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  • Servier’s $1.55 B Edgewise muscular dystrophy trial fails to meet primary and secondary endpoints
    Servier invested roughly $1.55 billion in Edgewise Therapeutics' muscular dystrophy program and, five months after the deal, announced that the pivotal phase 2 trial in Becker muscular dystrophy di…
    Fierce Biotech · 5d agoNegative readoutNegative↗ source
  • Big pharma Q3 earnings roll out as FDA launches rare-disease initiative and uniQure's Huntington's gene therapy enters review
    Over the past week, a dozen major pharmaceutical companies reported third‑quarter results, including AstraZeneca, Biogen, GSK, Bristol Myers Squibb, Merck, Sanofi, AbbVie and Regeneron. Pfizer pos…
    BioSpace · 4d agoEarningsNeutral↗ source
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  • Ultragenyx to cut 20% of staff and halve R&D budget as it pivots to commercial sales
    Ultragenyx announced it will reduce its workforce by about 20%, bringing headcount down to roughly 1,000 employees. The cuts follow an earlier 10% reduction announced in February and are part of a…
    Fierce Biotech · 5d agoLayoffsNegative↗ source
  • Ultragenyx to lay off roughly 20% of staff as it halts two rare disease drug programs
    Ultragenyx announced it will cut about 20% of its workforce as it ends development of two rare disease drug candidates. The move follows a recent clinical study that failed to meet its primary end…
    STAT · 4d agoLayoffsNegative↗ source
  • CVS Health to split into two separate companies, separating insurance and pharmacy operations
    CVS Health announced that it will divide the business into two independent companies. One entity will house its insurance arm Aetna together with pharmacy‑benefit manager Caremark, while the other…
    STAT · 4d agoDealNeutral↗ source
  • Charles River partners with Ipsen to employ virtual control groups in toxicology studies
    Charles River Laboratories and French biopharma Ipsen announced a collaboration to use Charles River’s virtual control group (VCG) database in an upcoming toxicology study. The VCG approach aims t…
    Fierce Biotech · 4d agoDealPositive↗ source
  • Sanofi drops Nurix STAT6 degrader program amid molecule-specific concerns
    Sanofi has halted development of an early‑stage STAT6 degrader program that it licensed from Nurix Therapeutics. The decision was announced after the asset entered Phase 1 trials for type 2 inflamm…
    BioSpace · 4d agoNeutral↗ source
  • Servier’s $1.55 billion Becker muscular dystrophy drug fails pivotal Phase 3 trial
    Servier announced that its experimental drug for Becker muscular dystrophy did not meet its primary endpoint in a Phase 3 trial called GRAND CANYON. The drug, sevasemten, was acquired from Edgewis…
    STAT · 5d agoNegative readoutNegative↗ source
  • Neurocrine launches new safety study after another death linked to Prader-Willi drug Vykat
    Neurocrine Biosciences announced it will start a new study to evaluate dosing and safety of its rare disease drug Vykat in patients with Prader-Willi syndrome who have complex health issues. The m…
    BioSpace · 5d agoRegulatoryNegative↗ source
  • Scientists question safety and benefits of three-parent IVF as new UK data raise concerns
    The mitochondrial replacement technique, often called three‑parent IVF, was hailed after the United Kingdom reported the birth of eight healthy children whose mothers carried disease‑causing mitoch…
    STAT · 5d agoRegulatoryNegative↗ source
  • Compact Medical issues recall and updated instructions for ButterflyBVM bag-valve-mask resuscitators due to bellows holes
    The FDA's Center for Devices and Radiological Health has issued an early alert about a potential high‑risk problem with certain Compact Medical ButterflyBVM bag‑valve‑mask devices. Compact Medical…
    fda medwatch · 6d agoCRLNegative↗ source
  • Ultragenyx cuts 20% of workforce and abandons Wilson disease gene therapy after mixed Q3 results
    Ultragenyx said it will cut about 20% of its workforce, marking a second round of layoffs this year. The reductions will affect roughly a fifth of the company's employees. At the same time, the fi…
    Endpoints · 5d agoLayoffsNegative↗ 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 · 5d agoRegulatoryNeutral↗ source
  • UniQure slated for FDA decision by May 1 as analysts see potential for first Huntington’s disease therapy
    UniQure said the Food and Drug Administration has set a decision date for its Huntington's disease gene therapy and is likely to hold an advisory committee meeting. The agency indicated a faster‑th…
    BioPharma Dive · 5d agoRegulatoryNeutral↗ source
  • Novartis ends $800M neuroscience partnership with BioArctic after 14 months
    Novartis signed an $800 million neuroscience partnership with BioArctic in August 2025, providing $30 million upfront to merge BioArctic’s BrainTransporter technology with a Novartis antibody targe…
    Fierce Biotech · 6d agoDealNegative↗ source
  • Vertex, ahead of FDA decision, calms fears surrounding kidney drug
    On a conference call, executives expressed conviction in the coming launch of an IgA nephropathy medicine viewed by Wall Street investors as important to Vertex’s future.
