Aldevron Breakthrough Blog

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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Rare Disease and a Repeatable Process

Of all the presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, a presentation by renowned physician and geneticist Dr. Francis Collins caught our attention. In his talk, Dr. Collins spotlighted an encouraging path forward in the treatment of rare diseases with cell and gene therapies. While acknowledging that gene therapy is being taken to new heights in many areas, he noted there are still thousands of diseases that have not yet had a focus for treatment development.

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Unique Perspectives, Unique Opportunities

TIDES USA pivots to RNA technology

We often hear how interest in, and use of, RNA technology is growing because of the COVID-19 pandemic. One discussion at the TIDES USA conference had a unique perspective: how Moore’s Law may now apply to RNA technology.

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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The FDA recently released draft guidance on Investigational New Drug (IND) applications for Human Gene Therapy Drug Products Incorporating Human Genome Editing technology to edit human somatic cells.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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Lessons Learned & Looking to the Future

Thoughts from the World Vaccine Congress

Managing the H1N1 outbreak in 2009 was considered a practice run for the recent SARS-CoV2 pandemic and, over the last two years, we certainly learned a great deal in being prepared for the next virus to gain a foothold. That was the backdrop as we returned to the World Vaccine Congress in Washington, DC.

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Seeking Faster Paths to the Clinic

Need for speed emphasized at American Biomanufacturing Summit

The American Biomanufacturing Summit, held in San Francisco in April, is a senior-level event attracting over 200 biopharmaceutical executives sharing the latest information from the industry. One top item consistently discussed was how the expectations of speed have been dramatically changed after how quickly COVID-19 vaccines were developed and moved to market, most particularly the mRNA vaccines.

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100-Day Review – From Research to Clinic, and a Pet Project

Insider Insights Inspire Ideas

I’ve spent most of the last 30 years in marketing and sales for molecular biology research products. During that time, I’ve been responsible for software platforms and instrumentation, but my main focus has been on reagents and consumables – nucleic acid purification, PCR, cloning tools, and so forth.

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Webinar On Demand: mRNA/saRNA Manufacturing - Considerations to Accelerate the Path to Clinic

GEN webinar featuring Dr. Venkata Indurthi, Aldevron Chief Scientific Officer

Messenger RNA (mRNA) and self-amplifying RNA (saRNA) offer a unique opportunity for the development of novel therapeutics to treat a wide range of diseases. These modalities offer distinctive advantages over traditional therapies, from both biological and production standpoints. The success of RNA programs may depend heavily on the timeline to manufacture RNA molecules.

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A Complete Suite of rAAV Constructs

New ITR Cloning and Reporter Constructs Now Available
By Jeff Briganti, Senior Director, Global Strategic Marketing

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.

For recombinant adeno-associated virus (rAAV) viral vector production, we now offer several off-the-shelf ITR cloning backbones and GFP reporter constructs to complement our standardized set of OTS helper and packaging plasmids. This product set is available license and royalty free for research through commercial rAAV applications.

Utilizing this standardized portfolio can provide significant time and cost reduction, expediting time to market and de-risking specialized, transformative therapeutic development. By partnering with a contract development and manufacturing organization (CDMO) offering a complete suite of plasmids for viral vector production, researchers can streamline their upfront development while minimizing supply chain disruption throughout their program’s lifecycle.

For full information about our new product offering or to request a sample, visit our pALD-ITR Products page.

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