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  • Merck and Moderna report positive late-stage melanoma vaccine results, adding to 2026 cancer breakthrough year
    Merck and Moderna announced that their personalized mRNA neoantigen vaccine achieved positive outcomes in a late-stage trial for melanoma patients. The data suggest the vaccine can stimulate an imm…
    STAT · 2mo agoPositive readoutPositive↗ source
  • FDA approves Ultragenyx gene therapy and Regeneron ultra-rare disease drug
    The Food and Drug Administration gave the green light to a gene‑therapy product from Ultragenyx, marking a new treatment option in the company’s pipeline. In a separate decision, Regeneron secured…
    STAT · 2mo agoApprovalPositive↗ source
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  • Roche commits $750 million to double Oregon plant capacity as obesity drug push grows
    Roche's Genentech announced a $750 million investment to enlarge its device fill-finish facility in Hillsboro, Oregon. The expansion will double the plant's size and add end-to-end capabilities fo…
    BioSpace · 2mo agoDealPositive↗ source
  • Scribe Therapeutics’ early-stage IPO surges 43% as epigenetic cholesterol drug enters clinic
    Scribe Therapeutics filed for an IPO while its lead epigenetic therapy moves into early clinical testing, a rare combination in a market that prefers later-stage, de-risked offerings. The company…
    BioSpace · 2mo agoPositive↗ source
  • Regeneron secures FDA approval for Pasatru, second U.S. treatment for rare bone disease FOP
    Regeneron received FDA approval for its antibody garetosmab, marketed as Pasatru, for treatment of fibrodysplasia ossificans progressiva (FOP) in adults. The approval makes Pasatru the second ther…
    BioSpace · 2mo agoApprovalPositive↗ source
  • Layoffs hit over 1,300 workers across seven biopharma M&A deals in 2026
    Workforce reductions this year have affected at least seven biopharma companies that recently completed mergers or acquisitions, eliminating a minimum of 1,359 jobs, according to BioSpace data. Th…
    BioSpace · 2mo agoLayoffsNegative↗ source
  • Trump nominates Heidi Overton as FDA Commissioner as agency releases CGT Q&A guidance
    The U.S. Food and Drug Administration issued final guidance that answers common questions about developing cellular and gene therapy products. The document aims to clarify regulatory expectations f…
    raps · 2mo agoRegulatoryNeutral↗ source
  • Ultragenyx secures FDA approval for Glenglycos, first gene therapy for GSD Ia
    Ultragenyx announced FDA approval of Glenglycos, a one-time gene therapy for glycogen storage disease type Ia, a rare genetic disorder. The therapy delivers a functional gene to the liver to resto…
    BioPharma Dive · 2mo agoApprovalPositive↗ source
  • Ultragenyx secures first gene therapy approval for GSDIa as Sanfilippo asset decision pending
    Ultragenyx received accelerated FDA approval for its gene therapy DTX401, marketed as Genglycos, to treat glycogen storage disease type Ia (GSDIa). The approval comes as the company awaits a secon…
    BioSpace · 2mo agoApprovalPositive↗ source
  • Capricor Therapeutics likely faces FDA rejection of its Duchenne cell therapy
    Capricor Therapeutics is working to avoid an imminent FDA rejection of its cell‑based treatment for Duchenne muscular dystrophy. The company says it remains confident in the drug, but the regulator…
    STAT · 2mo agoRegulatoryNegative↗ source
  • Capricor’s Duchenne drug deramiocel receives negative FDA advisory vote on heart outcome
    Mindy Leffler, a parent of a young man with Duchenne muscular dystrophy, writes an opinion piece about Capricor’s phase 3 study of its drug deramiocel. She describes her son’s experience living ind…
    STAT · 2mo agoRegulatoryNegative↗ source
  • Health systems deploy AI chatbots to streamline patient record review
    Several large health systems are rolling out generative AI chatbots that can query and summarize electronic medical records. Clinicians report that the tools save time and help uncover information…
    STAT · 2mo agoPositive↗ source
  • Biotech firms EMD Serono and Exelixis cut remote R&D staff as they enforce office return policies
