Genome Editing

New research published by teams from Duke University Medical Center and Stanford University describes modular, programmable live RNA-sensing technologies with broad applications in biology, biotechnology, and RNA medicine.

Ginkgo Bioworks announced the acquisition of two companies—the French company Altar and California-based Circularis. Altar will bring their automated adaptive laboratory evolution (ALE) instruments—which carry their proprietary adaptive evolution platform—to Ginkgo's Foundry in Boston. And Circularis, which has a proprietary circular RNA and promoter screening platform, strengthens Ginkgo's platform for development of cell and gene therapies.

During this GEN webinar, our distinguished guest Dr. Bernardo Cordovez will tell us more about an instrument—Aura^® GT—that his company created to characterize gene therapies for stability and CQAs using a minuscule amount of sample.

LogicBio Therapeutics, a clinical-stage genomic medicine company, announced on Monday that the company is being acquired by Alexion, AstraZeneca Rare Disease. The company has both a gene editing technology platform and an AAV delivery system. LogicBio focuses on the development of gene therapy solutions for pediatric patients with rare diseases. The first drug in the company’s pipeline is for methylmalonic acidemia, a life-threatening condition affecting newborns.

Graphite Bio is pioneering a precision gene editing approach that has the potential to transform health by achieving one of medicine’s most elusive goals: to precisely “find & replace” any gene in the genome. GEN Edge talked through Graphite Bio’s underlying strategy to develop several genetic cures with CEO Josh Lehrer, MD, MPhil, FACC.

Sanofi will apply Scribe’s CRISPR genome editing technologies, designed to enable genetic modification of novel natural killer (NK) cell therapies for cancer. Sanofi has been granted non-exclusive rights to Scribe’s proprietary CRISPR platform of wholly owned enzymes for creating ex vivo NK cell therapies. Scribe has developed a suite of custom engineering genome editing and delivery tools called CasX-Editors, based on the CasX enzyme and other novel foundations.

Without spelling out a specific amount, Prime said net proceeds from the IPO would in part fund continued research and development (R&D) of its immediate target indications, including through achieving preclinical proof-of-concept in several unspecified target indications. Prime also plans to use part of its proceeds for general corporate purposes, as well as to develop its early-stage manufacturing processes and build out a dedicated chemistry facility for its medicinal chemistry, process development, and analytical chemistry groups.

Massachusetts General Hospital (MGH) researchers created a system called Split Prime Editing (Split-PE). These results and reagents have important implications for improving prime editing and should help to accelerate the optimization and application of prime editing for research and therapeutic uses. The research article titled “Engineered CRISPR prime editors with compact, untethered reverse transcriptases” was published on September 26, 2022, in Nature Biotechnology.

Aarhus University researchers harnessed CRISPR-Cas9 gene editing technology and somatic cell nuclear transfer cloning to generate Göttingen minipigs carrying a SORL1 gene mutation, which develop signs of Alzheimer’s disease as young adults. The researchers say the cloned pigs support the notion that SORL1 mutation can be causal for AD. Studying the animals could potentially help to identify new biomarkers of preclinical Alzheimer’s in humans, and to unlock future treatment strategies for the disease.

Bluebird’s latest regulatory nod was an FDA Accelerated Approval following Priority Review for Skysona^® (elivaldogene autotemcel)—which the company also abbreviates as “eli-cel”—a one-time treatment designed to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD).