Two studies reveal how 3D genome architecture shapes early human development. One shows enhancers pre-connect with future target genes before activation, while the other finds germ cells restructure chromosomes before meiosis — a step critical for lab-grown sperm and eggs.
Prime Medicine receives a Buy upgrade from H.C. Wainwright after New Zealand clears its first in vivo prime editing trial for Wilson's disease. The FDA also granted RMAT designation to its CGD therapy PM359. Jacob Funds and ARK Investment have recently added to their positions in the gene-editing company.
Researchers developed a lipid nanoparticle gene-editing approach that inserted a full CFTR gene into airway cells and restored 88% to 100% of normal channel function in a cystic fibrosis lab model.
A lipid nanoparticle gene-editing approach inserted a full healthy CFTR gene into human airway cells and restored 88% to 100% of normal CFTR function in lab tests. The nonviral strategy is intended as a mutation-agnostic path for cystic fibrosis.
NVIDIA and Eli Lilly announced a $1 billion AI co-innovation lab as AI drug discovery expands across research, trials and manufacturing. Companies including Recursion and Tempus are using large biological and clinical datasets to speed development and support precision medicine.
Researchers discovered that adding three common amino acids to lipid nanoparticles dramatically improves mRNA and CRISPR gene editing delivery, boosting efficiency up to 20-fold and raising gene editing success rates from 25% to nearly 90%.
Researchers develop multiple strategies to enhance CAR T-cell therapy, including dual PGE2 receptor knockout for solid tumors and small-molecule switches using venetoclax and caffeine to control cellular activity and improve safety.
The FDA has proposed a new "plausible mechanism" pathway to approve customized treatments for rare diseases after testing in only a handful of patients, aiming to remove barriers for gene editing and other bespoke therapies that don't fit traditional approval systems.
The FDA has issued draft guidance that may speed approval pathways for rare disease therapies using genome editing technologies, potentially benefiting CRISPR Therapeutics' pipeline of six clinical-stage candidates.
The FDA has proposed new guidelines to create a pathway for approving bespoke therapies tested in small patient groups, specifically targeting rare genetic conditions and gene editing treatments that don't fit traditional approval systems.
