Gene therapy is only possible with the use of vectors for safe and protected delivery to the target cells. Currently viral vectors such as lentivirus, adenovirus and associated adenovirus (AAV) are the main vectors used. There are a few drawbacks of using viral vectors and one of them is the natural immunity that most people have due to natural infections. This typically means that clinical efficacy can only be achieved injecting relatively high amounts of the vector. It would be beneficial for both safety and efficacy if less viral vector would be necessary leading to the same clinical efficacy.
This is where Nuvec® can help.
Our gene therapy research
N4 pharma have recently completed studies to infect human breast cancer cells (MCF-7). The work showed that the addition of both bare silica and Nuvec® increase the transfection efficacy significantly and indicate that much less AV is needed in combination with Nuvec® to achieve the same results than adenovirus alone.
A further important use of viral vectors is in ex-vivo gene therapy, In separate research, N4 Pharma has demonstrated that Nuvec® is capable of a 20-30 fold increase in the efficiency using LV alone. The use of viral vectors is a key element of the very high cost associated with ex-vivo gene therapy. These results show Nuvec® can be used to make an increased amount of a patients’ genes using a fraction of the original lentivirus thereby making this process safer and more efficient.
N4 Pharma’s programme of work with AAV is ongoing.
To see our proof of concept data contact us here.