Tag Archives: nusinersen

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 […]

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 […]

Posted in Boston, Boston blog main, Boston top stories, National blog main | Tagged Alexion Pharmaceuticals, Antibody Drug Conjugate (ADC), Atlas Venture, Biogen, Biotech, clinical trials, deals, Drugs, Dyne Therapeutics, FDA, Forbion, investing, Life Sciences, MPM Capital, Myotonic Dystrophy Type 1, nusinersen, oligonucleotides, RaNA Therapeutics, rare disease drugs, Rare diseases, Romesh Subramanian, Spinal Muscular Atrophy, startups, Translate Bio, Venture Capital | Comments Off

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 […]

Posted in Boston blog main, Boston top stories, Boulder/Denver blog main, Boulder/Denver top stories, Detroit blog main, Detroit top stories, Indiana blog main, Indiana top stories, National, National blog main, National top stories, New York blog main, New York top stories, Raleigh-Durham blog main, Raleigh-Durham top stories, San Diego blog main, San Francisco blog main, San Francisco top stories, Seattle blog main, Seattle top stories, Texas blog main, Texas top stories, Wisconsin blog main, Wisconsin top stories | Tagged 89Bio, AbbVie, Akero Therapeutics, alirocumab, Allergan, Alnylam Pharmaceuticals, Amgen, AstraZeneca, AveXis, bempedoic acid, Biogen, BioNtech, Biotech, Bristol-myers Squibb, Cassandra Trimnell, clinical trials, CRISPR, CRISPR Therapeutics, CureVac, Cystic Fibrosis, data, Editas Medicine, Esperion Therapeutics, evolocumab, FDA, Gene editing, GenFit, Gilead Sciences, Global Blood Therapeutics, human parainfluenza virus, inclisiran, Intercept Pharmaceuticals, Ivacaftor, Kalydeco, Keytruda, Life Sciences, Madrigal Pharmaceuticals, Merck, metapneumovirus, ModeRNA, mRNA, NASH, NGM Biopharmaceuticals, nonalcoholic steatohepatitis, Novartis, nusinersen, obeticholic acid, Orkambi, PCSK9, pembrolizumab, Pfizer, Praluent, ProteoStasis Therapeutics, PTC Therapeutics, Regeneron Pharmaceuticals, Repatha, risdiplam, RNA Interference, Roche, sickle cell disease, Spinal Muscular Atrophy, Spinraza, Symdeko, The Medicines Co., Translate Bio, Vertex Pharmaceuticals, Viking Therapeutics, voxelotor, Zolgensma | Comments Off

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, […]

Posted in Boston, Boston blog main, Boston top stories, National blog main, National top stories, New York blog main, New York top stories, San Diego blog main, San Diego top stories | Tagged Adrian Krainer, Alexandria Venture Investments, Apple Tree Partners, Biogen, Biotech, cannabidiol, Cormorant Asset Management, deals, dravet syndrome, Ed Kaye, epilepsy, GW Pharmaceuticals, Ionis Pharmaceuticals, IPO, Janus Henderson Investors, Life Sciences, nusinersen, Perceptive Advisors, RA Capital Management, Redmile Group, RNA, RTW Investments, Sphera Funds Management, Spinal Muscular Atrophy, Spinraza, startups, Stoke Therapeutics, VC, Zogenix | Comments Off

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 […]

Posted in Boston blog main, Boston top stories, National, National blog main, National top stories, New York blog main, New York top stories, San Diego blog main, San Diego top stories, Texas blog main, Texas top stories | Tagged Adrian Krainer, Biogen, Biotech, Breakthrough Prize, Breakthrough Prizes, Frank Bennett, Ionis Pharmaceuticals, Life Sciences, nusinersen, RNA splicing, SMA, spinal musclar atrophy, Spinraza | Comments Off

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 […]

Posted in Boston blog main, Boston top stories, Boulder/Denver blog main, Boulder/Denver top stories, Detroit blog main, Detroit top stories, Indiana blog main, Indiana top stories, National, National blog main, National top stories, New York blog main, New York top stories, Raleigh-Durham blog main, Raleigh-Durham top stories, San Diego blog main, San Diego top stories, San Francisco blog main, San Francisco top stories, Seattle blog main, Seattle top stories, Texas blog main, Texas top stories, Wisconsin blog main, Wisconsin top stories | Tagged 2025, ad-comm, Affimed, Akcea Therapeutics, Allogene Therapeutics, Amicus Therapeutics, Approval, apremilast, base editing, BIO, Biogen, Biome, Biotech, Blueprint Medicineas, briefing docs, CAR-T, Celgene, Cervical Cancer, clinical study, Clinical Trial, commissioner, CRISPR, CRISPR Therapeutics, Cybersecurity, Democrats, detection, Digital Health, Diversity, Donald Trump, endpoints, fail, failure, false, false statements, FDA, funding, Gardasil, Gene editing, Gene Therapy, GlaxoSmithKline, Glympse Bio, Gotham Therapeutics, Halozyme, hATTR, Helen Torley, in utero, inotersen, IPO, Jim Wilson, lies, Life Sciences, Loxo Oncology, medical devices, medicare, medullary thyroid cancer, MeiraGTx, Menlo Therapeutics, messenger RNA, nanotech, NASH, non-Hodgkin lymphoma, nonalcoholic steatohepatitis, Novartis, nusinersen, Otezla, Parkinson's Disease, President, RET, scalp psoriasis, Scott Gottlieb, serlopitant, sickle cell, Sitryx, SMA, Spinal Muscular Atrophy, Study, Tech, tegsedi, transthyretin amyloidosis, Trevena, tweetorial, USA TODAY, Vaccine, VC, Venture Capital, Vertex | Comments Off

