DISEASE CATEGORY: Cancer INDICATION: Acute myelogenous leukemia (AML) Targeting LTR2B and the related LTR2, both long terminal repeat retrotransposons, could treat AML by reducing expression of the lipoprotein component APOCI. Analysis of a The Cancer...

Akcea and Ionis announced plans to develop AKCEA-APOCIII-LRx for familial chylomicronemia syndrome based on Phase II safety and efficacy results from hypertriglyceridemia patients. It gives the companies a second shot at marketing an APOC-III antisense...

CureVac, Genmab to develop RNA-encoded antibody Genmab A/S (NASDAQ:GMAB; CSE:GMAB) and CureVac AG have partnered to discover an mRNA-encoded antibody candidate to treat cancer. Genmab will pay $10 million up front and invest €20 million...

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...

Akcea Therapeutics lost roughly $412 million in market cap as the executive team was unexpectedly gutted on Monday. For now, an interim CEO hailing from majority owner Ionis Pharmaceuticals has been installed. The shakeup could...

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...

Waylivra hits Phase II/III endpoint in FPL Akcea Therapeutics Inc. (NASDAQ:AKCA) and Ionis Pharmaceuticals Inc. (NASDAQ:IONS) said Waylivra volanesorsen cut serum triglyceride levels by 88% vs. 22% for placebo (p<0.001) in patients with familial partial...

Akcea's Waylivra approved in EU The European Commission granted conditional approval to Waylivra volanesorsen from Akcea Therapeutics Inc. (NASDAQ:AKCA) and Ionis Pharmaceuticals Inc. (NASDAQ:IONS) as an adjunct to diet in adult patients with familial chylomicronemia...