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Silence Therapeutics’ siRNA Therapeutic Divesiran Achieves 88% Response Rate in Phase II Trial for Rare Blood Cancer Polycythemia Vera
BioXconomy UK Overview Silence Therapeutics announced positive interim Phase II results from its SANRECO trial for divesiran, an siRNA therapeutic targeting polycythemia vera (PV), a rare blood cancer. The trial successfully met its prim... -
Korro Bio Accelerates RNA Editing Platform into Clinic with First-in-Human Trial for Hyperammonemia and Designation of AATD Candidate
GlobeNewswire (via Korro Bio, Inc.) USA Overview Korro Bio has announced plans to initiate first-in-human clinical trials for its hyperammonemia treatment candidate, KRRO-121, in late 2026. Concurrently, the company designated KRRO-111 a... -
ProQR Therapeutics Marks Landmark: Axiomer RNA Editing Platform Achieves First Clinical Validation for Cholestatic Liver Disease Therapy
Quartr Sweden Overview ProQR Therapeutics has reported the first clinical validation of its proprietary Axiomer RNA editing platform, achieved through Phase 1 target engagement data for AX-0810. This candidate demonstrated modulation of ... -
ReCode Therapeutics Secures Funding, Partners with Gene Editing Firm to Fast-Track Cystic Fibrosis Gene Therapy with SORT LNP Platform
BioSpace (via Business Wire) USA Overview ReCode Therapeutics has announced new funding from the Cystic Fibrosis Foundation and a strategic partnership with a leading gene editing company. These initiatives will accelerate the developmen... -
Peking University Unveils SynTar LNPs: A Precision Breakthrough for Targeted mRNA Lung Delivery
SINOPEG China Overview Researchers at Peking University have developed an innovative SynTar lipid nanoparticle (SynTar LNP) platform, overcoming a critical bottleneck in targeted mRNA delivery to the lungs. This system significantly enha... -
AI-Powered In Silico Design Revolutionizes RNA-LNP Therapies for Targeted Delivery and CRISPR Gene Editing
InSilicoMinds USA Overview In silico (computational) design is fundamentally transforming the development of lipid nanoparticle (LNP) and RNA therapeutics by enabling the precise optimization of LNP properties for tissue-specific drug de... -
Nanoparticle Breakthroughs: Piercing the Blood-Brain Barrier to Revolutionize CNS Disease Treatment, Including Glioblastoma
MDPI Switzerland Overview Nanoparticle-based drug delivery systems are emerging as a pivotal solution for overcoming the blood-brain barrier (BBB), a critical challenge in treating central nervous system (CNS) diseases. A recent review h... -
Unlocking mRNA LNP Design: Cholesterol Found Unnecessary for Non-Hepatic Delivery
ACS Nano USA Overview New research thoroughly dissects the functional roles of individual lipid components within mRNA lipid nanoparticles (LNPs), revealing that while ionizable lipids are critical for delivery efficacy, cholesterol is e... -
Researchers Uncover Key Cellular Uptake Pathway for Antisense Therapies, Paving Way for Enhanced Efficacy
ecancer Global Overview Researchers have made a significant discovery, identifying the crucial intracellular uptake pathway for antisense oligonucleotides (ASOs), a therapeutic approach to halt aberrant protein production in various dise... -
Ractigen Therapeutics Completes Phase II Enrollment for SOD1-ALS siRNA RAG-17; LiCO Therapy RAG-18 for DMD Receives Rare Pediatric Disease Designation
Facebook China Overview Ractigen Therapeutics announced the completion of patient enrollment and first dosing in its Phase II clinical trial for RAG-17, an investigational siRNA therapy for SOD1-mutated amyotrophic lateral sclerosis (ALS...
