Last week, pharmaceutical giant Biogen Inc. and start-up Scribe Therapeutics announced a joint project to develop therapies for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Scribe was co-founded by Jennifer Doudna, a newly crowned Nobel Laureate for her work on CRISPR, the gene editing technology that will be at the heart of this project.
ALS is a progressive condition that attacks the nervous system. It is famously associated with Gehrig, the Yankees great who was part of the “murderers row line-up” that terrified opposing pitchers through the 1920s and ’30s. It is associated, also, with Stephen Hawking, the English theoretical physicist who suffered from a slowly developing form of the disease.
Indeed, those two famous patients illustrate how different one case of ALS can be from another. Gehrig died just two years after his diagnosis: Hawking was diagnosed in 1963 and lived for another 55 years.
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This is not Biogen’s first press on the ALS front. Years ago it invested a lot of money in dexpramipexole, which seemed to hold promise against ALS. In 2013, though, dexpramipexole failed in a critical trial and Biogen had to stop development. Its return to the front in partnership with Scribe may prove an old truth: nothing is settled until it is settled right!