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661 stories matching this filter · ← front page

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  • ARPA-H earmarks $160 million for custom gene-editing therapies, sparking debate on adequacy
    ARPA-H announced a $160 million investment to support development of bespoke gene‑editing treatments. This follows a recent case where an infant was rescued from a lethal disease using a personaliz…
    Endpoints · 2mo agoNeutral↗ source
  • AstraZeneca and Ionis see pivotal trial miss for ATTR-CM drug Wainua
    AstraZeneca announced that its experimental therapy Wainua, developed with Ionis, failed to meet primary endpoints in a pivotal study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). T…
    STAT · 2mo agoNegative readoutNegative↗ source
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  • Father spends $70 million on gene therapy trial for daughter with ultra-rare disease, faces FDA hurdles
    Matt Wilsey has spent a decade and about $70 million trying to develop a gene therapy for his 15-year-old daughter Grace, who suffers from a fatal ultra-rare disease. His personal quest has turned…
    STAT · 2mo agoNegative↗ source
  • ARPA-H launches $160 million THRIVE program to fund seven labs developing custom gene-editing therapies for rare diseases
    ARPA-H, the U.S. Advanced Research Projects Agency for Health, announced a new initiative called THRIVE with up to $160 million in funding. The program will support seven research teams over five…
    STAT · 2mo agoPositive↗ source
  • Prime Secures Arbitration Victory Over Beam, Paving Way for Clinical Trials
    Prime, a biotech firm developing a gene‑therapy candidate, won an arbitration case against Beam Therapeutics. The ruling removes a legal hurdle and allows Prime to advance its program into clinical…
    STAT · 2mo agoRegulatoryPositive↗ source
  • AstraZeneca/Ionis heart-failure drug Wainua fails pivotal trial as White House reviews FDA commissioner candidates
    The White House is reviewing a shortlist of three candidates for the FDA commissioner position, according to STAT. The list includes White House adviser Heidi Overton, oncologist and health‑system…
    STAT · 2mo agoNegative readoutNegative↗ source
  • Father invests $70 million in gene therapy quest for daughter with ultra‑rare NGLY1 deficiency, faces FDA scrutiny
    Matt Wilsey has spent a decade and roughly $70 million trying to develop a gene therapy for his daughter Grace, who suffers from NGLY1 deficiency, an ultra‑rare genetic disorder. NGLY1 deficiency…
    STAT · 2mo agoRegulatoryPositive↗ source
  • FDA issues early alert on non‑sterile BD ChloraPrep applicators in Medline convenience kits
    FDA CDRH issued an early alert about a potentially high‑risk device issue involving BD ChloraPrep applicators in Medline convenience kits that may not be sterile. Medline sent a letter on June 12…
    fda medwatch · 2mo agoRegulatoryNegative↗ source
  • Roche scraps two Ionis-partnered Huntington’s drugs
    The Swiss pharmaceutical giant told patient groups tominersen didn't delay disease progression in one trial, while a study of a second drug was scrapped because of a safety signal in animal testing.
