Genetic Engineering & Biotechnology News

MAY1 2015

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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Repositioning Old Drugs for New Uses Could CRISPR be the Magic Bullet? To continue to move the technology forward, scientists recently converged at the CRISPR Preci- sion Gene Editing Congress to discuss unmet needs and new fndings. The event, which took place in Boston, devoted particular attention to overcom- ing specifcity, effciency, and delivery challenges associated with the CRISPR/Cas9 system. Many of these challenges relate to the mecha- nisms a cell may use to repair CRISPR/Cas-induced double-strand breaks (DSBs). A cell has two path- way choices. Non-homologous end joining (NHEJ), an error-prone ligation process, can result in small insertions and deletions (indels) at cleavage sites, whereas homology-directed repair (HDR) employs homologous DNA sequences as templates to make specifc changes for precise repair. In most cells, NHEJ performs the majority of repair events. Identifying and minimizing off-target events are major challenges. To meet these challenges, the Alt laboratory at Boston Children's Hospital developed high-throughput genome translocation sequenc- ing (HTGTS), an enzyme- and target-agnostic technique to rapidly expose potential off-target problems. Frederick W. Alt, Ph.D., and col- leagues recently described the technique in an article see page 18 Database-intensive drug repositioning eforts are integrating "hard" data (chemical structures and pharmacological activities) and "soft" context- based information (developer statements and analyst assessments). Research scientists and tool suppliers in the life sciences continue to devote resources to CRISPR, which is still a relatively new tool. Much is still unknown, and the community needs a deeper understanding of the technology to better harness CRISPR for discovery and development as well as eventual clinical applications. A different and possibly less objectionable ap- proach to extending intellectual property is drug re- positioning. It fnds new uses for active pharmaceuti- cal ingredients (APIs) that were originally developed for different applications. The practice is older than the term, says Vienna- based consultant, Hermann Mucke, Ph.D., who has worked on several prominent repositionings. He lik- ens drug respositioning to Inuit-style whaling. With both practices, the idea is to use every part of the resource. Conventional drug devel- opment, in contrast, could be said Agnes Shanley For years, pharma's critics have decried the industry's practice of "evergreening," or extending drug patent life, arguing that it results in expensive and less inno- vative products. However, the desire to wrest value out of drug development costs, pegged at $2.6 billion in Tufts' latest fgures, might be understandable. MaryAnn Labant The research community's rapid acceptance of the CRISPR/Cas technology is propelling a stage of deep investment in technol- ogy development. Already, three companies have emerged focus- ing on CRISPR therapeutic applications: Intellia Therapeutics, Editas Medicine, and CRISPR Therapeutics. see page 32 TO O L S T E C H N O LO G I E S T E C H N I Q U E S May 1, 2015 Sticky Ends... One Size Doesn't Fit All 6 Brain Projects Get Researchers Thinking Big 7 Deciphering Immune Cell Chatter 12 Bring Mycoplasma Under Control 28 Quality, Yield Still Rule Protein Production 24 Thermo Fisher Scientifc / VasilySmirnov

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