News Column

New Gene Therapy Findings from University of Montreal Reported

June 26, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Biotechnology have been presented. According to news reporting out of Chicoutimi, Canada, by NewsRx editors, research stated, "Cellular immune responses to adeno-associated viral (AAV) vectors used for gene therapy have been linked to attenuated transgene expression and loss of efficacy. The impact of such cellular immune responses on the clinical efficacy of alipogene tiparvovec (Glybera; AAV1-LPLS447X; uniQure), a gene therapy consisting of intramuscular administration of a recombinant AAV1 mediating muscle-directed expression of lipoprotein lipase (LPL), was investigated."

Our news journalists obtained a quote from the research from the University of Montreal, "Five subjects with LPL deficiency (LPLD) were administered intramuscularly with a dose of 1x10(12) gc/kg alipogene tiparvovec. All subjects were treated with immune suppression starting shortly before administration of alipogene tiparvovec and maintained until 12 weeks after administration. Systemic antibody and T cell responses against AAV1 and LPLS447X, as well as local cellular immune responses in the injected muscle, were investigated in five LPLD subjects. Long-term transgene expression was demonstrated despite a transient systemic cellular response and a stable humoral immune response against the AAV1 capsid protein. Cellular infiltrates were found in four of the five subjects but were not associated with adverse clinical events or elevation of inflammation markers. Consistent herewith, CD8+ T cells in the infiltrates lacked cytotoxic potential. Furthermore, FoxP3+/CD4+ T cells were found in the infiltrates, suggesting that multiple mechanisms contribute to local tolerance. Systemic and local immune responses induced by intramuscular injection of alipogene tiparvovec did not appear to have an impact on safety and did not prevent LPL transgene expression."

According to the news editors, the research concluded: "These findings support the use of alipogene tiparvovec in individuals with LPLD and indicate that muscle-directed AAV-based gene therapy remains a promising approach for the treatment of human diseases."

For more information on this research see: Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy. Human Gene Therapy, 2014;25(3):180-188. Human Gene Therapy can be contacted at: Mary Ann Liebert, Inc, 140 Huguenot Street, 3RD Fl, New Rochelle, NY 10801, USA. (Mary Ann Liebert, Inc. -; Human Gene Therapy -

Our news journalists report that additional information may be obtained by contacting V. Ferreira, University of Montreal, Chicoutimi Hosp, Clin Res Center, Dept. of MedECOGENE 21, Chicoutimi, PQ G7H 7P2, Canada. Additional authors for this research include J. Twisk, K. Kwikkers, E. Aronica, D. Brisson, J. Methot, H. Petry and D. Gaudet (see also Biotechnology).

Keywords for this news article include: Biotechnology, Quebec, Canada, Lipids, Chicoutimi, Gene Therapy, Lipoproteins, Bioengineering, North and Central America, Clinical Trials and Studies

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Source: Gene Therapy Weekly

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