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1,136 stories matching this filter · ← front page

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  • Taysha and Neurogene close in on first Rett syndrome gene therapy as 2027 readouts loom
    Two companies, Taysha Gene Therapies and Neurogene, are nearing the final stage of development for gene therapies targeting Rett syndrome, a rare neurodevelopmental disorder with limited treatment…
    BioSpace · 14d agoPositive readoutPositive↗ source
  • U.S. biotechs capture 68% of global VC funding since 2025, led by Bay Area and Boston
    Since the start of 2025, U.S. biopharma firms have secured $37.2 billion in venture capital, representing about 68 % of the $54.5 billion raised worldwide. The San Francisco Bay Area and Boston do…
    BioSpace · 14d agoDealNeutral↗ source
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Nephrology757
  • J&J's Halda-derived prostate cancer drug shows 75% response in early Phase 1 trial
    Johnson & Johnson's recent $3 billion purchase of Halda Therapeutics added an oral prostate cancer candidate, JNJ-6143, to its pipeline. The drug targets a cancer-specific protein to trigger cell d…
    BioSpace · 14d agoPositive readoutPositive↗ source
  • Australian clinical trials attract more U.S. sponsors amid regulatory uncertainty
    CROs are reporting faster startup timelines, predictable regulation, and tax incentives as reasons for increasing interest in conducting clinical trials in Australia. State-level incentives in Que…
    BioSpace · 14d agoNeutral↗ 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
  • FDA clears Edwards' tricuspid valve repair device; Bayer's blood thinner asundexian approved in China
    The U.S. Food and Drug Administration has granted approval for Edwards Lifesciences' new tricuspid valve repair system, expanding treatment options for patients with tricuspid regurgitation. In Ch…
    raps · 14d agoApprovalNeutral↗ source
  • TGA opens consultation on temporary rise in Australian medical device annual fees
    The Therapeutic Goods Administration (TGA) in Australia announced a public consultation regarding a proposed temporary increase to the annual charges that medical device manufacturers must pay. Th…
    raps · 14d agoRegulatoryNeutral↗ source
  • ESMO 2026: Merck’s Kelun-partnered ADC cuts death risk by over 30% in endometrial cancer
    Sac-TMT significantly extended overall and progression-free survival for patients with advanced or recurrent endometrial cancer by 6.9 months over chemotherapy in a Phase 3 trial—becoming the first…
    BioSpace · 14d ago↗ source
  • Roche warns Germany faces last chance to regain pharma investment confidence
    Roche has said that recent German healthcare reforms threaten the country's attractiveness for pharmaceutical investment. The company paused its own German investment plans after the reforms were a…
    BioSpace · 17d agoRegulatoryNegative↗ source
  • Marengo's bispecific antibody invikafusp alfa shows tumor shrinkage in 8 cancer types in Phase 2
    Marengo Therapeutics presented Phase 2 data for its bispecific antibody invikafusp alfa at the European Society for Medical Oncology meeting in Madrid. The study, part of the Start-001 first‑in‑hum…
    BioSpace · 17d agoPositive readoutPositive↗ source
  • Alnylam to launch pivotal Huntington’s trial after early study shows strong huntingtin knockdown
    Alnylam Pharmaceuticals announced plans to move its Huntington’s disease RNAi therapy brezosiran into a pivotal, placebo‑controlled trial slated for 2027, pending regulatory alignment. The decisio…
    BioSpace · 17d agoPositive readoutPositive↗ source
  • Tango Therapeutics reports 82% response rate for vopimetostat plus Rasonque in pancreatic cancer, down from 92% earlier
    Tango Therapeutics announced updated data from its ongoing Phase 1/2 trial combining its PRMT5 inhibitor vopimetostat with Revolution Medicines' newly approved drug Rasonque (daraxonrasib) in secon…
    BioSpace · 17d agoNegative readoutNegative↗ source
  • BMS reports fatal septic pneumonia in MS patient during CAR T trial of zolacabtagene autoleucel
    BMS disclosed that a participant in its Phase 1 Breakfree-2 study of the CAR T cell therapy zolacabtagene autoleucel died from septic pneumonia. The patient, who had highly active relapsing multip…
    BioSpace · 17d agoNegative readoutNegative↗ source
  • US proposes new biotech investment limits on China, but licensing deals remain allowed
    The U.S. Treasury announced a proposal to curb outbound investments in Chinese biotechnology, citing national‑security concerns. The plan would limit U.S. persons from funding certain bio‑derived c…
    BioSpace · 17d agoRegulatoryNeutral↗ source
  • FDA lifts hold on Aardvark Therapeutics' ARD-101 after safety study shows no cardiac events
    FDA has lifted the clinical hold on Aardvark Therapeutics' ARD-101 after the company completed an additional safety study. The new data showed no cardiac events, addressing the concerns that prompt…
    BioSpace · 17d agoCRLPositive↗ source
  • Roche bets up to $813M on Zymeworks’ lung cancer trispecific T cell engager
    Roche will pay Zymeworks $55 million upfront plus another $40 million once the partnership asset enters Phase 1b development and up to $718 million in additional milestones.
