Aldevron Breakthrough Blog: Gene Editing

Eureca-Vᵀᴹ: What is it?

Meet a new catalog CRISPR protein

If you were at the Advanced Therapies Week in Miami from January 17-20, you learned first-hand about our newest catalog CRISPR protein, Eureca-V™ Nuclease. But what exactly is Eureca-V? In short, the core technology for Eureca-V is the proprietary MAD7® nuclease developed by Inscripta®. Aldevron and Inscripta have partnered to make this protein broadly available to the therapeutic, diagnostic, and agricultural research markets. 

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Combining Resources to Streamline and Standardize Therapies

Academics and manufacturing making processes better

Witnessing firsthand the growth of cell and gene therapy to progress more programs to the bedside is an exciting time in the biotechnology industry, particularly with new partnership approaches being developed. I was able to get an overview of one particular partnership during a podcast featuring Tom Foti, VP of Protein Business unit at Aldevron, and Kris Saha, Ph.D., Associate Professor of Biomedical Engineering, Medical History and Bioethics at the University of Wisconsin.

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Podcast: Insights into the Editing of the Human Genome

Where Can Novel Nonviral Polymeric Delivery Agents Take Us?

Collaboration has always been an important part of scientific research and discovery, especially when you combine the resources of strategic partners in science, clinical, engineering and manufacturing.

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DMFs for CRISPR

Applying an old concept to CRISPR applications

Historically speaking, drug master files (DMF) have been used in filing Investigational New Drug applications (IND). However, they’ve never been used for CRISPR because that’s a relatively new therapy. With new draft FDA guidance relating to cell therapies using CRISPR, we’re moving further ahead into a new frontier of genomic medicine.

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The Benefits of Streamlining and Standardizing Plasmid DNA for Cell and Gene Therapies

Manufacturing standardization for cell and gene therapies (C&GT) has received increased focus in the last several years, spurred by the rapid exponential growth witnessed in this field. The resulting need for improved access to high-quality raw materials, including plasmid DNA, has made standardization integral to streamlining the development process for this biologic product category. 
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The Pivotal Role of Plasmid DNA

Plasmid DNA has historically been key to the development of biologic drug manufacturing. Today, it plays a critical role in the production of next-generation cell and gene therapies and vaccines. With our nearly 23 years of plasmid DNA manufacturing expertise, Aldevron has helped facilitate the advance of these important therapeutics. We continue to invest in additional capacity and novel capabilities to support biopharma manufacturers into the future.
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Contributing to a Cure for Sickle Cell Disease

Every year, an estimated 300,000 children are born globally with a severe form of sickle cell disease. This is a genetic disease that causes red blood cells to be a sickle shape, leading to episodes of pain and anaemia, along with the potential of stroke or kidney damage.

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Efficient, reproducible results with Cas9

By Krishanu Saha, Ph.D., Department of Biomedical Engineering & Wisconsin Institute for Discovery, University of Wisconsin-Madison

At the University of Wisconsin - Madison, one focus of our group is understanding and optimizing CRISPR-Cas9 gene editing for therapeutic and disease modeling applications. To conduct our research, we need reliable, consistent and highly efficient Cas9 protein.

Model system for Cas9 gene editing
To perform targeted gene editing, the Cas9 protein, which cuts the genome, and a guide RNA (gRNA) that encodes where in the genome to cut, need to be complexed together into a ribonuclear protein (RNP) complex and transfected to cells to reach the nucleus. Once the DNA is cut, imprecise DNA repair may cause disruption at the cut site, which can result in knock out of a gene.

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