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701 stories matching this filter · ← front page

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  • Ionis shares tumble over $1 billion as Tryngolza sales forecast cut in half
    Ionis Pharmaceuticals saw its market value drop by more than $1 billion after it lowered its sales outlook for Tryngolza, a triglyceride‑lowering therapy. The company now expects $50 million in sa…
    STAT · 9h agoNegative↗ source
  • Amneal and BridgeBio top BioSpace 2027 Best Places to Work list
    BioSpace released its 2027 Best Places to Work list, recognizing 50 life‑science employers with U.S. operations. Amneal Pharmaceuticals, based in Bridgewater, New Jersey, took the top spot among l…
    BioSpace · 5h agoPositive↗ source
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FDA approves Bayer’s Sunharbo, first Factor XIa inhibitor to lower stroke recurrence risk
The Food and Drug Administration has approved Bayer's new medication intended to prevent another stroke in patients who have already experienced one. The drug, sold under the brand name Sunharbo,…
STAT · 12h agoApprovalPositive↗ source
  • Lilly's oral GLP-1 Foundayo cuts sleep apnea severity in Phase 3, paving way for second FDA filing
    Lilly said it will submit its oral GLP-1 drug Foundayo for FDA approval in obstructive sleep apnea after a late‑stage trial showed strong efficacy. The Phase 3 ATTAIN-OSA study enrolled about 712…
    BioSpace · 16h agoPositive readoutPositive↗ source
  • FDA warns GLP-1 weight-loss drugs may cause rare eye stroke condition
    The Food and Drug Administration issued a warning that GLP-1 drugs used for weight loss may be linked to a rare eye condition called non-arteritic anterior ischemic optic neuropathy, sometimes desc…
    STAT · 18h agoRegulatoryNegative↗ source
  • Novartis' Phase 3 HORIZON trial shows pelacarsen lowers Lp(a) but fails to cut cardiovascular events
    Novartis and Ionis reported that the Phase 3 HORIZON trial of the antisense drug pelacarsen did not improve major adverse cardiovascular events despite markedly lowering lipoprotein(a) levels. The…
    BioSpace · 19h agoNegative readoutNegative↗ source
  • FDA launches Rare Disease Framework to embed patient input early in drug development
    FDA's Center for Biologics Research and Evaluation (CBER) director Karim Mikhail announced a new Rare Disease Framework Initiative aimed at moving patient perspectives from an optional, late‑stage…
    BioSpace · 1d agoRegulatoryPositive↗ source
  • FDA mandates label update for all GLP-1 drugs to warn of rare eye disease risk
    The FDA issued a class‑wide labeling requirement for all approved GLP‑1 receptor agonists, adding a warning about non‑arteritic anterior ischemic optic neuropathy (NAION), a rare eye condition that…
    BioSpace · 16h agoRegulatoryNeutral↗ source
  • Compass Pathways to file FDA approval for psilocybin depression therapy within weeks
    Compass Pathways announced it will complete its FDA filing for the psilocybin‑based depression treatment COMP360 in the next two weeks. The company expects the rolling submission to enable a drug…
    BioSpace · 18h agoRegulatoryNeutral↗ source
  • Global liver experts agree on definition of acute-on-chronic liver failure
    Liver doctors from many countries have reached a common definition for acute-on-chronic liver failure, a serious complication that occurs when an existing liver disease is suddenly worsened by an a…
    STAT · 1d agoPositive↗ source
  • American Heart Association meeting reports neutral outcomes for inflammation and Lp(a) trials
    The American Heart Association's scientific sessions in Chicago highlighted a theme of neutral findings across cardiovascular research. Two high profile trials, ZEUS targeting inflammation and HOR…
    STAT · 17h agoNeutral↗ source
  • CLINUVEL to cut up to 20% of staff and move headquarters to New York
    CLINUVEL announced a strategic reorganization aimed at focusing resources on the U.S. market. The company will reduce its global workforce by 10% to 20%, affecting up to 21 of its 104 employees.…
    BioSpace · 20h agoLayoffsNegative↗ source
