Genome Editing

The temporary suspensions affect the Phase I/II HGB-206 trial, an ongoing, open-label study designed to evaluate the efficacy and safety of LentiGlobin gene therapy for sickle cell disease (SCD); and the Phase III HGB-210 trial, an ongoing single-arm open-label study designed to evaluate the efficacy and safety of LentiGlobin in patients between two years and 50 years of age with SCD.

Focusing on a 28-amino-acid segment, investigators generated more than 200,000 variants of the AAV2 wildtype sequence, which yielded some 110,000 viable engineered capsids, half of which surpassed the average diversity of natural AAV serotype sequences with 12-29 mutations across the region.

Rentschler is collaborating with the Cell and Gene Therapy Catapult to facilitate and accelerate the creation of a new initiative in one of Europe’s largest cell and gene therapy hubs.

A team from Google Research, Harvard’s Wyss Institute, and Dyno Therapeutics has demonstrated the use of AI to generate unprecedented diversity in AAV capsids. In doing so, they overcame one of AAVs limitations by identifying functional variants capable of evading the immune system.

The key to increasing the efficiency of bioprocessing is developing robust platform processes with enhanced productivity and recovery that maintain safety and quality profiles.

Genopis operates a specialized, 500-liter-capacity fermentation line for the manufacture and purification of pDNA. Further small-scale fermentation and downstream processing facilities for pDNA are currently under construction at Genopis’ site in San Diego.

Entegris is committed to its customers’ success. Their vast knowledge base assists in establishing repeatable safe and highly effective processes for the successful qualification of SUT in complex workflows, especially cold processing and cryogenic operations. These attributes have been critical to manage the optimization, qualification, and scale-up of Aramus 2D assemblies utilized in the bulk drug distribution workflow of approved COVID therapies.

Sangamo’s hemophilia A gene therapy giroctocogene fitelparvovec, now overseen by Pfizer, leads a pipeline of 17 wholly-owned and partnered genomic medicines—four in clinical phases; the rest, preclinical.

AskBio founder R. Jude Samulski talks about the evolution and future of AAV gene therapy following his company’s blockbuster acquisition by Bayer in 2020.

New Vectura site has been designed to enable growth in the critical transfection reagent demand from exponential increase of the gene and cell therapy sector.