Genetic Engineering & Biotechnology News

DEC 2018

Genetic Engineering & Biotechnology News (GEN) is the world's most widely read biotech publication. It provides the R&D community with critical information on the tools, technologies, and trends that drive the biotech industry.

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GENengnews.com | Genetic Engineering & Biotechnology News | DECEMBER 2018 | 7 then performing sophisticated edits. "All these things fit together," Dabrowski declares. Our philosophy is to make full-stack genome engineering solutions very accessible to scien- tists." Consequently, he expects researchers to make high-quality genome edits quickly and effectively, saving significant time (possibly several weeks) on each editing project. Advancing Genetic Medicine to the Clinic Dabrowski's overarching goal is to create an ecosystem in which gene editing becomes a clinical tool to actually cure genetic disor- ders. "The first gene and cell therapies are coming on the market now. Most are priced between $800,000 and $1.5 million," he says. Neither patients nor payers can afford them. "We think that if a few areas of the R&D and commercialization process can be changed, a new area of medicine with cures that have costs of $10,000 per patient can be created." By advancing a full-stack genome engineering mindset, he says, results will be more predictable, easier to generate, and will incur fewer delays. In that way, clinical gene and cell therapies will become practical for researchers to develop. There are two steps at which Synthego can help the industry achieve that goal. "The first is R&D, using engineered cells and bio- informatics to choose the correct targets. We have those products," he says. "The second is manufacturing the ther- apy," he continues. That requires cGMP- grade materials. "We're ramping up the abil- ity to create cGMP gRNAs for customers who are looking to go into the clinic." That's especially exciting, he explains, because it suggests the industry surround- ing CRISPR is mature enough to branch out from pure research and begin entering the therapeutic space. That in turn moves gene and cell therapies closer to mainstream use. It doesn't hurt that industry successes also increase Synthego's potential customer base. Synthego 2024 Dabrowski equates gene editing to a type- writer today. Within five years he wants it to be a word processor, with all the inherent improvements in ease of use and efficiency. In 2024, "it would be wonderful to be able to perform any type of genetic manipulation for our customers, perturbing 10 to 20 genes ef- fectively and showing that the cell lines work." On the therapeutic side, Dabrowski wants Synthego to participate in multiple clinical tri- als to help advance gene- and cell-based thera- pies. Forming partnerships is part of the plan, but that, he says, is too early to discuss. "A lot of technical progress needs to be made to continue improving the field," he says. At Synthego, that means more hiring. Its staff has grown roughly 250% in the past 18 months, and that doesn't appear to be slowing. The company is hiring in all depart- ments. "We're looking for qualified, motivat- ed individuals who are in the right place at the right time and are interested in joining a mission-driven company," he says. He's look- ing across the board at IT specialists, biolo- gists, chemists, and others, with the expecta- tion of continuing a similar rate of growth. Supporting such rapid growth risks divert- ing attention from the science to the business systems and processes that may be sidestepped when organizations are small, but that are vi- tal to the smooth operations of larger compa- nies. If Dabrowski is concerned about growth pains, he's keeping it to himself. mirusbio.com ©2018 All rights reserved Mirus Bio LLC. Mirus Bio and TransIT-X2 are registered trademarks and ANY DELIVERY METHOD. ANY NUCLEIC ACID. ANY CELL TYPE is a trademark of Mirus Bio LLC. All trademarks are the property of their respective owners. Move your research forward with a versatile transfection reagent that is the solution for hard-to-transfect and common cell types. Proven and reliable, TransIT-X2 ® allows you to avoid guesswork and get it right the Œrst time. Peak performance in a broad range of cell types Suitable for multiple applications Efficient delivery of plasmid DNA, siRNA/miRNA and CRISPR/Cas9 components Transfection with TransIT-X2 ® Dynamic Delivery System X2 X2 X2 "I was recently tasked with developing a CRIS P R protocol for primar y and bone - derived cell line s. Trans I T-X 2 ® D ynamic Deliver y S ystem was simple to use, 2-3 time s bet ter for transfection and much gentler on my cells than other products! I feel I have hit the jackpot and have already passed this exciting information on to my colleague s." Dr. Joshua Chou, Har vard S chool of Dental M edicine Your Cell Type Your Cell Type Your Nucleic Acid Your Nucleic Acid Request a FREE sample of TransIT-X2: mirusbio.com/sample TM On Your Radar Synthego Location 3696-A Haven Avenue Redwood City, CA 94063 Phone (650) 206-4011 Website www.synthego.com Principal Paul Dabrowski CEO Number of Employees 130 Focus Synthego is a genome engineering company based on CRISPR technol- ogy. It performs editing in-house and also develops tools and cell lines to help researchers make gene and cell modi"cations themselves. Vital Signs

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