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ASGCT News: Siren Biotechnology Hails Universal Gene Therapy for Oncology genengnews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from genengnews.com Daily Mail and Mail on Sunday newspapers.
Ambys Medicines is a biotechnology company focused on discovering and developing regenerative and restorative therapies. Founded by Holger Willenbring, Juan Carlos Izpisua Belmonte, Markus Grompe, and Martin Burke in 2018, Ambys Medicines is backed by Takeda, Alexandria Venture Investments, Takeda Ventures, Inc., Schroders Capital, and Third Rock Ventures and is headquartered in South San Francisco. ....
Ambys Medicines wants to go after diseases impacting the liver, a key regenerative organ, with cell therapies. The Takeda-partnered biotech snagged $47 million from the Japanese Pharma and venture shops to test its asset in human trials in 2023. ....
New approach to gene therapy uses common pain reliever to fight against genetic diseases Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science Translational Medicine describes how the novel technique successfully treated the blood-clotting disorder hemophilia and the debilitating metabolic disease known as phenylketonuria, or PKU, in mice. The approach uses a benign lentivirus to both correct disease-causing mutations and to insert a new gene that makes liver cells immune to the potentially toxic effects of acetaminophen. The latter enables treated cells to survive and kills off untreated cells when they re repeatedly exposed to the over-the-counter medication that s also known by the brand name Tylenol. This allows the treated cells to multiply and creates a greater portion of liver cells being corrected. ....