Gene Silencing/Gene Editing
News on CRISPR, gene editing, RNAi in genetics, genomics, and molecular diagnostics.
FDA Clears Intellia Therapeutics to Start Phase III Trial of CRISPR-Based ATTR Amyloidosis Therapy
NTLA-2001 is the first in vivo CRISPR-based gene-editing therapy to enter late-stage clinical development in the US, according to the firm.
MyoGene Bio Nets Additional Investment From CureDuchenne Ventures for CRISPR-Based Treatment
The Los Angeles-based biotech will use the funds to develop a gene editing approach targeting the DMD hotspot region where many disease-linked mutations occur.
Sirnaomics Launches Phase I/II Trial of RNAi Therapeutic for Facial Skin Cancer Lesions
The company will look for biomarkers in the TGF-B1 and COX-2 genes, which are overexpressed in squamous cell skin cancer.
Ideaya Biosciences, Broad Institute Partner on Synthetic Lethality-Based Cancer Drug Discovery
The Broad Institute's CRISPR paralog screening platform will inform Ideaya's synthetic lethality-based target and biomarker discoveries.
The company's approach combines whole-genome sequencing, CRISPR, liquid biopsy, and gene therapy to target cancer cells with gene fusions.