Standardized materials: Meeting the demand for gene therapies

For years, manufacturers have customized bioprocessing out of necessity. Whereas pharma has had time to develop efficient pathways to market, the newness of biotechnology approaches such as CRISPR gene editing has required a bespoke approach for things such as materials and process development.

As a result, cell and gene therapy (CGT) manufacturers have long worked from scratch, which has led to scale-up challenges: With supply and labor constraints creating backlogged custom-manufacturing schedules, developers have felt the pain in cost and speed to market in light of the demand for these emerging products.

At the same time, CGT’s popularity has generated new opportunities and guideposts for standardization. With several approved therapies already in market and another 10 to 20 expected each year by 2025, manufacturers can now benefit from existing commercialization pathways by learning from others whose products got there first.

In turn, many biotech development and manufacturing workflows that previously couldn’t be standardized can now move forward — and using off-the-shelf materials with the flexibility to choose between research- or GMP-grade is an efficient way to turn these time-critical programs into templates. Grade-specific products from the same vendor can smooth the transition from bench to bedside by avoiding rework, technology transfer, and scale-up challenges as product demand increases.

Whatever your goals for your product, you might not need a custom-manufactured solution to get there. Here are two materials you can usually get pre-made — at the right grade — and how they each help.

A note on material grades

While off-the-shelf biotech products have been around for years, supply chains have recently advanced by making them available across different grades. Research grade makes bench applications more affordable, while high-quality cGMP grade facilitates an efficient transition to clinical trials and beyond.

Using the right grades at the right times for the right needs supports a better workflow end-to-end. Manufacturers can more easily and affordably move candidates from research or preclinical phases into clinical or commercial stages without depending on custom materials that will inevitably fluctuate with market pressures such as supply or labor shortages.

Packaging and helper plasmid DNA

Packaging and helper plasmids are necessary for viral vector programs to facilitate gene structuring and viral replication. Having plasmids such as pALD-X80 available in specific quantities for research and GMP grade can fast-track workflows without having to utilize an expensive and time-consuming custom manufacturing process.  

In addition, standardized solutions can give another benefit beyond that of cost or time savings — that of making emerging trends in plasmid production more accessible to more manufacturers. For example, suppliers are increasingly offering systems without antibiotic markers or bovine-derived RNase in off-the-shelf products. These standardized products can help preempt regulatory and other concerns while giving manufacturers a head start on their bioprocessing.

CRISPR-associated nucleases

The ability to procure CRISPR-associated nucleases off-the-shelf at multiple grades stands to broaden gene editing’s footprint even more in the years ahead. When nuclease systems are more standardized, they become more cost-effective and, thus, more accessible along the pathway from research to commercialization. Moreover, nuclease systems are evolving with new options becoming commercially available, as exemplified by the recent partnership between Aldevron and Inscripta to offer a Type-V nuclease as a standard research grade and GMP catalog item with a planned launch date in 2022.

Standardized materials can also help support gene-editing products’ compliance with evolving Food and Drug Administration requirements. Recent draft guidance, for example, proposes the use of high-fidelity nucleases as one opportunity to minimize off-target effects — which are an increasing concern of CRISPR technology.

Standardizing for faster go-to-market

Ultimately, the influx of CGT products has led to an inflection point in bioprocessing. While the novelty of these programs has for years required a custom approach to manufacturing, their increasing prevalence has provided more opportunities to standardize while simultaneously generate more demand than ever before. Off-the-shelf materials that are grade-specific offer a flexible resource in the race to scale up and go to market, not just for speed but also cost efficiency.

As the science evolves and new therapies enter the market, even more grab-and-go products will undoubtedly be worth adding to your workflow — such as GMP-grade in vitro transcription enzymes for mRNA production. But for now, grade-specific plasmids and CRISPR nucleases are certainly worth the look.

Looking for other ways to standardize processes and accelerate your path to market? Stay tuned for Aldevron’s next post, where we’ll explore how drug master files associated with off-the-shelf CRISPR and plasmid DNA materials can save valuable time on your IND.

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