CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a vision to free people from a lifetime of genetic disease, today hosted a panel of investigators managing the patient experience in the Phase 2 FAB-GT clinical trial of AVR-RD-01, an investigational one-time gene therapy for Fabry disease, at the 14th International Congress of Inborn Errors of Metabolism (ICIEM), Nov. 21-23, 2021, in Sydney, Australia. Other presentations included updated safety data from the first lentiviral gene therapy clinical trials for Fabry disease and Gaucher disease type 1, as well as 10- to 12-month safety data from gene therapy-treated mice with mucopolysaccharidosis type II (MPSII), or Hunter syndrome.

Chaired by Rob Wynn, M.D., (consultant pediatric hematologist at the Royal Manchester Childrens Hospital, UK), the AVROBIO sponsored symposium, Advancing the patient experience during ex vivo lentiviral gene therapy for lysosomal disorders, featured trial investigators Mark Thomas, M.D., (Royal Perth Hospital, WA, AUS; clinical professor at the University of Western Australia Medical School), Kathy Nicholls, M.D., (Royal Melbourne Hospital, VIC, AUS) and Ben Carnley, M.D., (Royal Perth Hospital, WA, AUS), who discussed managing the patient experience with ex vivo lentiviral gene therapy based on their involvement in the FAB-GT clinical trial.

During the panel discussion, the clinicians discussed the patient experience during apheresis (the process by which the patients stem cells are collected), conditioning (the process to clear space in the patients bone marrow to receive their stem cells back following introduction of the therapeutic transgene), and infusion of the genetically modified stem cells, as well as after receiving the gene therapy.

Its been exciting and rewarding to be part of this first-in-the-world clinical trial to bring this investigational gene therapy to the Fabry disease community here in Australia, said Dr. Nicholls. While the current standard of care has made an important difference in the lives of patients, there are limitations in the impact of enzyme replacement therapy (ERT) on the heart, kidney and central nervous system (CNS) manifestations of Fabry disease. For instance, we know that renal progression will continue even on a combination of ERT and other renal protective measures. Wed like this not to be the case, of course. We need better treatment options for these patients and families.

Nine patients have been dosed in the FAB-GT clinical trial to date, all of them in Australia. While the first three patients were treated using an academic vector and melphalan as a conditioning agent, the other six patients were treated using AVROBIOs proprietary plato gene therapy platform, which includes a state-of-the-art lentiviral vector, a personalized busulfan conditioning regimen designed to implement precision dosing and an automated, closed manufacturing process intended to deliver potent and consistent drug product from manufacturing sites worldwide at commercial scale.

From our perspective, every patient has a different story, so personalization is the best approach when discussing and providing treatment, added Dr. Thomas. Making sure that you explain every step of the process preferably several times and in sections to give patients and families time to fully understand the information has been an important part of preparing patients for gene therapy. Comprehensive patient support, provided by a dedicated care team, sets clear expectations and can help improve the overall patient experience.

Based on the patients with various lysosomal disorders I have treated, I believe lentiviral gene therapy has the potential to be transformative because it can deliver more enzyme to the brain, to soft tissues, like the heart, lungs and liver, as well as into the skeleton, commented Prof. Wynn, who is not an investigator in the FAB-GT trial. This trial discussed today and others like it help pave the way for ex-vivo lentiviral gene therapy approaches for lysosomal disorders. I encourage everyone to come with us as we embark and continue on this journey of changing from ERT, allogeneic transplant therapy and supportive care to ex-vivo lentiviral gene therapy approach using a patients own genetically modified hematopoietic stem cells.

Updated safety data show no adverse events or serious adverse events related to drug productAVROBIO also presented updated clinical and preclinical data from the companys pipeline of lysosomal disorder programs in one oral and two poster presentations.

Phase 1 and Phase 2 studies for Fabry disease

The Guard1 study for Gaucher disease type 1

Preclinical data for Hunter syndrome

About AVROBIOOur 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, and follow us on Twitter and LinkedIn.

Forward-Looking StatementThis 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 our business strategy for and the potential therapeutic benefits of our prospective product candidates, results of preclinical studies, the design, commencement, enrollment and timing of ongoing or planned clinical trials, clinical trial results, product approvals and regulatory pathways, the timing of patient recruitment and enrollment activities, and product approvals, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, and the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, including the use of a personalized and ultra-precision busulfan conditioning regimen. 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 AVROBIOs current expectations, estimates and projections about our industry as well as managements 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 AVROBIOs 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 AVROBIOs 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 AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs 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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AVROBIO Presents Clinician Experience with FAB-GT Clinical Trial and Updated Safety Data for Investigational Gene Therapies in Fabry Disease and...

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