Gene Editing News
Gene editing is transforming the landscape of medicine, driving new approaches to treat genetic disorders, cancers, and other complex diseases. This page delivers a curated selection of the most important updates in gene editing drug development, highlighting breakthroughs in research, clinical trials, and regulatory news. Stay up to date on the key developments shaping the future of precision medicine.
Gene Editing News
Gene editing is transforming the landscape of medicine, driving new approaches to treat genetic disorders, cancers, and other complex diseases. This page delivers a curated selection of the most important updates in gene editing drug development, highlighting breakthroughs in research, clinical trials, and regulatory news. Stay up to date on the key developments shaping the future of precision medicine.
Inconsistent assay performance is a common but often overlooked cause of batch failures. Robust, reproducible analytical and bioanalytical assays help reduce risk, improve decision-making, and support reliable batch release across drug development and manufacturing...
A new prime editing strategy, PERT, enables disease-agnostic treatment of nonsense mutations by permanently converting an endogenous tRNA into a suppressor tRNA, with major implications for scalable gene-editing manufacturing and regulation...
Avance Biosciences’ NGS Center of Excellence delivers comprehensive sequencing solutions, including short-read, long-read, and advanced single-cell platforms. Our regulatory-compliant workflows support biologics, gene, and cell therapy programs with high-quality data, enabling informed development...
To support IND-enabling studies, a biotech company needed precise measurement of in vivo gene-editing efficiency in preclinical rat tissues. Avance Biosciences delivered a fully validated NGS amplicon sequencing assay capable of detecting edits as low as 1%, enabling accurate quantification across multiple tissues and guiding data-driven development decisions.
Verve Therapeutics’ VERVE-102 shows early success in lowering LDL cholesterol by 55% with a single dose, offering a potential one-time gene editing treatment for familial hypercholesterolemia...
Beam Therapeutics announces groundbreaking results from its Phase 1/2 trial of BEAM-302, demonstrating the first-ever clinical genetic correction for Alpha-1 Antitrypsin Deficiency (AATD), offering hope for a potential one-time cure for this genetic disorder...
TIGR (Tandem Interspaced Guide RNA) systems use RNA guides to target specific DNA sites, offering a compact and modular alternative to CRISPR. Scientists at MIT and the Broad Institute discovered over 20,000 TIGR-associated proteins, some capable of precise DNA cutting, potentially expanding genome...
Avance Biosciences is excited to announce its participation in the upcoming 2nd CRISPR-Based Therapy Analytical Development Summit, taking place from September 17-19, 2024, in Boston, MA. This premier event is a key gathering for professionals and innovators in the gene editing and CRISPR fields.[...]
Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel. Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia...
The guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing and testing, nonclinical safety...