Genetic Function
Tome Bio founders wrangle jumping genes to create new programmable gene insertion tool
PGI, Technology, Retrotransposons, Clustered Regularly Interspaced Short Palindromic Repeats
AIRNA nets $155M series B to launch clinical test of RNA editing tech
RNA Editing, Airna, AIR-001, RNA
Wave Life Sciences’ WVE-N531 Shows Promise in Duchenne Muscular Dystrophy Treatment
Duchenne muscular dystrophy (DMD), WVE-N531, exon 53 skipping, Phase II trial, dystrophin expression, muscle health improvement, FDA approval
Alnylam’s Amvuttra Approved for ATTR Cardiomyopathy, Challenging Pfizer and BridgeBio
Amvuttra, vutrisiran, ATTR-CM, FDA approval, RNAi therapeutic, cardiomyopathy
Arrowhead finds RNAi kidney disease candidate is well tolerated in phase 1/2 IgAN trial cohort
ARO-C3, IGA Glomerulonephritis, RNA Interference
Pfizer’s Talzenna-Xtandi Combo Shows Significant Survival Benefit in Advanced Prostate Cancer
Talzenna, Xtandi, metastatic castration-resistant prostate cancer (mCRPC), overall survival, TALAPRO-2 trial, PARP inhibitor, homologous recombination repair (HRR) gene mutations
Kura Oncology’s Ziftomenib Achieves Phase 2 Success in AML, Paving Way for Dual-Track Phase 3 Program
Ziftomenib, Acute Myeloid Leukemia (AML), NPM1 mutation, KMT2A rearrangement, Phase 3 trials, KOMET-001, KOMET-017, Menin inhibitor, Breakthrough Therapy Designation
J&J’s Subcutaneous Rybrevant Receives CHMP Backing for Lung Cancer Treatment Despite Recent FDA Rejection
Rybrevant, amivantamab, Johnson & Johnson, CHMP, FDA, subcutaneous formulation, non-small cell lung cancer, EGFR mutations
J&J’s Rybrevant-Lazcluze Combo Demonstrates Superior Survival Benefit Over Tagrisso in EGFR-Mutated NSCLC
Rybrevant, Lazcluze, Tagrisso, NSCLC, EGFR mutation, overall survival, Phase III MARIPOSA trial, chemotherapy-free regimen
Bausch + Lomb Collaborates with City Therapeutics on Novel RNAi-Based Treatment for Geographic Atrophy
Bausch + Lomb, City Therapeutics, RNA interference (RNAi), Geographic atrophy (GA), Age-related macular degeneration (AMD), Strategic collaboration, Ophthalmologic diseases