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AUM LifeTech, Inc., a Philadelphia-based preclinical stage biotech company, has received a Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) of the National Institutes of Health (NIH) to advance its preclinical program in lung cancer immunotherapy using a non-viral RNA-targeting gene therapy approach.
“This grant will help advance our preclinical immunotherapy program in lung cancer and provide us the next critical steps towards IND enabling studies,” said Veenu Aishwarya, founder and CEO of AUM LifeTech, Inc.
AUM LifeTech will use the funds to advance the development of its new form of cancer immunotherapy, which involves targeting the messenger RNA (mRNA) of a gene, FOXP3, and thereby inhibiting the function of immune cells, called T-regulatory (Treg) cells.
Tregs are known to have an immunosuppressive function and, in some instances, prevent the body from fighting cancer and lead to cancer progression. The functions of Tregs are critically dependent upon their expression of FOXP3.
AUM LifeTech’s FANA Antisense Oligonucleotide (FANA ASO) RNA silencing approach allows efficient and selective targeting of human FOXP3 at the RNA level, in a highly sequence-specific manner. By reducing the levels of FOXP3, the biological function of Tregs is inhibited, and many cancers can be eliminated.
Providing alternatives
AUM LifeTech’s therapeutic approach provides an alternative to conventional gene therapy using viruses.
The ability to modulate gene expression without viral vectors can be beneficial, for several diseases, without making permanent changes in the human genome. While there are FDA-approved RNA-targeting therapies, AUM LifeTech’s non-viral gene therapy approach could potentially become the first antisense oligonucleotide approach for cancer immunotherapy.
The study is being conducted in collaboration with Wayne Hancock, Chief of the Division of Transplantation Immunology at the Children’s Hospital of Philadelphia, who has been working in the cancer immunotherapy space for more than three decades.
“The United States continues to be at the forefront of biomedical innovation, technology, and business due to immigrant founders and leaders like Veenu, who leave no stone unturned and persevere even in the most challenging circumstances, especially during the COVID-19 period,” Hancock said.
“We have, slowly but steadily, generated very encouraging data showing the potency of our approach, including in humanized mice, and have identified several lead compounds that can eventually become clinical candidates. Considering our data, I am very optimistic that our approach will provide some much-needed advancements in the field of cancer immunotherapy.”
‘Potential game changer’
“FOXP3 is a therapeutically validated target; however, it is very challenging to directly target it with existing technologies, including small molecules or biologics,” said Steven Albelda, co-director, Translational Center of Excellence in Lung Cancer, at the Abramson Cancer Center and the Vice Chief of the Pulmonary, Allergy, and Critical Care Division at Perelman School of Medicine at the University of Pennsylvania.
“This seminal work targeting FOXP3 using AUM LifeTech’s RNA silencing technology has the potential not only for lung cancer therapy development but also for other advanced solid tumors. Their combination approach, using checkpoint inhibitors, could be a game-changer in the field of cancer immunotherapy.”
