Once again, Eli Lilly and Co. has signed a billion-dollar deal, this time with Boston-based Ascidian Therapeutics Inc., a company that is barely four years old and one that is focused on treating human diseases by rewriting RNA. “Our technology, we call it RNA exon editing,” said Ascidian Chief Scientific Officer Robert Bell. “It edits RNA, not DNA … but it does so at the kilobase scale.”

Shiga toxin-producing Escherichia coli (STEC) represents a public health threat that can lead to serious problems, such as bloody diarrhea and hemolytic uremic syndrome in children in up to 10%-15% of cases. Antibiotics that normally combat diarrhea are not recommended for STEC infections and patients are usually treated only for symptomatology. Now, French researchers from Eligo Bioscience SA and their collaborators have published a paper on a CRISPR-based antimicrobial approach, EB-003.

Shares of Fulcrum Therapeutics Inc. (NASDAQ:FULC) fell 54% after the company said it is discontinuing work on sickle cell disease (SCD) candidate pociredir, its only clinical-stage candidate, and reviewing strategic alternatives in the wake of the U.S. FDA’s safety concerns regarding the drug target.

Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development.