Three years after Ionis spun out its Akcea unit in an IPO, and nearly a year after the parent company installed a new CEO at the subsidiary, Ionis has chosen to retake full ownership of...

FDA approves a second targeted DMD therapyNS Pharma Inc.’s Viltepso viltolarsen received accelerated approval from FDA to treat Duchenne muscular dystrophy in patients amenable to exon 53 skipping. Vyondys golodirsen from Sarepta Therapeutics Inc. (NASDAQ:SRPT) was the...

When gene editing company Verve went out seeking new investors for its latest round of funding, existing shareholder GV decided it would rather lead the round itself. GV and existing investors Arch Venture Partners, F-Prime...

Buoyed by news of this week's takeout of Medicines Co., Arrowhead is riding a wave of investor enthusiasm for RNAi therapeutics that has quintupled the company’s share price this year, and has boosted its valuation...

Akcea will bank $250 million up front from a new licensing deal with Pfizer, adding to a balance sheet already fattened by a February deal with Novartis for another antisense oligonucleotide program based on Akcea's...

With a cleaner safety profile and better efficacy than marketed agents, Regeneron’s first-in-class evinacumab is poised to become the standard of care for homozygous familial hypercholesterolemia and other rare lipid disorders, including for the hardest...

Regeneron's evinacumab could give certain hypercholesterolemia patients a new treatment option, with an FDA submission looming based on new results from a Phase III trial evaluating the first-in-class therapy as an add-on to existing treatments....

Most CRISPR companies are targeting rare genetic disorders, but Verve is breaking from the trend with gene editing therapies for coronary artery disease that could have fewer side effects, greater efficacy and better compliance than...

Secarna Pharmaceuticals GmbH’s antisense oligonucleotides for immuno-oncology could have better tissue penetration than small molecules and antibodies against the same targets. The company was established in 2015 by the founders and investors who launched antisense...

Arrowhead Pharmaceuticals Inc.’s tox blowup in 2016 set back its clinical timelines, but it also prompted the company to bring a more versatile platform online faster than originally planned. Early data from replacement candidates suggest...