After Cancer Cases, Bluebird Bio Suspends Two Sickle Cell Trials

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.
3D illustration XY-chromosomes as a concept for human biology medical symbol gene therapy or microbiology genetics research

AI Gets Gene Therapy Over the Immunity Hump

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.
Medical Technician Preparing Stem Cells

Rentschler Establishes U.K. Center of Excellence for Cell and Gene Therapy

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.

AI Generates Unprecedented Diversity of AAV Capsids

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.
Bioreactor

Getting Bioprocessing Up to Speed

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

Wacker Chemie Acquires Plasmid DNA Manufacturer Genopis

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

Webinar Review: Single-Use System Design for Cryogenic and Cold Temperature Operations

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 CEO Ponders Pipeline, Reflects on Phase III ‘Proud Moment’

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.
Genetic engineering concept. DNA. Gene therapy. Medical technology.

AAV Jude: A Conversation with AskBio’s Jude Samulski

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.
Female research team studying DNA samples. Computer screens with DNA helix in foreground

Polyplus-Transfection Begins Construction of 4,000-Square-Meter Facility

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.

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