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 […]

Posted in Boston blog main, Boston top stories, Boulder/Denver blog main, Boulder/Denver top stories, Detroit blog main, Detroit top stories, Indiana blog main, Indiana top stories, National, National blog main, National top stories, New York blog main, New York top stories, Raleigh-Durham blog main, Raleigh-Durham top stories, San Diego blog main, San Diego top stories, San Francisco blog main, San Francisco top stories, Seattle blog main, Seattle top stories, Texas blog main, Texas top stories, Wisconsin blog main, Wisconsin top stories | Tagged Acne, Aduro BioTech, Amgen, amikacin liposome, Arrowhead Pharmaceuticals, Avrobio, Biogen, Biotechnology Industry Organization (BIO), bitoech, Blackstone Group, Boston Pharmaceuticals, cancer, cancer immunotherapy, cemiplimab, checkpoint inhibitors, Clarus Ventures, Dragonfly Therapeutics, Duchenne Muscular Dystrophy, Eli Lilly, emicizumab, Fabry Disease, galcanezumab, Gene Therapy, GlaxoSmithKline, Guardant Health, Helen Torley, hemophilia A, Insmed, investing, IPO, James Allison, Janssen Biotech, JLABS @ San Diego, Johnson & Johnson, Kodiak Sciences, Kyoto University, Life Sciences, Liquid Biopsy, Merck, Migraine, Modis Therapeutics, myobacterium avium complex, Nobel Prize, Novartis, nusinersen, omadacycline, Paratek Pharmaceuticals, pneumonia, Regeneron Pharmaceuticals, Renaissance Capital, risdiplam, RNA Interference, Roche, Sanofi, sarecycline, Sarepta Therapeutics, Spinal Muscular Atrophy, startups, Tasuku Honjo, Teva Pharmaceutical, The University of Texas MD Anderson Cancer Center, thymidine kinase 2 deficiency, TWist Bioscience, VelosBio | Comments Off

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, […]

Posted in Boston blog main, Boston top stories, Boulder/Denver blog main, Boulder/Denver top stories, Detroit blog main, Detroit top stories, Indiana blog main, Indiana top stories, National, National blog main, National top stories, New York blog main, New York top stories, Raleigh-Durham blog main, Raleigh-Durham top stories, San Diego blog main, San Diego top stories, San Francisco blog main, San Francisco top stories, Seattle blog main, Seattle top stories, Texas blog main, Texas top stories, Wisconsin blog main, Wisconsin top stories | Tagged Accelerator, aflibercept, allergy, Alnylam Pharma ceuticals, amazon, Amicus Therapeutics, apple, Approval, Biogen, Biotech, cancer, clinical trials, cost, CVS, CVS Caremark, CVS Health, drug price, EpiPen, Express Scripts, Eylea, Fabry Disease, fasinumab, FDA, FDA approval, Gene Therapy, generic, google, guidance, hereditary transthyretin amyloidosis, HIV, ICER, Institute for Clinical Economic Review, interference, Kaiser Health News, Life Sciences, Macular Degeneration, Magnolia Neurosciences, Microsoft, migalastat, National Institute for Health and Care Excellence, nusinersen, Oncology, opidoids, Orchard Therapeutics, Prices, Pricing, rejection, RNA interfernece, RNAi medicine, Safety, Spinraza, Teva Pharmaceutical, therapeies, Trump administration, VC, Venture Capital, ViiV Healthcare | Comments Off

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 […]

Posted in Boston, Boston blog main, Boston top stories, Indiana blog main, Indiana top stories, National blog main, New York blog main, New York top stories, San Francisco blog main, San Francisco top stories | Tagged Alexion Pharmaceuticals, Armo Biosciences, AveXis, Biogen, deals, IPOs, Kiniksa Pharmaceuticals, Life Sciences, Menlo Therapeutics, nusinersen, Regeneron Pharmaceuticals, Renaissance Capital, Sanj Patel, Scholar Rock, Spinraza, startups, Synageva BioPharma, VC | Comments Off

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 […]

Posted in Boston blog main, Boston top stories, Boulder/Denver blog main, Boulder/Denver top stories, Detroit blog main, Detroit top stories, Indiana blog main, Indiana top stories, National, National blog main, National top stories, New York blog main, New York top stories, Raleigh-Durham blog main, Raleigh-Durham top stories, San Diego blog main, San Diego top stories, San Francisco blog main, San Francisco top stories, Seattle blog main, Seattle top stories, Texas blog main, Texas top stories, Wisconsin blog main, Wisconsin top stories | Tagged 5AM Ventures, Acorda Therapeutics, American Academy of Neurology, AstraZeneca, AveXis, baricitinib, Biogen, Biotech, Broad Institute, Canaan Partners, cancer, cancer immunotherapy, Cedilla Therapeutics, Chris Garabedian, CRISPR, deals, Dengue Fever, durvalumab, Eli Lilly, Epizyme, FDA, Feng Zhang, Food and Drug Administration, Immunotherapy, investing, Ionis Pharmaceuticals, Jennifer Doudna, Life Sciences, Mammoth Biosciences, New Leaf Ventures, nusinersen, Pardis Sabeti, Parkinson's Disease, pimavanserin (Nuplazid), postpartum depression, Rallybio, rare disease drugs, Revolution Medicines, Rheumatoid Arthritis (RA), Sage Therapeutics, Sarepta Therapeutics, Scott Gottlieb, Shire, Spinal Muscular Atrophy, startups, Sunovion Pharmaceuticals, Takeda Pharmaceutical, tremelimumab, University of California Berkeley, University of Texas at San Antonio Health Science Center, Zika virus | Comments Off← Older posts Newer posts →

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