AVROBIO, Inc. (NASDAQ: AVRO) (the “Company”), a Phase 2 clinical-stage gene therapy company, today announced the participation of its senior management in sessions at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). These include sessions on advances in gene therapy research, manufacturing challenges during the development of gene therapy products, and the potential benefits of the Company’s plato™ platform. In addition, multiple oral presentations with preclinical data from the Company’s gene therapy programs in Gaucher disease and cystinosis are also on the program at the ASGCT Annual Meeting.

“After more than three and a half years of development, we continue to be on track towards our anticipated introduction of plato™ upgrades into our clinical programs in the second half of 2019,” said Geoff MacKay, AVROBIO’s President and CEO. “The plato platform includes a proprietary vector system, an automated cell manufacturing solution and refined conditioning regimen with therapeutic drug monitoring. We’re especially pleased that our Head of Operations, Kim Warren, PhD, is able to share our manufacturing and operations experience with the gene and cell therapy community at ASGCT. The plato platform is designed to overcome many of the identified constraints and challenges of developing, manufacturing and commercializing gene therapies.”

Dr. Warren is an invited expert panelist at the Pre-approval Commercialization Workshop on “Manufacturing Challenges During Late Phase Development of Gene Therapy Products.” During a session on the latest advances in cell therapy research, AVROBIO’s Senior Director of Manufacturing Operations, Azadeh Golipour, PhD, will present “Automated Gene Modified Cell Production: Concept to Process Verification” which describes the development of the automated cell process.

  • Title: Manufacturing Challenges during Late Phase Development of Gene Therapy Products
  • Presenter: Kim Warren, PhD, AVROBIO
  • Date/time: Sunday April 28; 3 PM - 4:30 PM
  • Location: Jefferson West
  • Title: Automated Gene Modified Cell Production: Concept to Process Verification
  • Presenter: Azadeh Golipour, PhD, AVROBIO
  • Date/time: Tuesday April 30; 1:00 PM - 1:25 PM
  • Location: Monroe

In several oral presentations at ASGCT, preclinical data will be presented by AVROBIO’s academic collaborators and their colleagues, related to the Company’s gene therapy programs in Gaucher disease and cystinosis.

Gaucher disease ASGCT oral presentation and gene therapy program

An oral presentation will be made at ASGCT with preclinical data results related to the gene therapy program for Gaucher disease led by Stefan Karlsson, MD, PhD, Professor at the Division of Molecular Medicine and Gene Therapy at Lund University in Sweden. AVROBIO has received clearance for its clinical trial application (CTA) from Health Canada. The Company plans to initiate its GAU-201 Phase 1/2 clinical trial of AVR-RD-02 in patients with Gaucher disease in the second half of 2019 utilizing the plato platform.

  • Title: A Clinical Glucocerebroidase Lentiviral Vector Corrects the Pathology and Clinical Signs in a Mouse Model for Type 1 Gaucher Disease
  • Presenter: Stefan Karlsson, MD, PhD, Lund University, Sweden
  • Date/time: Monday April 29; 10:45 AM - 11:00 AM
  • Location: Heights Courtyard 2

Cystinosis ASGCT oral presentations and gene therapy program

Three oral presentations will be made at ASGCT related to the cystinosis gene therapy program led by Stephanie Cherqui, PhD, Associate Professor at the Department of Pediatrics at University of California, San Diego (USCD). In January 2019, AVROBIO announced that the U.S. Food and Drug Administration (FDA) had cleared the investigational new drug (IND) application for the planned investigator-sponsored Phase 1/2 clinical trial for AVR-RD-04 gene therapy in cystinosis to be conducted by UCSD. Rights are licensed to AVROBIO to develop AVR-RD-04 as a potential one-time gene therapy for the treatment of cystinosis.

  • Title: Toxicology Studies for Hematopoietic Stem & Progenitor Cell Gene Therapy for Cystinosis Revealed an Unexpected Cytotoxicity of Polybrene to the Ctns-/- Sca1+Cells
  • Presenters include: Jay Sharma, University of California, San Diego
  • Session: Nonclinical Studies and Assay Development
  • Date/time: Tuesday April 30; 4:30 PM - 4:45 PM
  • Location: Heights Courtyard 2
  • Title: HSPC Gene Therapy for Cystinosis Remains Effective in Patients Carrying the Large Deletion abolishing SHPK expression
  • Presenter: Spencer Goodman, University of California, San Diego
  • Date/time: Wednesday May 1; 4:45 PM - 5:00 PM
  • Location: Holmead
  • Title: Hematopoietic Stem Cell Gene Therapy and Lysosomal Cross Correction for Cystinosis
  • Presenter: Stephanie Cherqui PhD, University of California, San Diego
  • Date/time: Thursday May 2; 8:00 AM - 8:30 AM
  • Location: International Ballroom East

“We believe that Dr. Karlsson’s Gaucher mouse model proof-of-concept work shows multiple positive preclinical findings, including prevention and reversal of disease manifestations, as well as improvement trends in multiple bone parameters. Bone data are important because according to The National Gaucher Foundation bone problems are common in people living with Gaucher disease,” said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. “Stephanie Cherqui, PhD, and her team are also sharing valuable insights into ex vivo gene therapy for cystinosis. Both of these programs, for Gaucher and cystinosis, are expected to begin clinical studies in the second half of 2019.”

About AVROBIO, Inc.

AVROBIO, Inc., is a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose. AVROBIO’s lentiviral-based gene therapies employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert functional copies of the gene that is defective in the target disease. AVROBIO is focused on the development of its gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Company’s plato platform is a proprietary vector system and automated, closed cell manufacturing solution designed to support worldwide commercialization. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit www.avrobio.com.

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 such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,” “plans,” “possible,” “potential,” “seeks,” “will,” and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy, prospective products and goals, the therapeutic potential of our product candidates, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, the design, commencement, enrollment and timing of ongoing or planned clinical trials, and the timing, scope and likelihood of regulatory filings and approvals. 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 the date of this release 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 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. 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2018, 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.