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Historically seen as a "platform monetization story," according to one analyst, Ionis now has a potential multibillion-dollar medicine in Tryngolza, which secured expanded approval...
Ionis Pharmaceuticals has offloaded regional rights to its near-approval rare disease asset zilganersen, pocketing $30 million upfront from Recordati for an ex-U.S. license to the...
AstraZeneca and Ionis Pharmaceuticals drug eplontersen, brand name Wainua, failed a Phase 3 test in cardiomyopathy caused by transthyretin amyloidosis. More details will be shared...
Eplontersen’s failure in transthyretin-mediated amyloidosis cardiomyopathy caught Wall Street analysts by surprise and boosted the outlook of rival drugs from Alnylam and BridgeBio...
Ionis Pharmaceuticals’ Tryngolza is now the first approved therapy for treating severe hypertriglyceridemia, a prevalent chronic metabolic disorder that can lead to acute pancreati...
Roche has halted development on two of its Huntington’s disease prospects, marking another blow for a patient community that has long awaited new medicines.
The Swiss pharmaceutical giant told patient groups tominersen didn't delay disease progression in one trial, while a study of a second drug was scrapped because of a safety signal...
The data showed that del-brax, an “antisense oligonucleotide conjugate” being tested against a muscle-wasting disease, met its primary endpoint in a Phase 1/2 study.
Alnylam has partnered with the person who helped put the “T” in ChatGPT, paying $30 million to work with Jakob Uszkoreit’s Inceptive Nucleics on artificial intelligence-enabled dru...
The RNA-focused drugmaker says Inceptive's tools will help prioritize the best development options as well as speed up the research process.
Founded by former MyoKardia executives, the startup has in mid- to late-stage testing three medicines licensed from the likes of Sanofi, Ionis and Bristol Myers Squibb.
Drug developed with Ionis could not reduce cardiovascular deaths or recurring heart problems
The findings supporting bepirovirsen were deemed a “historic moment” by physicians and validate a drug that has “clear blockbuster potential,” according to one analyst.
The planned acquisition of Kartos Therapeutics would hand Ipsen a late-stage asset for myelofibrosis. Elsewhere, Sobi hit a regulatory setback and BeOne unveiled more positive Bruk...
Co-founded by longtime Alnylam CEO John Maraganore, City has brought a clotting disorder drug into early human testing. An experimental therapy for Stargardt disease could soon fol...
Ascidian Therapeutics and Eli Lilly have signed a research collaboration potentially worth up to $1.9 billion, giving the Big Pharma company the chance to discover and develop RNA...
Immunovant shares climbed on results an analyst deemed “compelling” in tough-to-treat arthritis. Elsewhere, Wave’s pioneering rare disease program advanced and Takeda lost an antit...
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