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Philadelphia-based Biotech Company AUM LifeTech Awarded $400,000 NCI/NIH Grant to Advance Their Lung Cancer Immunotherapy Program Using RNA-Targeting Gene Therapy with Checkpoint Inhibitors

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Philadelphia-based Biotech Company AUM LifeTech Awarded $400,000 NCI/NIH Grant to Advance Their Lung Cancer Immunotherapy Program Using RNA-Targeting Gene Therapy with Checkpoint Inhibitors | Pharmtech Focus

AUM LifeTech, Inc., a Philadelphia-based preclinical stage biotechnology 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 their preclinical program in lung cancer immunotherapy using a non-viral RNA-targeting gene therapy approach. The federal funding in the amount of US $400,000 is funded under the award number R43CA275470.

“As an immigrant from India and a newly minted US citizen, I am excited that we were selected for this highly competitive award by the National Cancer Institute of NIH. 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 Chief Executive Officer of AUM LifeTech, Inc.

AUM LifeTech will use the funds to advance the development of their new form of cancer immunotherapy that 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.

“We have made significant progress over the past five years and are ready to continue our work toward an applicable therapy for patients. I would like to thank our collaborator Dr. Wayne Hancock, for his trust in me to lead this program. Dr. Hancock is a globally recognized immunologist; his extraordinary scientific leadership and guidance have been critical and valuable for our continued success as we move into our next phase of development. We look forward to advancing our combination approach for immunotherapy targeting both FOXP3 RNA (using self-delivering FANA ASOs) and additional proteins (with checkpoint inhibitors),” added Aishwarya.

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 Dr. 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 over three decades. Dr. Hancock is also a Professor of Pathology and Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania.

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,” said Dr. Hancock. “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,” he added.

“FOXP3 is a therapeutically validated target; however, it is very challenging to directly target it with existing technologies, including small molecules or biologics. 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,” said Dr. 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. The Hospital of the University of Pennsylvania (HUP) is a leading lung cancer surgery hospital in the United States and offers one of the world’s premier lung transplantation programs.

The collaborative project was established through a scientific networking activity at the American Association for Cancer Research (AACR) annual meeting in 2017. “This demonstrates a perfect example of industry-academia synergy leading to advancement of cancer research and therapy development. I am pleased that AACR could help facilitate such a collaboration. I commend the strong perseverance by innovators and entrepreneurs like Veenu Aishwarya and his strong desire to end cancer as we know it. We wish him the very best in his drug development journey.” said Mitch Stoller, Vice President of Development for AACR.  Philadelphia-based AACR is the world’s first and largest professional association dedicated to cancer research, with more than 52,000 members in 130 countries. Stoller also said, “We congratulate Veenu Aishwarya for being selected for this NCI/NIH grant and for all his efforts to advance biomedical research and cancer therapy development using innovative RNA-targeting technology.”

AUM LifeTech has been headquartered in Philadelphia since its inception and hopes to continue to expand its footprint in the city as it continues to become one of the leading global centers for cell and gene therapy drug development.

“The City’s Department of Commerce is proud to support immigrant entrepreneurs, and healthcare business leaders like Veenu Aishwarya who are advancing Philadelphia’s innovation economy with biotechnological breakthroughs,” said Commerce Director, Anne Nadol.Philadelphia is one of the fastest-growing medical hubs in the country expanding the job market and attracting many industry-leading companies. Congratulations to Aishwarya for his transformative leadership at AUM LifeTech to collaboratively advance the development of a lung cancer immunotherapy with NCI/NIH small business innovation research grant.”

As Commerce Director, Nadol oversees the City’s economic development efforts and advocates for strategic policies that support the growth of small businesses along with the attraction and retention of major companies.

“The Chamber of Commerce for Greater Philadelphia congratulates Veenu Aishwarya for being selected for NIH funding to further advance AUM LifeTech’s immuno-oncology preclinical pipeline using a validated non-viral gene therapy technology. This is a steppingstone for AUM LifeTech and will help attract future collaborations and alliances and further advance their drug development programs. We also welcome AUM LifeTech as our newest member of the Chamber and look forward to working with Veenu and his team to achieve our shared vision of the region being recognized as a global life sciences leader,” said Claire Greenwood, Senior Vice President of Economic Competitiveness, The Chamber of Commerce for Greater Philadelphia.

AUM LifeTech started at the University City Science Center’s Port Business Incubator and is currently located at the CIC Labs+ Innovation Campus in Philadelphia. CIC is one of the leading providers of life science office and lab space for fast growing startup companies. “I am happy to see that under Veenu’s extraordinary leadership, especially during these challenging times, a small business-like AUM LifeTech is being recognized and being awarded funding to advance the critical development of therapeutic cancer treatments. NCI, the largest funding organization for cancer research in the world, has the most selective and competitive vetting process. This federal grant provides both credibility and validation to AUM LifeTech’s breakthrough science and technology. CIC is proud to be a part of Veenu and AUM LifeTech’s journey,” said Dr. Michelle Ottey, Managing Director, CIC Labs+ Innovation Campus Philadelphia.

Before founding AUM LifeTech, Veenu Aishwarya received his biomedical research training under Dr. Alan Gewirtz at the University of Pennsylvania School of Medicine. “I would also like to dedicate this grant to Dr. Alan Gewirtz, a scientific leader in this field, and my mentor, who passed away due to lung cancer in 2010,” added Aishwarya.

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