Solving the Delivery Challenge
The field of gene and RNA therapy holds great promise. However most of these promises have been unfulfilled due to challenges in developing a system capable of delivering genetic and therapeutic payloads into targeted cells efficiently, reproducibly and permanently.
While other companies are trying to identify suitable delivery options and methods for their RNAi payloads, VIRxSYS has already successfully designed and tested in the clinic its lentiviral vector technology platform for RNA molecule delivery. VIRxSYS believes that the combination of the SMaRT™ RNA platform with its highly engineered viral vector-based nano-particle delivery platform will make a dominating combination in RNA therapy, potentially transforming the technology space. VIRxSYS is also using its expertise and technology in lentiviral vectors in vaccine delivery.
Visit our Publications page under the Resources tab above to see publications in top tier medical journals on our lentiviral technology.
Vectors
Vectors are vehicles that deliver genetic material into a cell’s nucleus. Viruses are very effective at doing this, so they frequently are used as vectors. Viral-based vectors use the backbone of viruses for this purpose. After removing all disease-causing elements from the virus, it leaves behind a natural delivery vehicle. VIRxSYS' HIV-based lentiviral vector platform has achieved this goal and has also overcome other challenges, such as high-efficiency gene transfer and stable transfer of genetic material into dividing and non-dividing cells.
Lentiviral Vectors
Lentiviral vectors are one of the most effective delivery systems for RNA-based therapies and can be used as highly effective vaccines, as demonstrated by preclinical data collected for our HIV vaccine candidate, VRX1273. Over the past 10 years, VIRxSYS has become the world’s leader in the design, development and manufacturing of lentiviral vectors for clinical applications. VIRxSYS also has the largest database of patient safety data on subjects who have participated in clinical trials using human lentiviral vectors. The Company’s lentiviral vector platform, which was the backbone of the vector used in the Company’s Phase II clinical trials, is based on technology first developed by and exclusively licensed from The Johns Hopkins University. This vector platform is based on the “lentivirus” (LV) class of virus which has remarkable properties for safe, efficient, predictable, and sustained delivery of payloads into cells.
Payloads delivered via a lentiviral vector generally have long staying power within a cell, and have been delivered in vivo repeatedly in non-human primates with no treatment-related serious adverse events. LVs have high delivery efficiency among viral-based vector delivery systems.
VIRxSYS’ lentiviral vector system has demonstrated a strong safety profile in clinical trials for the Company’s autologous cell therapy, Lexgenleucel-T which utilizes VRX496TM. That product candidate has more than 265 patient-years of data (as of December, 2010) with no treatment-related serious adverse events reported.
VIRxSYS’ vector delivery platform may be useful in therapies against viral infections, genetic disorders, cardiovascular diseases and others. The applications include vaccines, autologous ex-vivo cellular RNA immunotherapy, and direct injectable therapies. VIRxSYS has in vivo proof of principle data for each of these application areas.
The Company also is using and developing other viral and non-viral based vector systems to specifically tailor the delivery of the SMaRTTM technology, taking into account the unique biology of targeted tissues and organs.