What are the different types of gene therapy?
Gene therapy can take several forms. The kind AskBio is working on is considered gene replacement therapy, because a new therapeutic gene is delivered to the patient’s cells; this is also called in vivo gene therapy. Another type of gene therapy is called ex vivo because cells from the patient’s blood or bone marrow are removed and grown in the laboratory.

What is AAV gene therapy?
AAV gene therapy refers to the specific virus vector – adeno-associated virus – most commonly used for in vivo gene therapy. AAV is a harmless virus bioengineered to carry a healthy gene to target cells in a patient with a genetic disease. Viruses are ideal vectors because their sole purpose is to infiltrate a host’s cells and overwhelm them with their own DNA. The adeno-associated virus is preferred over others because it doesn’t sicken patients or cause harmful inflammation.

Is gene therapy safe?
Therapies that use the AAV vector are considered generally safe because the virus doesn’t cause infection and produces only a mild inflammatory response in patients. However, some immune response, such as fever, chills, and fatigue, may occur shortly after treatment but typically resolve quickly.

What diseases can gene therapy treat?
AskBio is primarily focused on potential cures for neuro-muscular, central nervous and respiratory genetic disorders. Most diseases are a result of inherited traits such as muscular dystrophy or cystic fibrosis.  Others can result from various environmental factors later in life such as cancer or Parkinson’s disease.  The future of gene therapy holds the potential to also treat acquired conditions such as pain, aging, obesity and addiction. 

How is gene therapy delivered and how long does it last?
Gene therapy is typically delivered to patients via IV infusion in a clinical setting. In theory, gene therapy should only require one dose but in some cases, additional dosing may be necessitated.  In order to facilitate redosing of gene therapy, AskBio is currently working on solutions to isolate neutralizing antibodies that prevent expression of subsequent gene therapy treatments.