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20 October 2010

BioMolecular Therapeutics, a new global player in RNAi therapeutics, launches company and adds key advisors

- Dr. Jayakrishna Ambati, prominent expert in ophthalmology, and Dr. Johannes Fruehauf, specialist in development of RNAi therapeutics, join BMT's scientific advisory committee and board of directors, respectively
- BMT to present poster at the 6th Annual Meeting of the Oligonucleotide Therapeutics Society
SEOUL, South Korea -- BioMolecular Therapeutics, Inc. (BMT, Inc.), a new Seoul-based RNAi therapeutics company with a proprietary technology designed to reduce significantly deleterious side effects triggered by conventional siRNA, announced that it has launched its global operations. The company is currently developing a novel RNAi therapeutic for age-related macular degeneration (AMD), based on its asymmetric shorter-duplex siRNA (asiRNA) technology, originally published in Molecular Therapy in 2009 as a featured article. Early studies suggest that the asiRNA technology will allow the application of RNAi therapeutics in fields where innate immune stimulation needs to be avoided, such as AMD. The goal of an RNAi therapeutic is to target specifically only the gene or genes implicated in the disease. In AMD, activation of the innate immune response could elicit off-target effects, potentially hastening the progression of the disease. Two previous clinical trials using traditional siRNA as an AMD therapeutic were terminated at Phase II and III because of unintended activation of the patients' immune response.
BMT's leadership team includes Dong-ki Lee, Ph.D., the inventor of the company's proprietary technology, and Sun woo Hong, Ph.D., BMT's founding CEO. Dr. Dong-ki Lee is associate professor of chemistry at Sungkyunkwan University in Suwon, Korea, and the recipient of the prestigious Global Research Laboratory grant from the Korean government for the development of novel RNAi therapeutics. Dr. Sun Woo Hong has strong expertise in siRNA and genomics research, and was research professor at Dongguk University in Seoul, Korea. The company has also named two key advisors, Jayakrishna Ambati, M.D., ophthalmologist and professor at University of Kentucky Medical School, and Johannes Fruehauf, M.D., Ph.D., an experienced biotechnology executive and a specialist in RNAi therapeutics.
Dr. Ambati will serve as head of BMT's scientific advisory board. He is an authority in ophthalmology and well-known for his seminal contributions in macular degeneration research. His lab made numerous foundational discoveries published in Nature, Nature Medicine, PNAS, and the Journal of Clinical Investigation; these include exceptional findings about the role of the innate immune system in the mechanisms and treatment of AMD. Dr. Ambati won the 2010 Cogan Award from The Association for Research in Vision and Ophthalmology (ARVO) for his contributions in macular degeneration research. "We are extremely honored that Dr. Ambati has agreed to join BMT as chair of our scientific advisory board," said BMT CEO, Sun Woo Hong. "He brings unparalleled expertise and experience in eye disease research, an area so fundamental to our success. We expect that the addition of his expertise will accelerate and strengthen our progress to develop RNAi therapeutics for AMD using our proprietary asiRNA platform."
Dr. Fruehauf has been appointed as the first external director to BMT's board. He is currently vice president of R&D at Aura Biosciences, and founder and president of ViThera Laboratories. Previously, he was a cofounder and VP of research at Cequent Pharmaceuticals, now Marina Biotech, where he oversaw the development from bench into clinical trials of the first oral RNAi therapeutic, CEQ508. Dr. Dong-ki Lee commented, "With Dr. Fruehauf joining our board, we are entering the next phase of BMT's growth. His expertise in building and managing early-stage biotech startup companies and achieving scientific progress towards key milestones is invaluable for our next steps: establishing business relationships and scientific alliances in the United States, and internationally."
The company is making its inaugural U.S. poster presentation at the 6th Annual Meeting of the Oligonucleotide Therapeutics Society, which begins tomorrow in Dana Point, California. The title is Reduced Innate Immune Stimulation by asiRNA Structure: Expanding Therapeutic Application of siRNA.

**Published in "First Word"

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