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.”

Less than two months after winning FDA approval for a second indication for Filspari (sparsentan), Travere Therapeutics Inc. added to its rare kidney disease pipeline by exclusively licensing civorebrutinib from Everest Medicines Ltd. in a deal that could be worth more than $1.14 billion.

X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.