The 38th JPMorgan Healthcare Conference took place earlier this month in San Francisco, California. It is considered a key investor event for the healthcare industry. The Biotech Showcase, co-produced by Demy Colton and EBD Group, was held in conjunction with the Conference.
At the Conference, Synthego, a self-described full-stack (“expresses the ability to understand, assemble, and control software code from core basics through the complexities of final products”) genome editing company started by two former SpaceX engineers, announced it has adopted Good Manufacturing Practice (GMP) standards to broaden the usage of its synthetic guide RNA (sgRNA) into clinical R&D. The Synthego offering cuts across large-scale research use only and intermediate GMP-like solutions, to fully certified GMP sgRNA material, starting at the end of the first quarter 2020. In 2019, the company closed a $119 million Series C round and already serves the academic and industrial market segments.
“With thousands of known genetic disorders impacting millions of people and no available treatments for them, it is critical for our industry to safely develop a new generation of accessible therapies,” said Paul Dabrowski, CEO and co-founder of Synthego. “Offering GMP products for use in genome editing brings us one step closer to our mission of making agile, precision R&D accessible to therapeutic developers. We are proud to collaborate with industry and academic institutions as they bring innovative treatments from the lab to clinical trials.”
After the announcement Synthego invited Dr. Daniel Dever, a Stanford instructor in Pediatrics & Stem Cell Transplantation, to share his translational research on therapeutic genome editing utilizing tools from Synthego, IDT and Agilent Technologies to replace disease causing genes with non-disease causing genes in hematopoietic stem cells. Dr. Dever is currently heading an Investigational New Drug (IND) enabling study for FDA approval for the first human trials to treat severe sickle cell disease using CRISPR-Cas9-based targeting techniques.
Another firm that is growing is Maravai Life Sciences’ company TriLink BioTechnologies, a specialist in nucleic acid manufacturing, which announced the grand opening of its new cGMP facility in November 2019 to address the demand for mRNA (messangerRNA) therapies. The new $40 million facility boasts a 50,000 square foot manufacturing space. End-markets for the company’s mRNA and custom oligonucleotides include research, diagnostic, therapeutic companies and researchers. The company also offers OEM products. The company offers CleanCap technology for the production of synthetic mRNA. “Capping is a critical process in creating viable mRNA constructs that remain biologically active without eliciting immune responses,” explains Anton McCaffrey, PhD, senior director of Emerging Science and Innovation for TriLink BioTechnologies. Earlier this month, the company announced two new patents for the technology.
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