    Biopharma workers at two biotech companies are losing jobs as employers shift away from remote work. The changes affect research and development staff who were previously allowed to work from home.…
    BioSpace · 2mo agoLayoffsNegative↗ source
  • FDA approves Ultragenyx gene therapy for glycogen storage disorder; Overton faces confirmation hurdles
    The U.S. Food and Drug Administration has granted approval to Ultragenyx's gene therapy aimed at treating a glycogen storage disorder. This marks a regulatory milestone for the company and the rare…
    raps · 2mo agoApprovalPositive↗ source
  • Regeneron wins FDA approval for Pasatru, targeting rare bone disease FOP
    The U.S. Food and Drug Administration has approved Regeneron Pharmaceuticals' drug Pasatru for the treatment of fibrodysplasia ossificans progressiva (FOP), a very rare condition in which muscle an…
    BioPharma Dive · 2mo agoApprovalPositive↗ source
  • Trump administration nominates Heidi Overton as FDA commissioner after acting chief declines
    The White House announced that Heidi Overton has been nominated to lead the Food and Drug Administration after acting commissioner Kyle Diamantas turned down a permanent appointment. Reports from…
    STAT · 2mo agoRegulatoryNeutral↗ source
  • American Cancer Society cancels wine and spirits gala amid conflict-of-interest concerns
    The American Cancer Society announced it will not hold the planned Wine and Spirits Industry Gala after internal review highlighted a conflict of interest with the alcohol sector, which is linked t…
    STAT · 2mo agoNeutral↗ source
  • Experts discuss persistent CMV challenges and new antiviral approaches for transplant patients
    In a recent episode of BioSpace's Denatured podcast, Ian McGowan, chief medical officer of Synklino, and James Hunter, an associate professor of transplantation at Oxford University, joined host Je…
    BioSpace · 2mo agoNeutral↗ source
  • Pediatric rare cancer research draws steady investment despite biotech market volatility
    The article notes that pediatric rare cancers continue to attract investment because they address the greatest unmet medical needs and allow clear measurement of patient impact. Even as the biotec…
    BioSpace · 2mo agoPositive↗ source
  • Experts call for modernizing rare disease evidence models using FDA plausible mechanism framework
    A recent paper in The Journal of Clinical Investigation argues that the evidentiary approach for rare diseases needs updating. The authors suggest applying the FDA’s plausible mechanism framework,…
    raps · 2mo agoNeutral↗ source
  • US allocates $2 billion to faith-based health groups, highlighting shift toward faith-driven capital in global aid
    The Trump administration this month announced a nearly $2 billion allocation for faith‑based organizations, the largest such commitment in over two decades. About $1.4 billion is earmarked for hea…
    STAT · 2mo agoNeutral↗ source
  • EMA launches consultation on draft framework to speed adoption of data standards
    EMA has opened a public consultation on a draft framework aimed at accelerating the implementation of data standards across the pharmaceutical sector. The agency says the new framework is intended…
    raps · 2mo agoRegulatoryNeutral↗ source
  • Experts warn protein restriction could harm older adults and GLP-1 patients
    Researchers have recently suggested that limiting dietary protein might improve metabolic health and extend lifespan, a claim highlighted in a review paper in Cell Press Blue. Nutrition specialist…
    STAT · 2mo agoNegative↗ source
  • Experts urge DSM to list muscle dysmorphia as eating disorder to improve care for men
    Males account for roughly one third of people in the United States who struggle with eating disorders, and many of these conditions begin in the teenage years. Some estimates suggest that as many a…
    STAT · 2mo agoNeutral↗ source
  • Merck and Moderna report success in late-stage trial of personalized mRNA cancer vaccine
    Merck and Moderna announced that their personalized mRNA cancer vaccine achieved its primary endpoint in a randomized late-stage trial, marking the first demonstration that such an approach can be…
    STAT · 2mo agoPositive readoutPositive↗ source