Gene Editing News

20 05, 2026

GUIDE-seq and NGS-based off-target analysis support genome editing safety assessment for engineered Treg therapy

2026-05-20T20:33:05+00:00

GentiBio recently presented genome editing safety data during the “On- and Off-Target Method Development” session st the 2026 ASGCT Meeting, highlighting the extensive off-target characterization strategy used to support development of GNTI-122, an engineered regulatory T cell (Treg) therapy for type 1 diabetes (T1D). The presentation is particularly timely given the recent FDA [...]

GUIDE-seq and NGS-based off-target analysis support genome editing safety assessment for engineered Treg therapy2026-05-20T20:33:05+00:00
6 01, 2026

How Consistent Assay Performance Reduces Batch Failures

2026-04-22T18:04:12+00:00

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...

How Consistent Assay Performance Reduces Batch Failures2026-04-22T18:04:12+00:00
30 12, 2025

What PERT Signals About the Future of Gene Editing Manufacturing and Regulation

2026-04-22T18:04:25+00:00

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...

What PERT Signals About the Future of Gene Editing Manufacturing and Regulation2026-04-22T18:04:25+00:00
3 09, 2025

Avance Biosciences NGS Center of Excellence: Comprehensive Sequencing Solutions for Biologics, Gene, and Cell Therapy Development

2025-12-24T14:43:18+00:00

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...

Avance Biosciences NGS Center of Excellence: Comprehensive Sequencing Solutions for Biologics, Gene, and Cell Therapy Development2025-12-24T14:43:18+00:00
12 08, 2025

Case Study: Quantifying In Vivo Gene Editing in Preclinical Tissues via NGS Amplicon-Seq

2025-12-24T14:46:02+00:00

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.

Case Study: Quantifying In Vivo Gene Editing in Preclinical Tissues via NGS Amplicon-Seq2025-12-24T14:46:02+00:00
6 05, 2025

Verve Therapeutics Reports Promising Results for PCSK9 Gene Editing in HeFH

2026-04-22T18:04:54+00:00

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...

Verve Therapeutics Reports Promising Results for PCSK9 Gene Editing in HeFH2026-04-22T18:04:54+00:00
11 03, 2025

Breakthrough in Genetic Medicine: Beam Therapeutics Shows Potential with BEAM-302 for Alpha-1 Antitrypsin Deficiency

2026-04-22T18:03:53+00:00

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...

Breakthrough in Genetic Medicine: Beam Therapeutics Shows Potential with BEAM-302 for Alpha-1 Antitrypsin Deficiency2026-04-22T18:03:53+00:00
4 03, 2025

New RNA-Guided System Uncovered, Offering Promise for Precision Gene Editing Therapies

2026-04-22T18:04:02+00:00

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...

New RNA-Guided System Uncovered, Offering Promise for Precision Gene Editing Therapies2026-04-22T18:04:02+00:00
17 09, 2024

CRISPR-Based Therapy Analytical Development Summit – 2024

2026-04-22T18:03:45+00:00

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.[...]

CRISPR-Based Therapy Analytical Development Summit – 20242026-04-22T18:03:45+00:00
20 08, 2024

World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England

2026-04-22T18:04:42+00:00

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...

World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England2026-04-22T18:04:42+00:00
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