Challenges with Conventional Gene Delivery
Conventional delivery of therapeutic genes to patients involves an injection directly into the bloodstream or tissue. This direct injection method has several barriers, including: 1) difficult and inefficient gene delivery to target cells; 2) unpredictable and quickly diminishing gene activity; and 3) adverse reactions from the patient's immune response to the vector, or "vehicle," used to deliver the genes.
Genteric's Gene Pill Approach
Researchers from Genteric have invented a new method of gene delivery that employs the patient's own gastrointestinal tract to produce and secrete therapeutic proteins into the bloodstream. In the therapy's mature stage, the patient could swallow a pill containing engineered DNA that encodes the needed protein. The DNA would then be "taken up" by the cells lining the luminal space of gastrointestinal tract (the side of the intestine that normally absorbs food material), which then produce and secrete proteins into the bloodstream to achieve the desired therapeutic results. A key feature of these intestinal cells is that, like skin cells, they are constantly replaced. Thus, the gene is expelled with the dying cells on a regular basis - about every other day. This natural feature enhances safety and efficient dose control.
This new oral gene therapy method presents the following advantages:
Oral administration is simple, painless and convenient.
Oral administration allows different dose levels to be given according to patient needs.
The body replaces cells at this site quickly, which gives greater control of gene delivery and helps to avoid long-term side effects.
Genteric's next steps are to continue current experiments and follow into Phase I/II clinical trials. During these stages, researchers will address challenges in oral delivery, such as methods to adjust the amount of insulin expressed and durability of coating materials used to protect the DNA as it is delivered to cells in the intestinal tract.