Data presented at the annual Oligonucleotide Therapeutics Society Meeting in Montreal, Canada
PHILADELPHIA, Oct. 5, 2016 /PRNewswire/ -- AUM LifeTech, Inc. with collaborators at the Beckman Research Institute at the City of Hope in California and McGill University in Canada has developed a new approach to combat HIV. Encouraging data showing the inhibition of HIV replication using AUM LifeTech's FANA antisense technology was presented at the annual Oligonucleotide Therapeutics Society meeting (OTS) in Montreal, Canada last week. OTS is a premier meeting in the field of nucleic acid therapeutics. "Delivery has always been a major challenge for the development of nucleic acid therapeutics, be it the Nobel Prize winning strategy of RNA interference or the more recent breakthrough of CRISPR based genome editing at the DNA level. The ability of AUM's FANA antisense oligonucleotides to be self-delivered in cellular and in vivo models without the use of any conjugates, carriers or formulations make them very attractive for development of nucleic acid therapeutics or more precisely RNA silencing therapeutics for a wide spectrum of genetic diseases. In this particular study we succeeded in effectively targeting HIV RNA and saw very promising preliminary results. FANA technology can also be used to target other viruses at the genetic level," stated Veenu Aishwarya, Founder and Chief Executive Officer of AUM LifeTech.
This work was done in collaboration with Dr. John Rossi who is a Lidow Family Research Chair and Morgan & Helen Chu Dean's Chair of the Irell & Manella Graduate School at Beckman Research Institute at City of Hope in California. Dr. Rossi is a distinguished scientist working on development of RNA targeting therapies for HIV and his expertise is globally recognized. "I am very excited about these developments. This is the very first time that FANA antisense oligos have been shown to target viral RNA. The ability of AUM's FANA antisense oligonucleotides to efficiently silence or regulate different types of RNAs including mRNA, miRNA, long non coding RNA and viral RNA is remarkable. Especially the self-delivery capability of FANA oligonucleotides is very promising for targeting HIV RNA. Importantly, in this study, no toxicity in primary PBMCs was observed. One of the major advantages of the FANA antisense strategy is that it is able to avoid viral escape and can inhibit multiple viral protein expression. Our initial data is very encouraging and we are now working on pre-clinical development. We have come a long way in the field of RNA silencing and such innovative technologies give us a lot of hope for the future of RNA based medicine," commented Dr. Rossi. "AUM LifeTech's FANA antisense technology should also be effective against other viruses such as Zika and Ebola," he added.
The lead author of the talk, Dr. Mayumi Takahashi, was awarded the Dr. Alan Gewirtz Award Scholarship for this work at the OTS meeting. In her oral presentation entitled "Inhibition of HIV-1 by gymnotically delivered 2'-deoxy-2'-fluoro-D-arabinonucleic acid modified antisense oligonucleotides (2'-FANA ASOs)" Dr. Takahashi showed that FANA ASOs can effectively target HIV by self-delivering (free uptake; gymnosis) into primary peripheral blood mononuclear cells (PBMCs) taken from different patient donors without causing immune response and toxicity. Along with impressive IC50 values the data also showed potent inhibition of HIV for two weeks by a single dose. Dr. Takahashi also presented important and interesting mechanistic data for this approach to inhibit HIV replication. The Dr. Alan Gewirtz Memorial Award Scholarship is given as a tribute to Dr. Gewirtz who was a pioneer in the field of antisense therapeutics and one of the co-founders of Oligonucleotide Therapeutics Society. Dr. Gewirtz worked at the University of Pennsylvania School of Medicine in Philadelphia and had made extraordinary contributions to the field of Nucleic Acids Therapeutics.
Inventor of FANA technology and AUM LifeTech's founding scientific advisory board member Dr. Masad Damha said "FANA technology is a chemistry with noteworthy properties and we are very happy to see this encouraging data. High stability, resistance to nucleases, specificity, potency and efficacy of FANA antisense oligonucleotides make them an ideal approach for such studies." Dr. Damha, a leader in this field, is globally known for his pioneering work on nucleic acids chemistry and is a James McGill Professor and Chair of the Department of Chemistry at McGill University in Canada.
Considering the benefits, cost effectiveness and ease of use of FANA RNA silencing and regulation technology, AUM LifeTech has also recently made it available to the biomedical community for research use. "In addition to our therapeutic goals, we would like to help the basic and translational research community all over the world. We hope that our FANA technology will save biomedical scientists significant amount of time, resources and money and help them to advance genetic research at a much faster rate," added Veenu Aishwarya founder and CEO of AUM LifeTech.
About AUM LifeTech, Inc.: At AUM LifeTech we are taking important steps towards development of personalized medicine at the genetic level. We are working on the development of next generation RNA silencing and regulation therapeutics for a wide spectrum of genetic diseases. In addition, AUM is also making its RNA silencing FANA technology available to scientists and researchers for basic and translational research. For research use, AUM LifeTech provides FANA antisense oligonucleotides for RNA silencing and regulation studies through its subsidiary AUM BioTech. AUM LifeTech is headquartered in Philadelphia, PA at the University City Science Center's Port business incubator. For more information visit: www.aumlifetech.com