Nucleic Acid, Nucleoside, or Nucleotide

Atsena, Retinoschisis, Juvenile, X-Linked, United States Food and Drug Administration, Dependovirus, Cloning Vectors, Hereditary, Retinal Diseases, Blindness, ATSN-201, ATSN-101

AIRNA nets $155M series B to launch clinical test of RNA editing tech

3 weeks ago talkbio0Tagged AIR-001, Airna, RNA, RNA Editing

RNA Editing, Airna, AIR-001, RNA

Solbinsiran Demonstrates Significant Lipid Reductions in Phase 2 Trial for Mixed Dyslipidemia

3 weeks ago talkbio0Tagged Apolipoproteins B, Eli, Eli Lilly, LDL-C, RNA, Small Interfering, solbinsiran, Triglycerides

Solbinsiran, siRNA, mixed dyslipidemia, Eli Lilly, PROLONG-ANG3, apolipoprotein B, LDL-C, triglycerides

Solbinsiran: Eli Lilly’s Promising ANGPTL3 Inhibitor for Lipid Management

3 weeks ago talkbio0Tagged ANGPTL3, Dyslipidemias, Lipids, Psychological inhibition, RNA, Small Interfering, solbinsiran

ANGPTL3 inhibition, solbinsiran, dyslipidemia, lipid reduction, siRNA therapy

Lilly’s Lepodisiran Shows Durable Lipoprotein(a) Reduction in Phase 2 Trial

3 weeks ago talkbio0Tagged Cardiovascular Diseases, Interferes with, Lepodisiran, long-acting therapy, Lp(a ), RNA

lepodisiran, lipoprotein(a), Lp(a), cardiovascular disease, RNA interference, long-acting therapy

Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy

1 month ago talkbio0Tagged Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Infrequent, Liver diseases, Primary hyperoxaluria, type I

Gene editing, CRISPR, Rare liver disease, Primary hyperoxaluria type 1 (PH1), Funding

Avidity’s Del-zota Shows Promise in Duchenne Muscular Dystrophy Treatment

1 month ago talkbio0Tagged Creatine Kinase, Del-zota, DMD, Dystrophin, exon, Muscular Dystrophy, Duchenne, production, RNA, safety profile, skipping

Del-zota, Duchenne muscular dystrophy (DMD), RNA therapeutics, exon 44 skipping, dystrophin production, creatine kinase reduction, safety profile