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  • FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
    The U.S. Food and Drug Administration today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA (MPS IIIA), also known…
    fda press · 1mo agoApprovalPositive↗ source
  • Novo Nordisk signs up to $1.4 billion deal with Orbis Medicines to develop oral macrocycle cardiometabolic drugs
    Novo Nordisk announced a partnership with Danish biotech Orbis Medicines, valued at up to $1.4 billion, to co-develop oral macrocycle drugs aimed at cardiometabolic diseases. The collaboration wil…
    BioSpace · 1mo agoDealPositive↗ source
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Nephrology645
  • Novartis' $12B Avidity deal falters as DM1 therapy misses Phase 3 endpoint
    Novartis' recent acquisition of Avidity Biosciences was intended to bring a novel therapy for Myotonic dystrophy type 1 (DM1) into its portfolio. The drug, del-desiran, an antibody-oligonucleotide…
    BioSpace · 1mo agoNegative readoutNegative↗ source
  • Biotech sector expected to weather Fed rate hike despite higher capital costs
    The Federal Reserve raised the federal funds rate by a quarter of a percentage point, a move that traditionally weighs on capital‑intensive industries like biotech. Analysts at BMO Capital Markets…
    BioSpace · 1mo agoRegulatoryPositive↗ source
  • Longeveron shares plunge 57% after stem-cell therapy fails in mid-stage heart trial
    Longeveron announced that its investigational stem cell product, laromestrocel, did not meet its primary endpoint in a Phase 2b study of infants with hypoplastic left heart syndrome (HLHS). The tri…
    BioSpace · 1mo agoNegative readoutNegative↗ source
  • Roche commits up to $1 billion to develop bispecific antibodies with Dualitas
    Roche announced a partnership with Dualitas Therapeutics, a California biotech, to advance bispecific antibody programs aimed at immunological and inflammatory diseases. The agreement includes an…
    BioSpace · 1mo agoDealPositive↗ source
  • Biopharma hiring surges in Massachusetts and California, with science/R&D and clinical roles leading demand
    BioSpace data shows that biopharma job postings on its site rose 17% between January and August 2026, signaling a broad increase in employment opportunities. Massachusetts and California dominate…
    BioSpace · 1mo agoNeutral↗ source
  • EU officials outline major pharmaceutical legislative reforms at RAPS Convergence 2026
    At RAPS Convergence 2026 in Charlotte, North Carolina, officials from the European Commission and the European Medicines Agency presented a set of reforms aimed at overhauling the European Union’s…
    raps · 1mo agoRegulatoryPositive↗ source
  • Webinar Explores Manufacturing Risks and Partner Strategies for the Next Five Years
    The webinar titled "Manufacturing in Turbulent Times" is scheduled for September 17, 2026 and is designed to help biopharma sponsors understand the manufacturing challenges that lie ahead. It note…
    BioSpace · 1mo agoNeutral↗ source
  • Senate schedules hearing for FDA nominee as Novo teams with Anthropic on AI drug development
    The Senate has announced a hearing to consider the nominee for the next FDA commissioner, signaling a forthcoming review of the agency's leadership. In parallel, biotech firm Novo has entered a pa…
    raps · 1mo agoRegulatoryNeutral↗ source
  • MHRA AI Commission Issues 44 Recommendations for Future UK Regulatory Framework
    A commission established by the Medicines and Healthcare products Regulatory Agency has released its findings. The group presented 44 recommendations aimed at shaping the United Kingdom’s regulato…
    raps · 1mo agoRegulatoryNeutral↗ source
  • Former FDA chief Califf calls for regulatory overhaul and predicts AI assistants in future oversight
    Robert Califf, who served as FDA commissioner, reflected on his early experience as a medical student in the 1970s working with a pioneering patient database that tracked cardiovascular procedures…
    raps · 1mo agoRegulatoryNeutral↗ source
  • Experts discuss wet AMD treatment limits and need for longer lasting therapies
    In a recent episode of the Denatured podcast, Professor Philip Jacob Rosenfeld of the University of Miami and Wesley Jackson, chief science officer at Valitor, joined host Jennifer C. Smith-Parker…
    BioSpace · 1mo agoNeutral↗ source
  • Over 15,000 Science/R&D jobs and 10,800 Clinical roles posted on BioSpace in first eight months of 2026
    BioSpace data shows seven biopharma disciplines each had at least 5,000 live postings during the first eight months of 2026. Science/R&D leads with 15,361 positions, ranging from manager roles at…
    BioSpace · 1mo agoNeutral↗ source
  • FDA officials stress administration priorities in draft PDUFA VIII commitment letter
    At a public meeting on Wednesday, FDA officials presented the proposed commitment letter for the upcoming PDUFA VIII program. The letter reiterates the administration's priorities, reflecting the…
    raps · 1mo agoRegulatoryNeutral↗ source
  • Novartis drops $125M for Sironax’s tech to cross blood-brain barrier
    Novartis stands alongside Big Pharma peers that have similarly invested in technologies to penetrate the blood-brain barrier, including GSK, Roche and Eli Lilly.