    BioSpace · 17d agoDealNeutral↗ source
  • Drug safety group petitions FDA to block marketing of unapproved compounded peptides; bipartisan bill seeks clinical trial reforms
    A drug safety advocacy group has filed a citizen petition with the FDA, asking the agency to stop the marketing of compounded peptide products that lack approval. A bipartisan group of lawmakers h…
    raps · 17d agoRegulatoryNeutral↗ source
  • Experts urge cultural shift and standards to unlock real-world evidence potential in medtech
    At The MedTech Conference in Boston, experts highlighted the need for a cultural shift to build trust between regulators and medtech firms when using real-world evidence (RWE) for device marketing.…
    raps · 17d agoNeutral↗ source
  • Gilead shops in IL-23, inking $1.3B upfront I&I deal with China’s PepStar
    Gilead Sciences has picked up a new inflammatory disease program from China’s PepStar Therapeutics to pair with the Phase 2 inflammatory bowel disease asset emvistegrast.
    BioSpace · 17d ago↗ source
  • FDA uncages Aardvark’s Prader-Willi study after new data cleared safety concerns
    After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aa…
    BioSpace · 17d ago↗ source
  • FDA uncages Aardvark’s Prader-Willi study after new data cleared safety concerns
    After cardiac safety signals led to a full clinical hold on Aardvark Therapeutics’ studies of ARD-101, the biotech conducted an additional safety study, in which no cardiac events were detected. Aa…
    BioSpace · 17d ago↗ source
  • FDA approves GSK-Ionis antisense drug Hibsago, opening functional-cure market for hepatitis B
    The U.S. Food and Drug Administration has granted approval to Hibsago (bepirovirsen), a partnership product of GSK and Ionis Pharmaceuticals, marking the first antisense oligonucleotide cleared for…
    BioSpace · 18d agoApprovalPositive↗ source
  • Roivant's brepocitinib cuts steroid use in non-infectious uveitis in Phase 3 trials
    Roivant Sciences and its subsidiary Priovant reported that their TYK2/JAK inhibitor brepocitinib met all primary and secondary endpoints in two identical Phase 3 studies (Clarity‑1 and Clarity‑2) i…
    BioSpace · 18d agoPositive readoutPositive↗ source
  • AbbVie’s HS drug lutikizumab meets Phase 3 goal but falls short of analyst expectations
    AbbVie reported that its investigational IL‑1α/β inhibitor lutikizumab achieved the primary endpoint in the Phase 3 INTREPID study for hidradenitis suppurativa, a chronic inflammatory skin disease…
    BioSpace · 18d agoNegative readoutNegative↗ source
  • FDA approves GSK hepatitis B drug; EQT to acquire Qiagen for $10.8 billion
    The U.S. Food and Drug Administration has granted approval to GlaxoSmithKline's new hepatitis B therapy, marking a regulatory milestone for the company. Private-equity firm EQT announced a definit…
    raps · 18d agoPositive↗ source