Gene editing technology advances with new assembly methods and in-vivo targets

Advancements in genomic engineering are expanding the potential for universal gene therapies. A new method called ‘prime assembly’, detailed in the journal Nature, allows for the precise insertion of long DNA fragments into living cells. Building on existing prime editing techniques, this single-step process uses DNA flaps to tether matching fragments to specific genomic locations. This approach aims to correct complex genetic diseases involving multiple mutations with a single method while reducing the risk of off-target effects and toxicity associated with untargeted insertion.

In the commercial sector, CRISPR Therapeutics is working to evolve its gene-editing capabilities. While the company currently has one approved medicine, Casgevy, for sickle cell disease and transfusion-dependent beta thalassemia, the treatment requires a complex logistical workflow involving cell collection and bone marrow clearing. The company is developing in-vivo editing processes to transform these treatments into standardized infusions, which could expand the addressable patient population from 150,000 to 400,000. Additionally, its CTX460 program, which utilizes SyNTase editing to correct mutations rather than just deactivating genes, has entered phase 1 trials to treat alpha-1 antitrypsin deficiency.