    BioSpace · 1mo ago↗ source
  • Psychedelic therapeutics near first FDA approval as Compass Pathways and Definium report promising results
    BioSpace notes that the psychedelic drug sector is regaining momentum after a setback in 2024 when Lykos’ MDMA therapy failed to win FDA clearance. The focus has shifted to new candidates, with Com…
    BioSpace · 1mo agoRegulatoryPositive↗ source
  • FDA holds psychedelic forum as Definium reports third successful Phase 3 trial of oral LSD for anxiety
    The FDA convened a public forum on Monday to discuss the near‑term outlook for psychedelic drug development, highlighting interest from companies such as Compass Pathways and Definium Therapeutics.…
    BioSpace · 1mo agoPositive readoutPositive↗ source
  • FDA issues warning letter to Bausch & Lomb over aseptic processing concerns
    FDA sent a warning letter to Bausch & Lomb after inspectors reviewed the company’s response to earlier observations at its Florida plant. The agency said the response did not demonstrate a commitme…
    BioSpace · 1mo agoRegulatoryNegative↗ source
  • Sling Therapeutics raises $123M Series C to advance oral thyroid eye disease drug
    Sling Therapeutics raised $123 million in a Series C financing round to move its oral drug candidate for thyroid eye disease forward. The capital will fund development of linsitinib, the only late…
    BioSpace · 1mo agoDealPositive↗ source
  • Biotech firms race to hire AI-savvy scientists as talent gap widens
    AI is becoming integral to biotech research and development, prompting companies to seek staff who can blend biology with machine learning and data engineering. Recruiters say the hardest-to-find…
    BioSpace · 1mo agoNeutral↗ source
  • Biotech firms increase product launches as capital markets improve
    A growing number of biotech companies are bringing new medicines to market, a trend analysts link to improving capital markets. While the majority of approved drugs still come from large pharmaceu…
    BioSpace · 1mo agoPositive↗ source
  • Experts urge more representative enrollment in clinical trials
    A panel of regulators and industry leaders gathered to discuss how clinical trials can better reflect the diversity of patient populations. The discussion included officials from the U.S. Food and…
    raps · 1mo agoRegulatoryNeutral↗ source
  • Novartis stops ALS TREM2 stabilizer after Phase 2 miss, marking fourth trial setback in a month
    Novartis has halted development of its TREM2 stabilizer VHB937, also known as lifonebart, for amyotrophic lateral sclerosis after the drug failed to meet either the primary or secondary endpoints i…
    BioSpace · 1mo agoNegative readoutNegative↗ source
  • Experts assess EU medical device and diagnostic regulations at RAPS Convergence 2026
    A panel of experts representing the European Commission, industry groups and notified bodies gathered in Charlotte, North Carolina during RAPS Convergence 2026. The group reviewed the current stat…
    raps · 1mo agoRegulatoryNeutral↗ source