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FDA Initiates Approval of Leucovorin for Cerebral Folate Deficiency Amid Autism Debate
The FDA is advancing approval for leucovorin calcium tablets to treat cerebral folate deficiency (CFD), a neurological disorder linked to developmental delays and autistic features. This move...
Breakthrough CRISPR-Enhanced CAR T Therapies Show Promise Against Multiple Myeloma
Innovative studies reveal that targeted gene knockouts via CRISPR technology significantly enhance CAR T cell therapeutic efficacy in multiple myeloma models. These genetic alterations improve...
Smart Wearable Pad Detects Disease Biomarkers via Menstrual Blood
Researchers at ETH Zürich developed MenstruAI, a wearable sensor-integrated menstrual pad that detects disease-related molecules directly in menstrual blood via a colorimetric lateral flow assay....
UniQure’s Gene Therapy Shows Major Market Impact With FDA Filing Plans
Following a pivotal Phase I/II success demonstrating substantial slowing of Huntington’s disease, UniQure plans regulatory submission in early 2026, potentially marking the first approved gene...
Novel CRISPR-Enabled Screening Accelerates Genome Editing Efficiency
Recent advancements harness natural CRISPR-associated transposons (CASTs) to improve one-step, targetable genome editing with enhanced biomedical applicability. Screening methods have been...
AmplifyMD Raises $20M to Expand AI-Driven Virtual Specialty Care Platform
AmplifyMD secured a $20 million Series B funding round to scale its AI-powered virtual specialty care platform and physician network. Key investor and customer Memorial Hermann Health System...
Human Cell Atlas Identifies Fibroblast Subtypes With Therapeutic Targets
The Human Cell Atlas project mapped distinct fibroblast subpopulations in healthy and diseased skin via single-cell sequencing and spatial transcriptomics, uncovering six subtypes in normal tissue...
UniQure's Gene Therapy Slows Huntington’s Disease by 75%
UniQure has reported groundbreaking phase I/II trial results for its gene therapy AMT-130 targeting Huntington’s disease. The treatment achieved a 75% reduction in disease progression over three...
CRISPR Screening Enhances CAR-T Therapies for Multiple Myeloma
Researchers at Mass General Brigham and the Broad Institute have leveraged in vivo CRISPR screens to discover genetic modifications that improve the persistence and function of CAR T cells in...
FDA Initiates Approval of Leucovorin for Cerebral Folate Deficiency Affecting Autism
The FDA has begun the approval process for leucovorin calcium tablets as a treatment for cerebral folate deficiency (CFD), a neurological condition linked to developmental delays and autistic...
Acadia Pharmaceuticals Drops Prader-Willi Syndrome Drug After Phase III Failure
Acadia Pharmaceuticals announced the termination of development for ACP-101, an intranasal therapy targeting hyperphagia in Prader-Willi syndrome patients, following a phase III trial that failed...
Sanofi Boosts Corporate Venture Capital Arm with $625 Million Investment
Sanofi has infused an additional $625 million into its corporate venture capital arm, Sanofi Ventures, bringing total funds to $1.4 billion. This strategic move aims to strengthen investments in...
Lonza and RION Collaborate to Scale Exosome-Based Therapeutics Manufacturing
Lonza has partnered with RION Biosciences to provide cGMP manufacturing and technical support for commercial-scale production of RION’s purified exosome product (PEP) drug substance. This...
AI Drives Merck Collaboration to Discover Small Molecules for Challenging Targets
Merck & Co. has entered a $349 million deal with Variational AI to apply generative AI technology in designing and optimizing small molecule drug candidates against two undisclosed therapeutic...
Immuneering Reports Promising Phase 2 Data for MEK Inhibitor in Pancreatic Cancer
Immuneering announced encouraging mid-stage clinical trial results for atebimetinib, its oral MEK inhibitor for pancreatic cancer. The combination of atebimetinib with chemotherapy yielded an 86%...
Scanogen Receives $2.9 Million NIH Grant for Rapid Bloodstream Yeast Infection Test
Diagnostics firm Scanogen secured a $2.9 million grant from the U.S. National Institute of Allergy and Infectious Diseases to advance development of a culture-free, rapid test for bloodstream...
Leveraging AI and Robotics to Accelerate Life Science Discoveries at Lila Sciences
Lila Sciences raised $235 million in Series A funding to scale its AI-driven scientific discovery platform that integrates generative AI, robotics, and automated lab systems. The company aims to...
UniQure’s Gene Therapy Slows Huntington’s Progression by 75%
UniQure reported pivotal Phase 1/2 trial results showing its gene therapy AMT-130 significantly slowed Huntington’s disease progression by 75% over three years. This rare neurodegenerative...
FDA Rejects Scholar Rock’s SMA Drug Over Manufacturing Issues
The FDA issued a complete response letter (CRL) to Scholar Rock’s selective anti-latent myostatin antibody apitegromab, intended for spinal muscular atrophy (SMA), citing unresolved quality...
Pfizer Reenters Obesity Market with $4.9B Metsera Acquisition
Pfizer announced a $4.9 billion deal to acquire Metsera, regaining a foothold in the obesity treatment space. Metsera’s pipeline includes oral and injectable incretin-based therapies aimed to...