This year’s Nobel Prize for chemistry was awarded Wednesday to the two discoverers of Crispr/Cas9, a defense mechanism in bacteria that’s become the basis for gene-editing treatments for inherited illnesses like sickle-cell anemia.
Emmanuelle Charpentier and Jennifer Doudna first proposed the idea of gene editing in 2012. The technology revolutionized biological research and led to the launch of a new generation of medical ventures, including
(ticker: CRSP)—co-founded by Charpentier—as well as Caribou Biosciences, Mammoth Biosciences, and Scribe Therapeutics—all co-founded by Doudna. Other public companies using Crispr-based techniques include
In early trading Wednesday, the gene-editing stocks were all up about 6% each, lifting Crispr Therapeutics to $93, Editas to $30.39, and Intellia to $22.20. Beam was up 5%, to $28.65.
Gene-editing technologies hold out promise for one-time treatments that could cure genetic illnesses that are caused by errors in the instructions coded in our DNA.
The first gene therapies to reach the clinic left malfunctioning DNA intact, while adding some corrective code to cells. Gene editing with Crispr/Cas9 actually changes the erroneous stretch of DNA, which might better fix some genetic diseases and make cancer treatments more successful. The technology’s ability to find and manipulate specific genes has also made it an everyday tool for exploring the biology of all living things and for diagnosing diseases.
Doudna works at the University of California, Berkeley. Just on Tuesday, her privately held Scribe Therapeutics announced a partnership with
(BIIB) that could bring Scribe over $400 million if they succeed in developing treatments for neurological diseases like amyotrophic lateral sclerosis, the progressive disorder that’s often called Lou Gehrig’s disease.
Charpentier is at the Max Planck Institute in Berlin. The firm she advises, Crispr Therapeutics, is furthest along among companies bringing gene-editing to the clinic. In Phase 1 trials, it has begun treating people who suffer from sickle cell. So far, the treatments have freed those folks from the disease’s awful symptoms.
Write to Bill Alpert at [email protected]