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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Genetic Engineering & Biotechnology News | | MAY 1, 2015 | 33 lona-based SOM Biotech, established in 2009, uses its own software based on molecular, not structural, analogs, to fnd other compounds with similar activities, says Nuria Reig, Ph.D., the company's R&D manager. SOM Biotech's virtual, ligand-based tech- nology can screen over 10,000 compounds, and it has developed a list of compounds that determine activity. With venture capital fund- ing from industrial and private investors, the company has evaluated 50 drugs so far, and it is running clinical trials on treatments for orphan diseases. "Interim results are positive, and we hope to present them at BIO in June," notes Dr. Reig. The company intends to of- fer licenses of Phase III-ready drugs to Big Pharma. SOM Biotech's goal is to complete 25 projects and generate six patents per year. A Focus on Validation In Palo Alto, CA, NuMedii, established in 2008 with venture capital backing, is using a technology platform that encompasses genom- ics, network-based methods, machine learning, and chemoinformatics to examine data. Origi- nally established by cofounders Atul Butte, M.D., Ph.D., and Gini Deshpande, Ph.D., at Stanford University, it examines disease classi- fcations on the molecular level. "The thinking was, if you could fnd diseases with molecular similarity, then you should be able to do the same for treatments," explains Dr. Deshpande, the company's CEO. Gene expression profling technology is at the heart of the platform, she says, and so far, over 300 diseases are represented in its data- base, ready to mine. "Our focus is not to look at where the drug would be used," Dr. Desh- pande points out, "but to examine the molecu- lar profle of the disease and where each drug's activities lie to fnd where the two intersect." Through a partnership arrangement, Nu- Medii uses Thomson Reuters MetaCore and Integrity content, as well as Thomson Reuters Life Sciences Professional Services expertise in systems biology, in conjunction with its unique Big Data technology and databases, to fnd FDA-approved drugs or discontinued development compounds that are appropriate for repurposing. NuMedii's technology has been validated, preclinically, Dr. Deshpande says. One proj- ect is an antiseizure drug that could be used to treat Crohn's disease; others are treat- ments for lung cancer and ulcerative colitis. At this point, the company's focus is on validating information rather than licensing its technology. "Big Pharma is focused more on new chemical entities," says Dr. Desh- pande, "But what we are doing with new bio- logics can be validated, and we are exploring discussions with pharma around novel mech- anisms." That way, she says, one can validate the biology in patients, then use that as a basis for new drug development. Rare Pursuits Across the pond in Cambridge, Healx started up last year to apply machine learn- ing and bioinformatics to drug repositioning. The company uses a cloud-based platform developed by Andreas Bender, Ph.D., lec- turer at Cambridge University, which is also alma mater for CEO Tim Guilliams, Ph.D., a specialist in biophysical neuroscience. "The tricky issue for this feld is access to data in the right format," Dr. Guilliams says. Healx is working with both public and con- fdential databases. So far, the company has assembled 180 databases with information on treatments for over 400 diseases. "Of our top predictions, so far, 80 have been validat- ed," Dr. Guilliams asserts. The company has recruited blue-chip inves- tors and executives, including CSO David Ca- valla, Ph.D., a drug repositioning expert, and most recently a senior executive from Roche. "It is easy to make predictions, but making meaningful products is hard, so we have re- cruited experts with 30 years of pharma expe- rience to help with this," explains Dr. Cavalla. The company plans to operate as a virtual company, working with CROs and academic labs on in vitro and in vivo and preclinical studies. Healx is also looking at generic and off-patent drugs. "We are testing the frst batch of com- pounds that would regulate specifc genes that aren't suffciently expressed," notes Dr. Guilliams. "We predicted that certain com- pounds would work. We are also looking at downstream effects, and each repositioning involves a model based on that case. "Small biotech is very interested in our platform, but in talks with Big Pharma, we found that companies were extremely reluc- tant to open up. So we turned the model up- side down, and went frst to those with the biggest interest in rare disease treatments: patient groups and charities." The company will also use a "social enter- prise" model, directing part of its profts to those groups. "I personally think it is the way forward, and that pharma got it wrong for a long time," Dr. Guilliams insists. The company, which has ties with rare diseases groups in the U.S., has established the Cambridge Rare Disease Network. It has also helped identify a rare glycosylation disor- der, NGLY defciency, in efforts to fnd target compounds to treat a six-year-old boy in the U.S. who suffers from the disorder. Despite all the progress being made in drug repositioning, current clinical trial prac- tice is a limiting factor, Dr. Mucke advises. He adds that adaptive trial methods, greater use of data, and a return to more empirical testing should help make a difference. Why delay your next breakthrough? Whether you want to design optimal TAL effector pairs or CRISPR-Cas9 gRNA, select an excellent transfection reagent, or simply have us generate your custom stable cell lines, let us help you answer your important scientifi c questions faster and with less effort. We offer a broad range of solutions to assist you in building your next breakthrough. GeneArt ® It faster and more easily at /genomeediting For Research Use Only. Not for use in diagnostic procedures. © 2015 Thermo Fisher Scientifi c Inc. All rights reserved. All trademarks are the property of Thermo Fisher Scientifi c and its subsidiaries unless otherwise specifi ed. CO210178 0415 GeneArt ® It—with GeneArt ® genome editing tools TRANSLATIONAL MEDICINE

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