Synthego has adopted Good Manufacturing Practice standards for producing customized GMP-grade synthetic guide RNA (sgRNA) at its facilities, an initiative the company said will benefit its academic and industry customers as they develop advanced cell and gene therapies.

Starting in the current quarter, Synthego said Tuesday, its GMP-grade sgRNA will be available for ordering in lots of up to 10 grams, based on sequences designed by customers to support their research efforts.

“That is a scale that is generally appropriate for investigators to move into exploratory trials and take that next step into the clinical space,” Jason Steiner, PhD, who leads business development and strategy at Synthego, told GEN Edge. “What we see this as is essentially enabling that transition from a benchtop research facility into that first step in the clinical setting.”

Synthego said its GMP-grade sgRNA encodes target sequences designed by industry and academic partners to support specific scientific research efforts.

“So many of our customers are maybe moving forward into the more clinical application space, and this is an important step for us to provide them with the continuity of products and services to do that,” Steiner added.

Privately-held Synthego has not disclosed its cost of attaining GMP. That effort took up to 12 months, and involved building clean room facilities, calibrating equipment and instrumentation to ensure it is in a validated/qualified state, and adhering to documentation standards that are associated with the quality parameters for GMP.

Synthego’s GMP offerings may move beyond sgRNA, Steiner added; “RNA was our first business, and it’s a business that we’re continuing to develop. We may or may not elect to do GMP manufacturing of cells in the future. But right now, RNA is a critical need in the industry, and one that was a natural next step for our business.”

The growth of cell and gene therapy clinical development and manufacturing was one important factor in Synthego beginning to offer GMP-standard reagents for clinical development. Another is the growth of CRISPR genome editing, Steiner said.

Driven by CRISPR

“CRISPR and genome editing more broadly is a large driver of this in that it enables so many potential things to be done across a wide variety of areas, and it’s such a flexible tool that can essentially be programmed using RNA molecules or other guide molecules,” Steiner said. “We focus a lot on genome engineering and CRISPR specifically, as do our customers. And it has been a large driver of this transition in the space.”

Steiner said Synthego has developed a pipeline portfolio of products and services for the research community that includes reagents for CRISPR editing, and engineered cells.

“Customers use those to do a lot of different kinds of research. Many of them do research in the space of cell and gene therapies,’ Steiner said. “Customers that we work with on the research side have interests in a lot of different applications, and they may simply want the same types of orders but simply in a GMP quality standard.”

Their applications are quite varied, Steiner said, though Synthego does not disclose its customers or the specific applications they are working on. “Any case where an RNA guide might be used might be a potential application. So, this step forward for us is very much in expanding that portfolio that enables our customers to take their research one step further.”

GMP products are intended to adhere to the highest standard of material purity, manufacturing consistency, and quality control through regulations requiring that manufacturers, processors, packagers of drugs, medical devices, some food, and blood proactively ensure that their products are safe, pure, and effective.

GMP standards have been promulgated by the FDA through the Federal Food, Drug, and Cosmetic Act, with the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) working to achieve greater harmonization in standards worldwide through scientific consensus among regulatory and industry experts.

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