Tag Archives: nusinersen

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Report: Biogen, Novartis Should Drop Prices of Billon-Dollar SMA Drugs

A new report from a U.S. drug-pricing watchdog takes issue with the high cost of the only approved treatment for spinal muscular atrophy (SMA), a rare genetic disease that affects young children and in some cases leads to a quick death. The report, from the Boston-based Institute for Clinical and Economic Review (ICER), is loaded […]

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Dyne Therapeutics Emerges With $50M to Take On Rare Muscle Disease

One problem with drugs for muscular disorders is that not enough medicine reaches the muscle, says Romesh Subramanian, CEO of Dyne Therapeutics. Subramanian’s startup has developed a way to deliver drugs to all muscle types, and it is emerging from stealth with $50 million in funding to advance its compounds toward human testing. Dyne was […]

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15 For ’19: Key Clinical Data to Watch For Next Year (Part 2)

On Monday, Xconomy began our annual look ahead at clinical trials that could define biotech and have profound healthcare effects in the coming year. Today we’re circling back with the rest of the list, which includes studies of drugs for nonalcoholic steatohepatitis, cystic fibrosis, spinal muscular atrophy and more. Read on for the details. [Editor’s […]

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With $90M Round, Stoke Eyes a “Spinraza For Epilepsy” And a 2019 IPO

Ed Kaye stepped aside from Sarepta Therapeutics (NASDAQ: SRPT) last year after leading the company through one of the most noteworthy and controversial drug approvals in recent memory, for the Duchenne muscular dystrophy drug eteplirsen (Exondys 51). But his hiatus from a publicly traded biotech could be short-lived. Kaye’s new startup, Bedford, MA-based Stoke Therapeutics, […]

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Spinraza Inventors Take Home $3M Breakthrough Prize in Life Sciences

The inventor and developer of a drug, nusinersen (Spinraza), are among the winners of the Breakthrough Prize in Life Sciences. Adrian Krainer (pictured) of Cold Spring Harbor Laboratory and Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, share the $3 million award. The Breakthrough Prizes, now it their seventh year, tout themselves as […]

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Bio Roundup: BIO Diversity, Allogene IPO, CRISPR In Utero & More

Nearly four years after investors snapped up new shares of Juno Therapeutics in a $265 million IPO, cancer immunotherapy remains a hot ticket. Like Juno, Allogene Therapeutics just pulled in a massive IPO haul, nearly $300 million. And like Juno, Allogene is working with a live immune-cell treatment called CAR-T, but it is harvesting cells […]

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Bio Roundup: Nobel Prizes, Duchenne Steps, Guardant’s IPO & More

Cancer immunotherapy is all the rage these days. The idea of harnessing the immune system to treat cancer has sparked billions of dollars in research, led to a vast matrix of clinical trials, and started to change the way how several cancers are treated. The reason: When it works, cancer immunotherapy can lead to longer-lasting […]

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Bio Roundup: Big Approvals, ICER Influence, Drug-Price Pushback & More

[Updated 8/17/18, 10:21 a.m. See below.] We’ll start the roundup this week with two drug approvals that came late last Friday. Both were landmarks for the companies receiving the nod, Alnylam Pharmaceuticals and Amicus Therapeutics. We also saw a new biotech emerge in the muggy New York heat, a couple deals for new flu vaccines, […]

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Biotech IPOs Roll On as Kiniksa, Scholar Rock Get $227M Combined

The IPO window remains wide open for biotechs. Two more, Kiniksa Pharmaceuticals and Scholar Rock, make their Wall Street debuts this morning having raised $227 million combined in their IPOs last night. Kiniksa exceeded its funding goals. The Lexington, MA, company sold 1.44 million more shares than it projected, or 8.44 million total, at $18 […]

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Bio Roundup: CRISPR Tests, Parkinson’s Questions, Opioid Bills & More

Politics is more partisan than ever these days, but the opioid crisis might be one of the few issues where elected officials find common ground. This week, members of Congress exercised a bipartisan effort in both chambers to advance dozens of measures proposing various approaches to fighting the opioid epidemic. A Senate committee voted unanimously […]

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