Aldevron Blog

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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Cost-effective Cas9 – Higher concentrations bring results

By Mark Osborn, Ph.D., Minnesota Stem Cell Institute, University of Minnesota

CRISPR/Cas9 is a vital part of our research at the University of Minnesota and the Cas9 recombinant protein, used at high concentration, has allowed for highly efficient modification of T-cells. 

By introducing a Cas9 nuclease guide RNA complex (RNP), we target a specific spot in the genome, where the nuclease cuts the DNA. The DNA break is repaired in one of two ways: homologous recombination, which is high-fidelity, or non-homologous endjoining (NHEJ), which is more error-prone.

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Insights from the Gene and Cell Therapy Meeting on the Mesa: 2016

Last month I took part in a workshop on gene and cell therapy manufacturing at the Alliance for Regenerative Medicine’s Cell and Gene Meeting on the Mesa. It was a pleasure to participate with very distinguished colleagues: Matthew Caple, Vice President of Site Operations at Brammer Bio; Mike Kelly, Director, Asset Leadership and Portfolio Management, Gene Therapy, Biogen; and Michael Roberts, Ph.D., Chief Scientific Officer, Synpromics.

The workshop was moderated by Jessica Carmen, Ph.D., Director of Business Development, Cellular Therapy Partnerships, MaxCyte and sponsored by MaxCyte and Brammer Bio. Dr. Carmen did a great job taking us on a journey through manufacturing from construct design through raw material manufacturing, viral vector manufacturing, and ultimately commercialization.

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Using Quick Response Manufacturing (QRM) to Reduce Turnaround Times

"Biomanufacturing" and "efficiency" are two terms that are not commonly seen together, but researchers at the University of Wisconsin-Madison Engineering Department along with protein manufacturing experts here at Aldevron are taking big steps to change that.

The application of Quick Response Manufacturing (QRM) theory to biological processes is a unique initiative whose goal of reducing lead times for manufacturing operations is the core focus of the UW and Aldevron partnership. 

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The Distinction Between Development and Manufacturing is Becoming Blurred

Last month, I participated in a panel discussion at the Rejuvenation Biotechnology 2016 Conference sponsored by the SENS Research Foundation at the Buck Institute for Research on Aging in Novato, California. The title of our panel was "As Paths To Clinic Are Expedited, The Distinction Between Development And Manufacturing Is Becoming Increasingly Blurred".

One of the panelists was Alan Moore, Vice President and Commercial Chief for Biologics and Advanced Therapies of Wuxi AppTec. Alan and I have known each other for some time and it was great to reconnect with him. His presentation covered the impressive work that Wuxi has done in autologous cell processing.

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3 Reasons CRISPR Gene Editing Success Relies on Partnering with an Experienced Manufacturer

Groundbreaking technologies arise every decade or so, advancing genomics research and the development of new medicines. Restriction enzymes in the 70s, polymerase chain reaction in the 80s and next-generation sequencing in the 90s all represented huge leaps forward. Recently, however, the CRISPR revolution has shown the potential to overshadow them all.

CRISPR, or clustered regularly interspaced short palindromic repeats, is a long-sought breakthrough for making highly specific changes to genetic codes. Compared to established technologies such as zinc-finger nucleases (ZFNs) and transcription activator like effector nucleases (TALENs), the CRISPR system using Cas9 nuclease is faster, easier, more precise and more flexible.

CRISPR technology will have great impact on basic and applied life-science research. Therefore, important questions should be asked when choosing a partner that can develop and manufacture CRISPR components such as Cas9 to meet your present and future needs for Cas9 product design, scale and quality.

We’ve reviewed three important reasons why experience and expertise matters when choosing such a partner. 

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