Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

The Phase 1/2 trial is a first-in-human, open-label, multi-national study designed to evaluate the safety, tolerability, and efficacy of TSRA-196 in adults with AATD. Trial participants will receive a ...

A new gene-editing technique enables the correction of multiple genetic mutations simultaneously, transforming the prospects for millions living with complex inherited diseases, such as cystic ...

Scientists have discovered a new CRISPR mechanism with precise activity, expanding the potential applications of the existing CRISPR toolbox.

Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.

Data Demonstrate Potent in vivo Gene Editing of Hematopoietic Stem Cells (HSCs) in the Bone Marrow with Systemic Delivery in Preclinical Murine Models Using Fusogen Technology Broadens Application of ...

As CRISPR moves from lab success to real-world medicine, 2026 will test whether these revolutionary gene-editing therapies can reach the patients who need them most or remain costly, complex ...

Gene editing is now reaching the mainstream, ushering in a new era of genetic manipulation. Traditionally, inserting or deleting entire genes, regulating their expression, and altering specific ...

Advancements in genome editing technologies the growing emphasis on safe and accurate gene correction, and strong biotechnology R&D investment drive the base editing market.Austin, Texas, Jan. 21, ...