Aldevron Breakthrough Blog: Protein

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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How New FDA Guidelines Can Affect Cell Therapy Development, part 2

The effect on autologous and allogeneic therapies projects

In my previous post on new draft FDA guidelines that concern cell therapies, I covered key control considerations for CRISPR reagents and the GMP manufacturing considerations for drug substances affected by that guidance. In this post, I’ll go over a couple approaches to consider when working with gene-modified cell therapies, and how they could be affected.

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How New FDA Guidelines Can Affect Cell Therapy Development, Part 1

Defining control considerations for CRISPR reagents is key

The FDA recently released draft guidance on Investigational New Drug (IND) applications for Human Gene Therapy Drug Products Incorporating Human Genome Editing technology to edit human somatic cells. This much-anticipated draft highlights manufacturing, quality, and regulatory considerations for drug developers preparing IND filings for novel, gene-modified cell therapies.

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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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Growing Agricultural Productivity with CRISPR-Cas9

When we talk about the use of CRISPR-Cas9 technology, it’s usually in the context of developing treatments for human diseases. But there’s another aspect to the technology that has potential to have just as much impact on our lives: its use in the genetic modification of food crops. 

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