Genome Editing

Headquartered in Alameda, CA, Scribe develops CRISPR-based treatments through its genetic modification platform, designed to build and apply its suite of CRISPR technologies in therapeutic areas that include neurodegenerative diseases; ophthalmological diseases; multi-system, muscle, and metabolic disorders; and hematopoietic disorders. That platform aims to address safety, delivery, poor editing outcomes, and the long-running bitter legal battle over who invented CRISPR-Cas9. To avoid intellectual property uncertainty, Scribe has engineered its own CRISPR effectors.

Regarding using flexible manufacturing technologies, Kyverna points out that it is an early-stage firm and has work to do before its products are commercialized. Team officials admit that they are not sure what the technology platforms will be in the future, as their footprint has halved in the last six years, so they say they need to stay vigilant to identify and incorporate new advances.

Epigenomic editing in the amygdala can ameliorate adult psychopathology after adolescent alcohol exposure, a new study from researchers at the University of Illinois claims. This study offers insights on what happens in developing brains when they are exposed to high concentrations of alcohol and provides animal model evidence that gene editing may reset the epigenomic reprograming at a critical enhancer element in the amygdala to prevent adult anxiety and alcohol use disorder due to adolescent binge drinking.

The new facility will quadruple Cytiva’s capacity for manufacturing Sefia, Sepax, and Xuri consumables with two new ISO class 7 cleanrooms. The facility’s design adopts GMP principles from raw materials entering the building, through the manufacturing process, to the shipment of finished products. An R&D center is located on the first floor, which consists of several labs designed for multidisciplinary engineering and cell biology processing capabilities.

Avantor reports that it will collaborate with Cytovance Biologics to accelerate plasmid DNA development for biopharma customers. The collaboration is expected to advance plasmid optimization and sourcing services for new and existing viral vector and mRNA-based vaccine and therapeutic customers.

In this GEN webinar, sponsored by WuXi, our distinguished presenters, Dr. Heather Malicki and Dr. Andreas Solomos will discuss the regulatory requirements and challenges involved with bringing new AAV-based gene therapies to market.

On this GEN Live, we’ll be joined by two cell therapy experts whose companies are trailblazing a path in an old field to create new cures.

Matica Bio provides viral vector GMP manufacturing services for cell and gene therapies, vaccines, oncolytic vectors, and other advanced therapy products. Its GMP facility in College Station, TX, is designed for the rapid development, scaleup, and production of viral vectors and cell therapy products for clinical supply. Matica Bio offers process development, GMP production, product release, and stability assessment together with quality oversight and regulatory guidance.

Researchers designed and engineered artificial, genetically tractable, photosynthetic endosymbiosis between photosynthetic cyanobacteria and budding yeasts. Mutants of model photosynthetic cyanobacteria were engineered as endosymbionts within yeast cells where, the engineered cyanobacteria perform bioenergetic functions to support the growth of yeast cells under defined photosynthetic conditions. Potential applications include evolutionary studies, particularly related to organelle evolution, and for synthetic biology.

Adeno-associated virus (AAV) vectors may be the best option to deliver genetic cargo to cells in the body. But they are far from perfect....