    BioPharma Dive · 2mo agoRegulatoryNeutral↗ source
  • Lung transplants boost survival for select stage 4 lung cancer patients, study finds
    Northwestern Medicine surgeons and oncologists performed double lung transplants on a small group of stage 4 lung cancer patients whose disease was confined to the lungs. The JAMA study compared 1…
    STAT · 2mo agoPositive readoutPositive↗ source
  • Prime Medicine wins arbitration over gene-editing dispute with Beam, clearing path for AATD trial
    Prime Medicine announced it had prevailed in an arbitration against Beam Therapeutics, ending a dispute over gene-editing technology. The arbitrator determined that Prime’s work on a gene-editing…
    STAT · 2mo agoRegulatoryPositive↗ source
  • FDA approves Vera Therapeutics' Trutakna for IgA nephropathy, pricing set at $425,000 per year
    The U.S. Food and Drug Administration has granted approval to Trutakna, a new therapy from Vera Therapeutics, for the treatment of IgA nephropathy. IgA nephropathy is a kidney disorder where immun…
    STAT · 2mo agoApprovalPositive↗ source
  • Arbitration panel rules Prime Medicine did not breach Beam deal over AATD therapy
    An arbitration panel has ruled that Prime Medicine did not violate its 2019 collaboration agreement with Beam Therapeutics by advancing a treatment for alpha‑1 antitrypsin deficiency (AATD). The de…
    BioPharma Dive · 2mo agoNeutral↗ source
  • FDA targets online ketamine sellers as health premiums surge, and children 2+ can now obtain CGMs without prescription
    The Food and Drug Administration is intensifying its enforcement against online retailers that sell ketamine, a controlled substance, citing concerns about unregulated distribution and public safet…
    STAT · 2mo agoRegulatoryNeutral↗ source
  • FDA clears first over-the-counter glucose monitor for children 2 and older
    The FDA cleared Dexcom's Stelo continuous glucose monitor for over-the-counter sale, allowing children age 2 and up to obtain the device without a prescription. The agency says real-time glucose d…
    STAT · 2mo agoRegulatoryNeutral↗ source
  • Vertex to acquire Crinetics in $10 billion deal, expanding rare endocrine disorder pipeline
    Vertex announced on July 7, 2026 that it will acquire Crinetics Pharmaceuticals for $10 billion, marking the company’s largest-ever transaction. Crinetics is a biotech focused on developing treatm…
    STAT · 2mo agoDealNeutral↗ source
  • Vertex to acquire Crinetics in $10 billion deal, adding rare-disease drug Palsonify to its portfolio
    Vertex Pharmaceuticals announced a $10 billion transaction to purchase Crinetics Pharmaceuticals. The deal will bring Crinetics' recently launched drug Palsonify into Vertex's portfolio. Palsonify…
    STAT · 2mo agoDealPositive↗ source
  • FDA grants accelerated approval to Vera Therapeutics' Trutakna for IgA nephropathy
    The FDA has granted accelerated approval to Trutakna (formerly known as atacicept) from Vera Therapeutics for adult patients with IgA nephropathy, a chronic kidney disease that can progress to rena…
    BioPharma Dive · 2mo agoApprovalPositive↗ source
  • Vertex to buy Crinetics for $10 billion, expanding into endocrine disease drugs
    Vertex announced an agreement to acquire Crinetics Pharmaceuticals for $10 billion in cash, paying $85 per share. The purchase brings two recently launched drugs for a rare hormonal disorder into…
    BioPharma Dive · 2mo agoDealNeutral↗ source
  • Oberland invests up to $400M in MeiraGTx eye gene‑therapy pipeline
    Oberland Capital Management announced a financing package that could total $400 million to support MeiraGTx's development of gene therapies for inherited eye disorders. The arrangement includes up…
    BioPharma Dive · 2mo agoDealPositive↗ source
  • Vertex to buy Crinetics for $10 billion, adding acromegaly drug Palsonify
    Vertex Pharmaceuticals announced a $10 billion acquisition of Crinetics Pharmaceuticals, marking the largest deal in Vertex’s history. The purchase gives Vertex control of Crinetics’ marketed ther…
    STAT · 2mo agoDealPositive↗ source
  • Celea Therapeutics raises $180 million to advance next-generation IPF drug deupirfenidone
    Celea Therapeutics, a PureTech Health spinout, announced a $180 million venture round to fund development of its next-generation idiopathic pulmonary fibrosis (IPF) therapy, deupirfenidone. The ro…
    BioPharma Dive · 2mo agoDealPositive↗ source
  • Biotech Index Surges 30% Year-to-Date as Market Rally Accelerates
    Mid-year data show the biotech sector continuing a strong rally that began in April 2025. The XBI index, the most-watched biotech gauge, rose 19% in June and is up about 30% for the year, trading…
    STAT · 2mo agoPositive↗ source
  • Genetix, formerly Bluebird Bio, claims rapid turnaround and aims to treat 1,000 sickle-cell patients annually by 2030
    David Meek completed a distressed buyout of Bluebird Bio last year and has rebranded the company as Genetix. He says the transaction rescued the firm from the brink of bankruptcy and set it on a pa…
    STAT · 2mo agoDealPositive↗ source
  • Teen use of AI mental-health chatbots jumps 40% in a year, experts call for safety rules
    A recent study published in JAMA Pediatrics found that the proportion of adolescents turning to AI chatbots for mental-health advice rose from roughly one in eight to one in five within a single ye…
    STAT · 2mo agoRegulatoryNeutral↗ source