Aldevron Breakthrough Blog: Gene Editing

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.

Read More

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. 
Read More

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.
Read More

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.

Read More

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.

Read More

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. 

Read More