    BioPharma Dive · 5d agoEarningsNeutral↗ source
  • FDA sets May 1 decision date for uniQure's Huntington's disease gene therapy after year-long regulatory saga
    The U.S. Food and Drug Administration has accepted uniQure's biologics license application for its Huntington's disease gene therapy and placed it on the priority review track, with a May 1 target…
    BioSpace · 6d agoRegulatoryPositive↗ source
  • FDA issues final guidance on potency assurance for cell and gene therapy manufacturers
    The FDA released final guidance today outlining a science- and risk-based Potency Assurance Strategy for cellular and gene therapy (CGT) products. The guidance replaces the agency’s 2011 potency t…
    fda press · 6d agoRegulatoryNeutral↗ source
  • FDA issues draft guidance to streamline approval of personalized gene therapies
    The FDA released a draft guidance that outlines an "adapted pathway" for regulating gene‑edited medicines. The new framework is intended to speed the development and approval of personalized gene t…
    BioSpace · 7d agoRegulatoryPositive↗ source
  • Karyopharm files for Chapter 11 bankruptcy as it pursues merger with Ayrmid
    Karyopharm Therapeutics has filed for Chapter 11 bankruptcy while seeking a merger with U.K. biotech Ayrmid. The court‑supervised process is intended to provide a financial lifeline for the company…
    BioSpace · 6d agoNegative↗ source
  • FDA accepts first Keytruda biosimilar application as Merck faces looming patent cliff
    The U.S. Food and Drug Administration has accepted the first publicly announced filing for a Keytruda biosimilar, identified as FYB206, which could receive approval as early as the third quarter of…
    BioPharma Dive · 6d agoRegulatoryNeutral↗ source
  • Woman travels to Albania for experimental mitochondrial replacement therapy amid global fertility industry concerns
    A STAT reporter detailed a case of a woman named Heidy Willis who, after multiple failed IVF cycles, sought an experimental fertility option abroad. Willis traveled to Albania to undergo mitochond…
    STAT · 6d agoNegative↗ source
  • FDA accepts review of Biohaven’s ataxia drug, boosting shares as ArriVent’s lung cancer trial miss drags its stock down
    Biohaven saw its shares jump after the FDA agreed to review its rare‑disease drug for spinocerebellar ataxia. The regulator’s decision lifted the stock by roughly 25% in late October, offering the…
    BioSpace · 6d agoRegulatoryNeutral↗ source
  • Women travel abroad for unproven three-parent IVF marketed to older patients
    STAT investigators found that women are journeying thousands of miles to undergo a mitochondrial‑replacement IVF procedure that is promoted as a fertility option for older patients, even though the…
    STAT · 6d agoNegative↗ source
  • Saudi Arabia pushes ahead with biotech hub despite regional instability
    Saudi Arabia is constructing a regulatory framework, partnership networks and intellectual property structures to nurture a biotech ecosystem, even as regional political tensions persist. Experts…
    BioSpace · 7d agoPositive↗ source