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.