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Synthego has opened its new GMP-compliant RNA manufacturing facility in Redwood City, Calif., for CRISPR-enabled genomics therapeutics.
Synthego, a US-based provider of genome engineering solutions, has opened its new good manufacturing practice (GMP) synthesis facility. According to a May 11, 2023 company press release, the 18,000-ft2 facility will support the growing demand for the development of high-quality, clustered regularly interspaced short palindromic repeats (CRISPR)-enabled in-vivo and ex-vivo therapeutics. Services at the new facility will also include investigational new drug-, new drug application-, and biologics license application-enabling clinical studies.
The new facility features multiple multiplexed manufacturing lines, fully automated controls, and an integrated manufacturing execution system. The company will have the capability to produce a range of oligonucleotides, including synthetic single guide RNAs. The facility also includes dedicated process development, analytical testing, and quality control areas, which will ensure that all aspects of the manufacturing process are closely controlled. The facility meets the requirements of both domestic and international regulatory authorities including FDA, the European Medicines Agency, the Medicines and Healthcare products Regulatory Agency, and Health Canada, according to the press release.
“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,” said Adrian Thrasher, a clinician and professor of Pediatric Immunology at University College London Great Ormond Street Institute of Child Health, in the press release. Thrasher’s 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," Thrasher said in the release.
“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,” said Don Kohn, distinguished professor and clinician of Pediatric Hematology and Oncology at University of California, Los Angeles. Kohn’s laboratory pioneered genetic therapies for immunocompromised children.