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STAT+: Uniqure’s Huntington’s Gene Therapy Marks Breakthrough Progress
UniQure’s investigational gene therapy, AMT-130, achieved a 75% reduction in disease progression for Huntington’s disease after three years, representing the most compelling data in the field so...
Unprecedented Gene Therapy Success Slows Huntington’s Disease Progression
UniQure NV announced promising Phase I/II clinical trial results for its gene therapy AMT-130 targeting Huntington’s disease, demonstrating a significant 75% slowing in disease progression after...
Acadia Pharma Halts Prader-Willi Drug After Phase III Failure
Acadia Pharmaceuticals discontinued development of ACP-101, an intranasal therapy aimed at mitigating hyperphagia in patients with Prader-Willi syndrome (PWS), after its 12-week phase III trial...
AI Advances Propel Drug Discovery and Cancer Immunotherapy
Multiple high-impact breakthroughs demonstrate artificial intelligence’s growing role in biopharma innovation. The Arc Institute’s Germinal platform couples AlphaFold-Multimer structural...
FDA Issues Setbacks and Strategic Shifts in Biopharma Pipeline
The FDA posed a complete response letter to Biogen for a high-dose formulation of Spinraza demanding more manufacturing data, underscoring regulatory scrutiny in spinal muscular atrophy treatment...
Novel Biomarkers and Diagnostic Tools Emerging in Oncology and Infectious Diseases
Recent studies introduce cutting-edge molecular tools and biomarkers expanding diagnostic and therapeutic precision. A gene panel predicting response to CDK4/6 inhibitors in breast cancer could...
Breakthroughs in Regenerative Medicine and Cell Therapy Platforms
Multiple innovations promise to transform regenerative treatments. A novel engineered biphasic scaffold has been developed to promote bone regeneration without growth factors, marking a potential...
Major Step in Pandemic Preparedness and Infectious Disease Surveillance
The University of Ottawa secured $3 million federal funding to lead infectious disease research enhancing Canada’s readiness for future pandemics. Similarly, the Global Virus Network initiated a...
Cancer Research Yields New Molecular Targets and Therapies
Cutting-edge research reveals molecular insights that may translate into improved cancer treatments. Studies show that deletion of the Trp53 gene promotes immune evasion and tumor progression in...
Innovations in AI and Robotics Enhance Healthcare and Biotech Research
Advances in artificial intelligence and robotic technologies are reshaping biomedical research and healthcare delivery. Virginia Tech researchers developed an efficient method to separate...
uniQure’s Gene Therapy Slows Huntington’s Disease by 75% in Phase I/II Trial
UniQure has reported groundbreaking results from its pivotal Phase I/II study of AMT-130 gene therapy for Huntington’s disease, demonstrating a 75 percent slowing of disease progression at 36...
Acadia Pharma Terminates Phase III Prader-Willi Syndrome Drug Development
Acadia Pharmaceuticals announced the discontinuation of its investigational intranasal carbetocin (ACP-101) following a failed Phase III clinical trial to treat hyperphagia in patients with...
CRISPR Screening Identifies Genetic Enhancements for CAR T Cell Therapies
Researchers at Massachusetts General Brigham and the Broad Institute used in vivo CRISPR screening to uncover gene targets that enhance chimeric antigen receptor (CAR) T cell function and...
FDA Initiates Approval of Leucovorin for Cerebral Folate Deficiency Amid Autism Debate
The FDA has initiated approval of leucovorin calcium tablets, a generic form of folinic acid, for treatment of cerebral folate deficiency, a neurological disorder affecting folate transport to the...
Sanofi Ventures Boosts Biotech Investment With $625 Million Funding
Sanofi has strengthened its corporate venture capital arm, Sanofi Ventures, by injecting an additional $625 million in committed capital, increasing its fund to $1.4 billion. This financial boost...
Moderna Expands mRNA Research Hub in Australia Amid Global Funding Shifts
In contrast to the scaling back of mRNA vaccine funding in the United States, Moderna is increasing investment in Australia, establishing a regional research and manufacturing hub. This strategy...
Advanced AI Drug Discovery Platform Lila Sciences Secures $235 Million
Lila Sciences has raised $235 million in a Series A financing round to scale its AI-driven scientific research platform that integrates generative AI, robotics, and automated lab systems. The...
New Liquid Metal Nanocomposites Enhance Cancer Photoimmunotherapy
Researchers at the Japan Advanced Institute of Science and Technology have developed multifunctional liquid metal nanocomposites incorporating lactic acid bacteria components to improve...
US Government Allocates £50 Million R&D Fund to Support Life Sciences
The UK government has announced a £50 million research and development fund aimed at addressing concerns raised by biopharma companies about the country’s life sciences environment. This funding...
UniQure’s Gene Therapy Slows Huntington’s Disease Progression by 75%
UniQure has reported pivotal Phase I/II clinical trial results for AMT-130, a gene therapy targeting Huntington’s disease, demonstrating a significant 75% slowing in disease progression over 36...