Sangamo’s Study Of Genome Editing In Rare Disease Is First To Target Effects On Humans
Biotech company Sangamo Therapeutics, headquartered in Richmond, Calif., is expected to release preliminary results next month in the first clinical trial using genome editing to treat real patients. In other health research news: a banking and regulatory adviser works to get private investors to bankroll a cure for blindness; the "perfect lab animal"? worms; and drug comparative effectiveness research.
Nine Things To Know About Sangamo And Its First-In-Human Genome Editing Study
The first clinical trial using genome editing to treat real patients is nearing an early but crucially important data reveal. Until now, the only data available involving the removal or repair of disease-causing DNA have come from experiments in test tubes and animals. But late last year, the biotech company Sangamo Therapeutics started a clinical trial using a one-time, genome-editing fix to treat people born with a rare, inherited disease. (Feuerstein, 8/20)
The Wall Street Journal:
The Woman Who Has A Plan For Wall Street To Help Cure Blindness
Karen Petrou, an influential adviser to bankers and regulators, has made a career of deciphering complicated financial regulations. Now she’s trying to decode another type of puzzle. The conundrum: Matching medical researchers who need money with investors who have it. A bill outlining her strategy, which would include a government guarantee, was introduced in the House of Representatives last month. (Rexrode, 8/18)
The Perfect Lab Animal Is Strikingly, Surprisingly Beautiful
The worms turned out to be the perfect lab animal. They were simple creatures that lived in the dirt and ate bacteria but were just complicated enough to provide biological insights applicable to human health. C. elegans was the first organism to have its genome sequenced and, aside from humans, have been sent into space more than any other animal. (Chen, 8/20)
The New York Times:
This Drug Is Safe And Effective. Wait. Compared With What?
We spend many billions of dollars each year on the discovery and development of new drugs, but almost none of it addresses two crucial questions: How do these new therapies compare with already known ones? What are the relative benefits and harms in a particular situation, for a person like you? Such questions can best be answered by comparative effectiveness research. (Carroll, 8/20)