Philadelphia’s Acuity Pharmaceuticals has planned for this fall the first-ever human tests of RNA interference (RNAi), in which small pieces of RNA are used to turn off disease-causing genes. Acuity aims to test RNAi’s mettle as a treatment for a severe form of age-related macular degeneration, which is a common cause of adult blindness.
A heart-failure drug that will be marketed specifically to African Americans did so well in late-stage human tests that the tests were halted early so that volunteers in the control group could take the drug too. One of a handful of new “ethnic drugs” (see “Genes, Medicine, and the New Race Debate,” TR June 2003), the heart-disease treatment could be on the market a year ahead of schedule, says Michael D. Loberg, CEO of Lexington, MA-based Nitromed, the drug’s developer.
Genetically engineering crops poses no unique health risks, concludes a report by a committee of the National Academy of the Sciences. The committee urged that the safety of all new foods – including those produced through conventional breeding – be evaluated on a case-by-case basis.
Researchers at the Chinese University of Hong Kong and Boston University have found a way to detect genetic defects in fetuses by testing their mothers’ blood. The technique, which could spare pregnant women invasive tests like amniocentesis, uses chip-based mass spectrometry technology from San Diego’s Sequenom to analyze the minute amounts of fetal DNA that circulate in the maternal bloodstream.
The U.K. Human Fertilisation and Embryology Authority has granted researchers at the nonprofit Centre for Life in Newcastle, England, a license to use cloning techniques to create human embryos (below) and harvest stem cells from them. Though such cells could eventually help treat ailments such as Parkinson’s disease and heart failure, those created under the license – the first of its kind in the United Kingdom – will be used for research only.
While other biotech firms were pulling planned IPOs and slashing their offering prices, Valencia, CA-based MannKind raised $87.5 million – more than it had originally projected. The biopharmaceutical company’s lead product, an inhaler that delivers powdered insulin, is undergoing human testing.