Nucleic Acid, Nucleoside, or Nucleotide
Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs
Sarepta Therapeutics, Arrowhead Pharmaceuticals, siRNA programs, Global Licensing and Collaboration Agreement, Rare genetic diseases, Gene therapy, RNA interference
Breakthrough in Heart Disease Treatment: Lilly’s Muvalaplin and Silence Therapeutics’ Zerlasiran Show Promising Results in Lowering Lipoprotein(a) Levels
Lipoprotein(a) (Lp(a)), Heart Disease, Muvalaplin, Zerlasiran, Cardiovascular Risk, Gene Silencing Therapy, RNA Interference, Atherosclerotic Cardiovascular Disease
Rona’s Sanofi-Sourced siRNA Demonstrates Early Efficacy in Dyslipidaemia Treatment
siRNA, dyslipidaemia, Rona, Sanofi, RNA interference, lipid disorders, therapeutic development
Emerging Therapies for Huntington’s Disease: A Review of Promising Treatments
Huntington’s disease, gene therapy, antisense oligonucleotides, stem cell therapy, disease-modifying treatments
Sarepta Therapeutics Halts Development of Vesleteplirsen, a Next-Generation Exon-Skipping Therapy for Duchenne Muscular Dystrophy
Sarepta Therapeutics, Vesleteplirsen (SRP-5051), Duchenne Muscular Dystrophy (DMD), Exon-Skipping Therapy, FDA Feedback, Therapeutic Landscape
Mitigating Risks in mRNA Manufacturing: Strategies for Smooth Transition from Preclinical to Clinical Stages
mRNA manufacturing, risk management, preclinical to clinical transition, quality control, regulatory compliance, raw material selection, process automation, analytical methods.
Revolutionizing AATD Treatment: Emerging Therapies to Replace Augmentation Therapy
Alpha-1 Antitrypsin Deficiency (AATD), Genetic Disorder, New Therapies, Augmentation Therapy, RNA Interference (RNAi), Gene Editing, CRISPR
Intellia’s CRISPR Therapy NTLA-2002 Shows 81% Reduction in Hereditary Angioedema Attacks in Phase 2 Study
CRISPR therapy, Intellia Therapeutics, NTLA-2002, Hereditary Angioedema (HAE), Gene editing, Phase 2 study, Swelling attacks, Functional cure
Intellia’s CRISPR Gene Editing Therapy Shows Promising Results in Reducing HAE Attacks
Intellia Therapeutics, CRISPR Gene Editing, Hereditary Angioedema (HAE), NTLA-2002, Clinical Trials, Gene Therapy, Rare Diseases
Roche and Dyno Therapeutics Launch $1B+ Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Neurological Diseases
Roche, Dyno Therapeutics, AAV gene therapy vectors, Neurological diseases, AI-driven gene delivery, LEAP technology, Gene therapy collaboration