Healthcare Industry News:  cell therapy 


 News Release - May 5, 2016

Investigational Gene-Based Therapy for Alpha-1 Antitrypsin (AAT) Deficiency Demonstrates Durable Response at Five Years

Research led by scientists from the University of Massachusetts Medical School presented at the American Society of Gene and cell therapy (ASGTC) Annual Meeting

WASHINGTON, May 05, 2016 -- (Healthcare Sales & Marketing Network) -- Researchers from the University of Massachusetts Medical School today announced new data evaluating the efficacy of an investigational adeno-associated virus (AAV) vector gene therapy treatment for alpha-1 antitrypsin (AAT) deficiency, an inherited genetic defect that results in severe loss of lung function. The investigational gene therapy was developed by Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of gene therapies for the treatment of rare diseases. Study results were presented in a podium session at the American Society of Gene and cell therapy (ASGCT) 19th Annual Meeting, taking place from May 4 – 7.

The abstract titled “Sustained Expression with Partial Correction of Neutrophil Defects 5 Years After Intramuscular rAAV1 Gene Therapy for Alpha-1 Antitrypsin Deficiency,” describes a multicenter study evaluating five-year follow-up results of a one-time intramuscular injection of a recombinant AAV-AAT vector in patients with AAT deficiency. Researchers reported that expression of therapy-derived AAT is present at steady levels in the serum and injected muscle five years after dosing. Additionally, study investigators noted a measurable regulatory T cell response to the gene therapy vector that contributed to the stable gene expression, despite the presence of an immune response directed against the vector. The group concluded that stable levels of AAT may exert beneficial effects despite serum levels below the threshold of what is traditionally considered therapeutic and may be used in the future to improve biomarkers and bioassays indicative of disease in this population.

“These findings provide encouraging support for continued efforts to develop a long-term therapeutic approach to AAT deficiency,” said lead study investigator Terence R. Flotte M.D., Dean of the School of Medicine, Provost and Executive Deputy Chancellor, and The Celia and Isaac Haidak Professor of Medical Education at the University of Massachusetts Medical School. “The regulatory T cell response we are observing, coupled with a stable expression at 2.5% of the target for therapy may indicate that we have established functional immune tolerance to the AAV vector in the trial, particularly since we are seeing partial correction of certain biomarkers even at this reduced level.”

“We appreciate the efforts of our collaborators for their commitment to advancing the clinical potential of gene therapy approaches to AAT deficiency and other inherited diseases," said Sue Washer, CEO of AGTC. “We are pleased with these results, underscoring previous findings showing that AAV vectors made using AGTC's proprietary manufacturing method are able to achieve durable sustained expression of the AAT protein in serum and muscle following one-time treatment.”

About the University of Massachusetts Medical School

The University of Massachusetts Medical School in Worcester is a world-class research institution, consistently producing noteworthy advances in clinical and basic research. Researchers at UMMS have made pivotal advances in HIV, cancer, diabetes, infectious disease, and in understanding the molecular basis of disease. Programs and centers of international distinction include those devoted to RNA therapeutics; gene therapy; gene function and expression; systems biology; and ALS.

About AGTC

AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC's lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function, and which currently lack effective medical treatments.

AGTC's product pipeline includes six named ophthalmology development programs across five targets (X-linked retinoschisis, X-linked retinitis pigmentosa, achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and early research studies in additional indications. AGTC employs a highly targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need, clinical feasibility and commercial potential. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as, expertise in the formulation, manufacture and physical delivery of gene therapy products.

Forward Looking Statements

This release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs. These statements relate to a variety of matters, including but not limited to, the anticipated utility of AAV vectors made using AGTC's proprietary manufacturing method in the treatment of AAT deficiency. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors, which include, but are not limited to, the following: success in animal studies or early clinical trials may not be indicative of results obtained in later trials, no gene therapy products have been approved in the United States and AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate; AGTC relies on third parties to conduct, supervise and monitor its clinical trials and to conduct certain aspects of its product manufacturing and protocol development; and increased regulatory scrutiny of gene therapy, we may find it difficult to enroll patients in our clinical trials, which could delay or prevent clinical trials of our product candidates, and genetic research could damage public perception of AGTC's product candidates or adversely affect AGTC's ability to conduct its business. Additional factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Item 1A—Risk Factors" in AGTC's Annual Report on Form 10-K for the fiscal year ended June 30, 2015, as filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions and beliefs only as of the date of this release. Except as required by law, AGTC assumes no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

Source: Applied Genetic Technologies

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