AUM LifeTech Develops A New Approach To Combat HIV By Using FANA RNA Silencing Technology To Inhibit HIV Replication
Data also presented for neurodegenerative disorders and prostate cancer studies at the annual Oligonucleotide Therapeutics Society Meeting in Montreal, Canada
PHILADELPHIA, Oct. 11, 2016 /PRNewswire/ -- US based biotechnology company 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 recent annual Oligonucleotide Therapeutics Society meeting (OTS) held in Montreal, Canada. OTS is a premier scientific conference in the field of nucleic acid therapeutics. "Delivery has always been a major challenge for the development of nucleic acid therapeutics (the therapies that work at the genetic level), be it the Nobel Prize winning strategy of RNA interference or the more recent breakthrough of CRISPR based genome editing. 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.
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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 she showed that AUM LifeTech's 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.
In addition to HIV studies, new data presented at the conference demonstrated that AUM LifeTech's next-generation FANA RNA silencing technology can potentially be used for therapeutic development for neurodegenerative disorders and prostate cancer. AUM LifeTech is also exploring applications of its technology for diseases like Alzheimer's and psoriasis.
In a study from University College London, UK, AUM's collaborators presented the high specificity and efficacy of AUM's FANA technology for allele specific silencing in certain degenerative disorders of the nervous system (neurodegenerative disorders). This study for hereditary sensory neuropathy type 1 was done in collaboration with Dr. Francesco Muntoni. Dr. Muntoni is a Chair of Paediatric Neurology at University College London's Institute of Child Health and has lead several clinical trials for rare diseases. "We work on several neuromuscular genetic diseases affecting children with a focus on gene discovery and translational applications. The self-delivery aspect of AUM's FANA technology is very beneficial especially for RNA silencing and regulation studies. We see FANA RNA silencing and regulation technology as a great research tool with a huge potential to be used in pre-clinical and potentially in future clinical application across a wide spectrum of genetic diseases," commented Dr. Muntoni.
New data was also presented on prostate cancer using AUM's technology. In another study done in collaboration with Dr. John Lewis, the Frank and Carla Sojonky Chair in Prostate Cancer Research at the University of Alberta, Canada they regulated the progression of human cancer metastasis using AUM's FANA antagomirs against a panel of miRNAs. The team performed an in vivo whole human miRNAome screen for miRNAs that mediate key steps in metastasis. They identified miRNAs that regulate discrete but overlapping pathways that drive tumor cell invasion. The group showed that gymnotically delivered (self-delivered) FANA antagomirs targeted against miR130b reduced cancer cell motility and blocked cancer cell attachment to vascular walls in vivo. "Metastasis is what kills prostate cancer patients, and considering this new encouraging data I am excited about the possibility of using AUM's FANA antisense technology to learn more about the key pathways that regulate prostate cancer progression, and to develop new drugs to block the spread of prostate cancer," stated Dr. Lewis.
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, Pennsylvania, USA at the University City Science Center's Port business incubator. For more information visit: www.aumlifetech.com.
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