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115 stories matching this filter · ← front page

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  • Harvard scientist George Church quits AI startup Radical, alleging partner stole his credit
    George Church, a prominent Harvard researcher known for advising biotech and synthetic biology ventures, has stepped down from his role at the artificial‑intelligence startup Radical. In a public…
    Endpoints · 2d agoNegative↗ source
  • Sarepta halts enrollment in confirmatory Phase 3 trial of Elevidys gene therapy for Duchenne muscular dystrophy
    Sarepta Therapeutics announced it will no longer enroll additional patients in the Phase 3 confirmatory study of its gene therapy Elevidys for Duchenne muscular dystrophy. The trial was intended t…
    Endpoints · 2d agoCRLNegative↗ source
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Psychiatry & CNS1162
Ophthalmology984
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Pain766
Nephrology735
  • Alethio Therapeutics raises $90 million Series A to develop antibody for essential thrombocythemia
    Alethio Therapeutics, a spinout from the University of Oxford, announced a $90 million Series A financing round. The capital will fund the company’s lead program, an antibody designed to treat ess…
    Endpoints · 4d agoDealPositive↗ 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 · 4d agoRegulatoryNeutral↗ 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
  • Early Alert: B. Braun Medical Inc. Removes Streamline Bloodline Set for Dialog+
    The dialyzer connector may not be attached appropriately, resulting in leaking and potential blood loss.
    fda medwatch · 7d agoRegulatoryNeutral↗ source
  • Setara Biosciences raises $90 million to advance rare-disease pipeline after Actio spin-out
    Actio Biosciences shipped its precision epilepsy drug to Jazz Pharmaceuticals and then separated the rest of its genetics-driven pipeline into a new company called Setara Biosciences. Setara has s…
    Endpoints · 9d agoDealPositive↗ source
  • FDA releases draft guidance on oversight of individualized gene-editing products
    The U.S. Food and Drug Administration issued draft guidance that outlines a proposed regulatory framework for individualized gene‑editing products, including cases where multiple product variants a…
    raps · 13d agoRegulatoryNeutral↗ source
  • FDA issues early alert on B. Braun Streamline Bloodline Set recall due to connector leak risk
    The FDA's Center for Devices and Radiological Health issued an Early Alert about B. Braun's Streamline Bloodline Set for Dialog+ after discovering a potential high risk issue. The problem is trace…
    fda medwatch · 13d agoCRLNegative↗ source
  • Mennonite family backs new gene-therapy startup
    A new gene‑therapy company is taking shape with financial support from members of the Amish and Mennonite communities. The story highlights the unusual partnership between a traditional religious g…
    Endpoints · 12d agoDealNeutral↗ source
  • FDA Commissioner Highlights New Rare Disease Framework Emphasizing Patient Voice
    Acting US Food and Drug Administration (FDA) Commissioner Kyle Diamantas highlighted the agency's new rare disease framework during a fireside chat at the NORD Breakthrough Summit. The framework i…
    raps · 14d agoRegulatoryPositive↗ source
  • Pfizer halts Phase 2 trial of PF-06835375 in primary immune thrombocytopenia
    Pfizer announced that it is stopping its Phase 2 study of the experimental program PF-06835375, which was being evaluated in patients with primary immune thrombocytopenia, a rare autoimmune blood d…
    Endpoints · 19d agoNegative readoutNegative↗ source
  • Merck KGaA to acquire Silence Therapeutics for $1.3 billion, expanding siRNA portfolio
    Merck KGaA, the German science and technology conglomerate, announced an agreement to purchase UK‑based Silence Therapeutics. The deal is valued at about $1.3 billion and gives Merck access to Sil…
    Endpoints · 20d agoDealPositive↗ source
  • Disc Medicine's rare sun-sensitivity drug meets all endpoints in Phase 3 trial
    Disc Medicine announced that its experimental therapy for a rare condition that makes sun exposure painful has completed a Phase 3 study. The trial reported success on every primary and secondary…
    Endpoints · 20d agoPositive readoutPositive↗ source
  • Oxford biotech OMass Therapeutics secures $310 million to advance genetic adrenal disorder program
    Oxford-based OMass Therapeutics announced a $310 million private financing round, one of the largest recent fundraises for a UK biotech. The capital will fund the development of therapies targetin…
    Endpoints · 25d agoDealNeutral↗ source
  • FDA launches rare disease framework initiative
    A new initiative from the US Food and Drug Administration (FDA) aims to develop “an optimized, fit-for-purpose” framework to facilitate the development and marketing of treatments for rare diseases…
    raps · 25d ago↗ source
  • FDA proposes new framework prioritizing consistency over flexibility for rare disease drug reviews
    The FDA announced a shift in its approach to evaluating rare disease therapies, emphasizing a more uniform assessment process. Agency officials explained that the flexibility shown over the summer…
    Endpoints · 25d agoRegulatoryNeutral↗ source
  • Ultragenyx sells second priority review voucher as Biomea shares tumble after oral GLP-1 data
    Ultragenyx Pharmaceutical, a commercial-stage rare disease company, completed its second sale of a priority review voucher this month. The voucher sale reflects the company's strategy of monetizin…
    Endpoints · 25d agoDealNeutral↗ source
  • Beam Therapeutics reports early Phase 1/2 data for base editing therapy in GSD Ia, partners with Pfizer and Enodia
    Beam Therapeutics disclosed early results from a Phase 1/2 study evaluating a base editing approach aimed at correcting the genetic defect that causes glycogen storage disease type Ia, a rare metab…
    Endpoints · 26d agoPositive readoutNeutral↗ source
  • Syndax halts development of Niktimvo after Phase 2 failure in IPF trial
    Syndax announced it is stopping work on its experimental drug Niktimvo for idiopathic pulmonary fibrosis (IPF) after the compound did not meet its primary endpoints in a placebo-controlled Phase 2…
    Endpoints · 26d agoNegative readoutNegative↗ source
  • Mirum's Livmarli cuts itching in ultra-rare liver disease trial, showing significant benefit
    Mirum Pharmaceuticals reported that its ileal bile acid transporter inhibitor Livmarli achieved a statistically significant reduction in cholestatic pruritus compared with placebo in a late‑stage s…
    Endpoints · 27d agoPositive readoutPositive↗ source
  • AstraZeneca attributes Phase 3 failure of bone disease enzyme therapy to trial design flaws
    AstraZeneca disclosed that its Phase 3 study of an enzyme replacement therapy for a rare genetic bone disorder did not meet its primary objectives. The company said the shortfall was largely due t…
    Endpoints · 28d agoNegative readoutNegative↗ source
  • Nuvig Therapeutics halts Phase 2 trial of NVG-2089 in chronic inflammatory demyelinating polyneuropathy
    Nuvig Therapeutics announced it is terminating one of its two ongoing clinical programs, a Phase 2 study of its candidate NVG-2089. The trial was designed to evaluate NVG-2089 in patients with chr…
    Endpoints · 1mo agoCRLNegative↗ source
  • Shionogi agrees $2 billion upfront purchase of IntraBio, adding oral rare-disease drug Aqneursa to its portfolio
    Shionogi announced it will acquire Austin-based biotech IntraBio for $2 billion paid upfront. The deal brings IntraBio’s oral therapy Aqneursa into Shionogi’s rare‑disease pipeline, expanding its…
    Endpoints · 1mo agoDealPositive↗ source