  • Update: FDA investigates Novartis' Zolgensma following 9 cases of tumors in treated patients
    After Novartis confirmed the reports of tumors in patients who have been treated with the spinal muscular atrophy gene therapy, the FDA will determine if any regulatory action is needed for Zolgens…
    BioSpace · 20h ago↗ source
  • New evidence links AAV gene therapies to tumor risk, raising safety concerns
    Recent cases reported this year provide strong evidence that adeno-associated viruses (AAVs), the vectors used to deliver therapeutic genes, can trigger tumor formation. The findings settle a two‑d…
    STAT · 3d agoRegulatoryNegative↗ source
  • Forge Biologics VP sees AI and Ohio talent driving next wave of gene therapy manufacturing
    Samantha Piteri-Molina, vice president of business and strategy at Forge Biologics, said her focus is on getting gene therapies into patients' hands. She helped steer Forge through a $620 million…
    BioSpace · 3d agoPositive↗ source
  • Agios cuts over 35% of R&D staff as FDA decision on sickle cell drug is delayed
    Agios announced it will eliminate more than 100 R&D positions, representing over a third of its research staff, after the FDA postponed a decision on its sickle cell disease candidate. The company…
    BioSpace · 3d agoLayoffsNegative↗ source
  • Verdicts for Summit/Akeso, Capricor, Vertex mark FDA’s stacked November
    Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
    BioSpace · 3d agoRegulatoryNeutral↗ source
  • Novartis reports seven new tumor cases in children treated with Zolgensma gene therapy
    Novartis has disclosed that seven children who received its SMA gene therapy, Zolgensma, have been found to have tumors that were not previously reported. The cases were brought to light by an adv…
    STAT · 4d agoRegulatoryNegative↗ source
  • AstraZeneca reportedly bids $10 billion for metabolic biotech Rhythm Pharmaceuticals
    AstraZeneca is said to have made a $10 billion offer to acquire Rhythm Pharmaceuticals, a biotech that develops treatments for rare neuroendocrine disorders. The proposal values Rhythm at $124 per…
    BioSpace · 4d agoDealPositive↗ source
  • Novartis reports nine tumor cases in patients treated with Zolgensma gene therapy
    Novartis has confirmed that nine cases of tumors have been reported in patients who received its spinal muscular atrophy gene therapy, Zolgensma. The company says two cases are confirmed and seven…
    BioSpace · 4d agoNegative↗ source
  • British biotech T-Therapeutics raises $133 million Series B to advance bispecific T-cell engagers
    T-Therapeutics, a British biotech, announced a $133 million Series B financing to advance its next-generation T-cell engager therapeutics. The round was co-led by Advent Life Sciences and the Brit…
    BioSpace · 4d agoDealPositive↗ source
  • Sickle cell gene-editing therapy shows signs of revival as developers revisit trial after earlier safety halt
    A recent STAT+ article highlights that a sickle cell gene‑editing therapy, whose trial was stopped three years ago after a serious adverse event in the first patient, may be making a comeback. The…
    STAT · 4d agoNeutral↗ source
  • Democrats’ midterm sweep positions Bernie Sanders to steer Senate health committee, raising hopes for major FDA reforms
    The recent midterm elections delivered a decisive victory for Democrats, giving the party control of Congress and setting the stage for sweeping health‑care changes. Among the anticipated shifts,…
    STAT · 4d agoRegulatoryNeutral↗ source
  • China clamps down on investigator-initiated biotech trials after patient deaths, halting rare-disease studies
    Chinese regulators have introduced new rules that sharply limit investigator‑initiated biotech trials after a series of patient deaths were reported this summer. The policy shift is intended to tig…
    STAT · 5d agoRegulatoryNegative↗ source
  • MDCG issues guidance on clinical evidence for orphan in-vitro diagnostics
    The Medical Device Coordination Group (MDCG) has published new guidance that explains how clinical evidence requirements apply to orphan in-vitro diagnostics (IVDs). The document is intended to he…
    raps · 5d agoRegulatoryNeutral↗ source