Synthego Opens State-of-the-Art GMP Guide RNA Manufacturing Facility to Enable CRISPR Genomic Medicines
Synthego, a leading provider of genome engineering solutions, announced today the opening of its state-of-the-art GMP synthesis facility to enable CRISPR medicines. This new 18,000 square-foot facility supports the growing demand for development of high-quality, CRISPR-enabled in vivo and ex vivo therapeutics by pharma and biopharma clients, including via IND and NDA or BLA enabling clinical studies.
The new facility follows current Good Manufacturing Practices (cGMP) and features 24/7 operations with advanced equipment and technologies, including multiple multiplexed manufacturing lines, fully automated controls, and an integrated Manufacturing Execution System (MES). This enables the company to produce a range of oligonucleotides, including synthetic single guide RNAs, setting the standard for quality and compliance in manufacturing gene editing components. The facility also includes dedicated process development, analytical testing, and quality control areas, ensuring that all aspects of the manufacturing process are closely controlled.
“This facility will be essential in supporting the burgeoning field of clinical phase, first-in-human, CRISPR-enabled medicines both for more common genetic diseases but also for rare diseases affecting children for whom other treatments do not exist for,” said Adrian Thrasher, M.D., Ph.D., a Clinician and Professor of Pediatric Immunology at University College London Great Ormond Street Institute of Child Health, whose laboratory is developing new CRISPR-enabled medicines for children with rare genetic diseases. “As we move into this new generation of cell and gene therapies, it will be crucial that there are strong, reliable manufacturing capabilities that exist for the necessary components of the CRISPR technology – such as the guide RNA – in order to accelerate the path to clinic and treat patients.”
Don Kohn, M.D., Distinguished Professor and Clinician of Pediatric Hematology and Oncology at UCLA, whose laboratory pioneered revolutionary genetic therapies for immunocompromised children, added, “Synthego pioneered the mass production of guide RNAs for discovery research that has enabled many of the CRISPR-based cell and gene therapy trials happening today. Having been a key leader in providing the technology for researchers and clinicians, it follows that they are now expanding their capacity to support this research as it translates into the clinic.”
The new facility has been designed to meet the requirements of both domestic and international regulatory authorities including the FDA, EMA, MHRA, and Health Canada.
“We are pleased to open Synthego’s new GMP facility, solidifying our commitment to provide partners with the highest quality CRISPR guides for the clinical development of therapeutics. This represents a significant step towards removing industry bottlenecks and helping accelerate access to life-changing and lifesaving CRISPR-enabled cell and gene therapies,” said Paul Dabrowski, CEO of Synthego. “The design of this facility is the fruition of a decade of experience creating guide RNAs and engineering cells, and sets the standard for quality and efficiency of GMP CRISPR reagent manufacturing.”
Synthego offers industry-leading, high quality synthetic guide RNAs in three different classes, research grade, GMP-like, and GMP, to support all stages of the therapeutic development process from bench through clinic. With the opening of this facility, Synthego is well-positioned to meet the strong customer demand for custom manufacturing of RNA molecules for development of cell and gene therapeutics, and to continue to provide clients with the highest quality products and services.
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