Nucleic Acid, Nucleoside, or Nucleotide – Page 4

Biopharma’s New Horizon: Industry Leaders Converge at JPM 2025

4 months ago talkbio0Tagged Biopharma, Biotechnology, Clustered Regularly Interspaced Short Palindromic Repeats, Doudna, Drug Industry, Gene Modification, Health care facility, healthcare, Janet, Jennifer, JPM, Robert Nelsen, Woodcock

JPM 2025, Biopharma, Jennifer Doudna, Janet Woodcock, Robert Nelsen, Scott Gottlieb, Healthcare, Gene Editing, CRISPR, Pharmaceutical Industry, Biotechnology

Editas Medicine Undergoes Major Restructuring, Cutting 65% of Staff and Halting Sickle Cell Therapy Development

4 months ago talkbio0Tagged Cell Therapy, Clustered Regularly Interspaced Short Palindromic Repeats, Editas Medicine, Gene Modification, Industry, restructuring, sickle

Editas Medicine, layoffs, restructuring, sickle cell therapy, CRISPR gene editing, biotech industry

Moderna’s RSV Vaccines Face Safety Concerns Over Severe Infections in Infants

4 months ago talkbio0Tagged Advisory Committees, concerns, Infant, Moderna, Respiratory syncytial virus, RNA, Messenger, Safety, United States Food and Drug Administration, Vaccines

Moderna, RSV vaccines, safety concerns, severe infections, infants, mRNA vaccines, FDA, advisory committee

Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs

5 months ago talkbio0Tagged Arrowhead, Collaboration Agreement, gene therapy, Global Licensing, Hereditary Diseases, Infrequent, Interferes with, RNA, RNA, Small Interfering

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

5 months ago talkbio0Tagged Atherosclerosis, Cardiovascular system, Gene Silencing Therapy, Heart Disease, Interferes with, Lipoprotein(a ), Lp(a ), Muvalaplin, Risk, RNA, zerlasiran

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

5 months ago talkbio0Tagged development aspects, Dyslipidemias, Interferes with, Lipid Metabolism Disorders, RNA, RNA, Small Interfering, Rona, sanofi

siRNA, dyslipidaemia, Rona, Sanofi, RNA interference, lipid disorders, therapeutic development

Emerging Therapies for Huntington’s Disease: A Review of Promising Treatments

5 months ago talkbio0Tagged Antisense, disease-modifying treatments, gene therapy, Huntington 's disease, Oligonucleotides, Stem Cell Therapy

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

6 months ago talkbio0Tagged Duchenne, Exon-Skipping Therapy, Feedback - system communication, Landscapes, Muscular Dystrophy, Muscular Dystrophy, Duchenne, SRP-5051, United States Food and Drug Administration, Vesleteplirsen

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

6 months ago talkbio0Tagged analytical method, Automation, bony process, clinical transition, Control Groups, Materials, Preclinical, Quality, regulatory, Risk Management, RNA, Messenger

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

6 months ago talkbio0Tagged alpha 1-Antitrypsin Deficiency, Alpha-1, Antitrypsin Deficiency, augmentation therapy, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Hereditary Diseases, RNA Interference

Alpha-1 Antitrypsin Deficiency (AATD), Genetic Disorder, New Therapies, Augmentation Therapy, RNA Interference (RNAi), Gene Editing, CRISPR