SEOUL, South Korea, Oct. 19 /PRNewswire/ -- 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.
About Age-Related Macular Degeneration (AMD)
A leading cause of vision loss and blindness, AMD is a disease associated with aging that gradually destroys sharp, central vision. According to the National Eye Institute, age-related macular degeneration affects more than 1.75 million individuals in the United States alone. This number is expected to increase to almost three million by 2020.
About BioMolecular Therapeutics, Inc. (www.bmtlab.co.kr)
An early-stage biopharmaceutical company, BMT is pioneering the development of novel RNAi therapeutics to prevent and treat age-related macular degeneration based on the company's proprietary technology, asymmetric shorter-duplex siRNA (asiRNA). This novel RNAi technology significantly reduces deleterious side effects triggered by conventional siRNA, especially, the stimulation of the innate immune system, which makes it feasible to use asiRNA for the treatment of age-related macular degeneration, a disease to which RNAi therapeutics could not be developed using conventional siRNAs. Other potential applications for BMT's technology include therapeutics targeting infectious disease, such as herpes simplex virus (HSV), and cancer. A privately held company based in Suwon, South Korea, BMT was established in 2010.
SOURCE BioMolecular Therapeutics, Inc.