gene therapies. As vice president, clinical development at AVROBIO, she has guided the strategic and scientific development of several AVROBIO gene therapy programs.

“As we head into 2022, we’re expecting to initiate five new clinical trials, including potential registrational trials for our clinical programs in Fabry disease, Gaucher disease Type 3 and cystinosis. To ensure we move forward with the urgency needed to meet the needs of the patient community we aim to serve, we will require broad expertise designing clinical trials for gene therapies for rare diseases, working with global regulatory agencies, as well as strong, streamlined execution, all areas where Essra offers deep, relevant experience,” said Geoff MacKay, president and chief executive officer of AVROBIO. “Considering her extensive track record at leading gene therapy and pharmaceutical companies, as well as her in-depth knowledge of AVROBIO’s strategy, capabilities and culture, we believe Essra will play an essential role in helping us advance multiple therapies through approval.”

Dr. Ridha is a global medical leader with extensive experience in clinical research and development, translational medicine and clinical medicine with specialist training in cardiology and internal medicine.

“I have been fortunate enough to work on multiple cell and gene therapy programs and believe strongly that ex vivo lentiviral gene therapy has massive potential for the treatment of lysosomal disorders,” adds Dr. Ridha. “I am therefore thrilled to lead the clinical development of such a strong lysosomal disorders pipeline, especially at this time as the early safety and durability data from several clinical trials, some of which incorporate the company’s innovative plato® platform, begin to emerge.”

Dr. Ridha’s experience includes strategically and scientifically advancing the ex vivo lentiviral gene therapy now licensed for the treatment of metachromatic leukodystrophy (MLD) and the first CAR-T regulatory cell therapy to enter the clinic for the induction of immunological tolerance in solid organ transplantation. She has worked in leadership positions within AVROBIO as well as Sangamo Therapeutics and GlaxoSmithKline, on highly innovative cell and gene therapy programs in rare neurological, metabolic (including lysosomal disorders) and immunological diseases. Previously, Dr. Ridha worked as a medical expert at Bristol Myers Squibb advising on late-stage clinical development, medical affairs, real-world evidence and HEOR in cardiovascular medicine.

In addition to her industry leadership, Dr. Ridha is a member of the Royal College of Physicians of London, as well as a Fellow of the Faculty of Pharmaceutical Medicine. She was also an expert panel member at the World Health Organization (WHO) Expert Advisory Committee to develop Global Standards for the Governance and Oversight of Human Genome Editing. Dr. Ridha earned her medical degrees from the Royal Free and University College London Medical School and earned her neuroscience degree with Basic Medical Sciences degree with honors from University College London.

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 therapeutic protein, even in hard-to-reach tissues and organs including 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 potential benefits from the appointment of Dr. Ridha to the position of chief medical officer, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, the anticipated overall safety profile of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, and the expected benefits and results of our implementation of the plato® platform in our clinical trials and gene therapy programs. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

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 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 enrollment and development timelines 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 on Form 10-Q, 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.

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