May 5, 2016 10:20am
The regulatory T cell response was observed, coupled with a stable expression at 2.5% of the target for therapy may indicate that AGTC has established functional immune tolerance to the AAV vector in the current trial; partial correction of certain biomarkers even at this reduced level has been seen.
Researchers from the University of Massachusetts Medical School today announced new data <at the American Society of Gene and Cell Therapy (ASGTC) Annual Meeting> 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.
Study results are being presented in a podium session at the American Society of Gene and Cell Therapy (ASGCT) 19^th 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.
The Bottom Line: The investigational gene therapy was developed AGTC who is conducting human clinical trials of gene therapies for the treatment of rare diseases. These findings provide encouraging support for continued efforts to develop a long-term therapeutic approach to AAT deficiency.
AGTC closed at $14.69 and is DOWN -$0.16 to $14.53. Pre earnings, we’ve seem most financial results announcers “dump” pricing, AGTC is opening the earnings “kimono” on Monday, May 9th at 5 pm.