Applied Genetic Technologies (AGTC) study shows results of gene transfer achieves sustained expression and a potential treatment option - BUY

January 20, 2016 7:14am

Data evaluating novel AAV-based gene therapy as a potential treatment for Alpha-1 Antitrypsin (AAT) Deficiency

Show that vector DNA persists at high levels for 12 months after one-time intramuscular injection and directs sustained expression of AAT protein

AGTC announced data evaluating the use of recombinant AAV-AAT vector gene delivery to muscle in patients with alpha-1-antitrypsin (AAT) deficiency, an inherited genetic disorder that results in severe loss of lung function and is caused by complete or partial deficiency of the alpha-1 antitrypsin protein.

Results were published online in the peer-reviewed journal Human Gene Therapy and will appear in the January print issue of the journal.

In this study, 3 cohorts of 3 subjects each had received rAAV1-AAT by intramuscular injection at doses of 6 × 1011 (low), 1.9 × 1012 (mid), or 6 × 1012 (high) vector genomes per kilogram of body weight. Muscle biopsies at the injection site were performed at 3 months after vector injection in all 9 subjects and at 12 months after vector injection in 8 of the 9 subjects enrolled in the trial. Samples of DNA obtained from these biopsies were characterized in depth by investigators at the Children’s Hospital of Philadelphia.

The Bottom Line: Important supporting data demonstrates sustained expression of protein (and dose response) through administration to muscle tissue which is important for systemic applications. 

Study results show that <AAV >vector DNA persists at high levels for 12 months after one-time intramuscular injection and directs sustained expression of AAT protein … currently, individuals affected by pulmonary manifestations of AAT deficiency are frequently treated with weekly intravenous infusions of AAT derived from donated human plasma, a procedure that is inconvenient and cost prohibitive,” said Philip R. Johnson, M.D., corresponding study author.

“P2 human studies evaluating gene transfer as a potential treatment option have been encouraging and these data further suggest that a one-time, AAV-based intramuscular AAT vector injection can have a durable response and direct sustained expression of the AAT protein.”

AGTC closed DOWN -$1.13 – Oversold, BUY