Two patients with a rare inherited disease are showing early signs of improvement after being injected with a groundbreaking genome-editing fix from Sangamo Therapeutics.
That’s the encouraging news emphasized by the biotech on Wednesday from its latest clinical trial, the first in which gene-editing is being used in patients to try to repair disease-causing DNA. But Sangamo is simultaneously downplaying other results from the study that are more confounding and may give outsiders reason to be concerned.
That’s because Sangamo is unable so far to show data conclusively linking its genome-editing therapy to the improvements seen in patients.
About the Author Reprints
My daughter has a rare disease called Dermatomyositis. She is 8 years old. Do you have anything that could help her?
To which person (and company to whom this person is affiliated) has the Court of Appeals “awarded” the Foundational Crown Patent for the “Gene Editing Process, cas 9” ? To Ms. Doudna of Intellia, Inc., Ms. Charpentier of Crisper Therapeutics AG, or Mr. Zhang of Editas Medicine, Inc. and on what date ?