Abstract
Oligonucleotides—short, synthetic nucleotide strands that can be used as drugs to modulate gene expression—were at the heart of two deals inked in December. Vertex Pharmaceuticals will pay Entrada Therapeutics $250 million and up to $485 million later to advance treatments for myotonic dystrophy type 1, including a preclinical oligonucleotide therapy from Entrada that potentially restores muscle function. And GSK will pay Wave Life Sciences $170 million up front plus future milestones and royalties as part of a pact to advance up to 11 oligonucleotide programs.
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