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Novel Tumor Suppressor Mechanism Identified in Blood Cancer
Scientists using genome-wide CRISPR screens have uncovered the GATOR1 complex as a critical tumor suppressor in Myc-driven lymphoma. This regulatory axis impedes cancer progression by modulating...
FDA Clears Ionis’ Drug for Hereditary Angioedema
The U.S. Food and Drug Administration approved Dawnzera, Ionis Pharmaceuticals' medication for hereditary angioedema, marking the third new drug approved this year for the rare genetic condition....
Superluminal and Eli Lilly Partner on Obesity Drugs with $1.3 Billion Pact
Superluminal Medicines, a Boston-based biotech specializing in AI-powered small molecule drug discovery, has entered a substantial collaboration with pharmaceutical giant Eli Lilly worth up to...
FDA Lifts Clinical Hold on Rocket Pharmaceuticals’ Gene Therapy Trial
Rocket Pharmaceuticals received FDA clearance to resume its Phase II gene therapy trial for Danon disease following a clinical hold prompted by a patient death. The trial will proceed at a lower...
Targeted mRNA-LNP Delivery Advances Therapeutics Beyond Vaccines
Monash University researchers have developed a targeted mRNA lipid nanoparticle (LNP) delivery system that improves cell specificity, overcoming a key limitation of conventional LNPs which lack...
Parkinson’s Therapy Improves with Neuromodulation and Wearable Monitoring
Innovative approaches in Parkinson’s disease management include the use of non-invasive neuromodulation devices and wearable technologies. Stimvia’s Uris device delivering transcutaneous...
Novel Antibiotic Structures Designed Using Generative AI Model
Researchers at the Massachusetts Institute of Technology developed a generative artificial intelligence model capable of designing novel antibiotic structures de novo and from chemical fragments....
Rocket Pharma Resumes Danon Gene Therapy Trial with FDA Approval
Rocket Pharmaceuticals has cleared a significant hurdle by obtaining FDA approval to resume its Phase 2 clinical trial of the RP-A501 gene therapy for Danon disease following a clinical hold...
Superluminal and Eli Lilly’s $1.3 Billion Collaboration Targets Cardiometabolic and Obesity Drugs
Superluminal Medicines announced a major partnership with Eli Lilly valued at up to $1.3 billion that will focus on discovering small molecule therapeutics against G protein-coupled receptors...
Graphene-Based Neuromodulation Accelerates Brain Organoid Development and Neuronal Function
Researchers at the University of California, San Diego, have developed Graphene-Mediated Optical Stimulation (GraMOS), a novel, non-genetic method utilizing graphene's optoelectronic properties to...
FDA Rejects PTC Therapeutics’ Friedreich’s Ataxia Drug, Urges Further Studies
The U.S. Food and Drug Administration has issued a complete response letter for PTC Therapeutics’ 15-lipoxygenase inhibitor, vatiquinone, intended for treating Friedreich’s ataxia, citing...
AI Integration Transforms Biopharmaceutical Development and Manufacturing
Biopharma industry leaders embrace AI-driven lab-in-the-loop workflows to consolidate compound design, ADME profiling, and compound management into unified, digitally enabled drug discovery...
Sarepta Therapeutics Restructures Amid Milestone Payment and Pipeline Shifts
Sarepta Therapeutics has activated a $100 million milestone payment to Arrowhead Pharmaceuticals following positive safety data in its SRP-1003 myotonic dystrophy program. To finance this, Sarepta...
Genedrive Newborn Hearing Loss Test Gains Funding and NHS Backing in UK
The Scottish government has allocated £800,000 to deploy the Genedrive MT-RNR1 ID Kit across neonatal units to identify genetic risk for hearing loss caused by gentamicin antibiotic treatment....
New Therapeutic Antibody Model Enhances Antibody Design with AI
Researchers at Zhejiang University have developed S²ALM, a Sequence-Structure multi-level pre-trained Antibody Language Model, an advanced AI framework to innovate antibody engineering. This model...
Viking Therapeutics’ Oral Obesity Drug Faces Challenges Due to Side Effects
Viking Therapeutics reported mixed Phase 2 data for its GLP-1/GIP dual agonist oral obesity treatment, achieving target weight losses but encountering up to 38% discontinuation rates mainly due to...
AI-Enhanced Mental Health and Neonatal Care Innovations Emerge
Artificial intelligence is increasingly shaping healthcare interventions, from AI-driven models predicting pulmonary hemorrhage in preterm infants to digital therapeutics reducing suicide attempt...
Graphene-Driven Brain Organoid Growth Accelerates Disease Modeling
Researchers from the University of California San Diego have developed a novel graphene-mediated optical stimulation method (GraMOS) to safely and effectively accelerate the maturation of human...
Rocket Pharmaceuticals Resumes Danon Disease Gene Therapy Trial
Rocket Pharmaceuticals has received FDA clearance to resume its Phase 2 gene therapy trial for Danon disease, a rare genetic disorder affecting multiple organs. The clinical hold issued in May,...
Viking Therapeutics’ Oral Obesity Drug Shows Mixed Phase 2 Results
Viking Therapeutics’ oral weight loss drug VK2735 demonstrated statistically significant reductions in body weight in a Phase 2 trial, with up to 12.2% weight loss in 13 weeks. However,...