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On the cutting edge
March 2016
by Jeffrey Bouley  |  Email the author
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A model mouse to fast-track cures, a technological expansion into orphan diseases, a license for the ARIES assay system, an organic chemistry prediction tool, a grant for quantitative real-time PCR and a novel multiplex DNA assembly approach—that’s what this month brings you as we get ready to emerge from winter into spring.
 

The Jackson Laboratory expands availability of mouse model
 
BAR HARBOR, Maine—The Jackson Laboratory, a leading provider of mouse models and drug discovery services, reported recently that it has dramatically expanded access to the NSG-SGM3 mouse model to scientists worldwide. This murine strain reportedly allows for the superior development of myeloid and CD4+ T cell populations after human hematopoietic stem cell transplantation, thereby enabling a more accurate representation of the human immune system.
 
The company expects this model to accelerate research in immuno-oncology, immunology and infectious disease.
 
The triple transgenic NSG-SGM3 strain shares the same genetic background as the popular NSG, while expressing human IL3, GM-CSF and SCF.
 
“The NSG-SGM3 model is unique in that it offers the most physiological co-engraftment of patient tumors and human immune cells, including myeloid cells that are critically important in modulating human responses to cancers,” according to Dr. James Keck, senior director of the In Vivo Pharmacology division at The Jackson Laboratory. “These results highlight the potential for use of this cutting-edge platform for preclinical efficacy testing of immuno-oncology compounds.”
 
“The NSG-SGM3 is a leap forward over existing highly immunodeficient models, and the potential that it brings to make scientific investigations at the bench and in the clinic more translatable is exciting,” added MaryEllen Joseph, senior business unit manager of JAX Mice at The Jackson Laboratory. “Early results using it as a preclinical tool in drug discovery are very encouraging.”
 

BioXcel expands into CNS orphan disease
 
BRANFORD, Conn.—BioXcel Corp., announced in February its expansion into central nervous system (CNS) orphan diseases that require novel treatment solutions for neurological manifestations, including neurodegenerative disorders, motor dysfunction disorders and lysosomal storage disorders. BioXcel will utilize its Big Data Innovation Lab and PharmGPS Orphan Disease Suite to discern common pathophysiological mechanisms in CNS-related disease clusters and identify potential disease-modifying agents.
 
“The CNS orphan disease space represents an underserved therapeutic area due to the high unmet medical need and lack of innovative treatments,” said Dr. Vimal Mehta, BioXcel’s chairman and CEO. “We believe that our unique approach is capable of yielding multiple clinical programs leading to human proof-of-concept studies and, potentially, an accelerated path to market for our therapeutic candidates.”
 
“We are entering into partnerships with large pharmaceutical companies with a repertoire of clinical agents and leveraging the capabilities of our first-in-class big data-powered platform to discover breakthrough therapeutics,” said BioXcel’s senior vice president of CNS R&D, Dr. Frank D. Yocca. “The extension of our platform into CNS orphan disease therapeutics represents an important step forward in our evolution into a big data-driven drug development company.”
 

Luminex receives medical device license in Canada
 
AUSTIN, Texas—Luminex Corp. recently announced that it has received medical device licenses from Health Canada’s Therapeutic Products Directorate, Medical Devices Bureau for the ARIES System and ARIES HSV 1&2 Assay. These ARIES products previously received U.S. Food and Drug Administration clearance in October 2015 and were subsequently launched in the U.S. market.
 
“We are excited to receive medical device licenses for the ARIES System and the ARIES HSV 1&2 Assay in Canada. We look forward to rapidly launching this revolutionary system there,” said Todd Bennett, vice president of global sales and customer operations at Luminex. “We are confident that the ARIES System will answer many of our Canadian clinical laboratory customers’ needs by increasing efficiency, reducing errors and delivering accurate and timely data to improve patient care.”
 

Better prediction for organic chemists
 
HOBOKEN, N.J.—Draw your target molecule and ChemPlanner will predict shorter, faster, cheaper routes to your target, even through predicted, never-before-reported reactions based on what it has learned about organic chemistry—that is the promise of New Jersey-based Wiley with regard to its reaction route prediction tool based on machine learning for organic chemists.
 
“In a climate of ever-increasing competition and risk, and skyrocketing costs, the pharmaceutical industry, for instance, is striving to achieve more drug approvals with smaller R&D investments,” Wiley notes. “Medicinal chemists are endeavouring to achieve greater productivity—more targets, more screening, more validation and more hypothesis testing—and are pursuing innovative pharmacological approaches. Consequently, chemical synthesis becomes a key to success, as it directly influences how quickly new ideas can be put to the test and at what cost.”
 

NIH grant to develop resQ-RNA
 
SANTA CRUZ, Calif.—Somagenics has been awarded a second Phase I SBIR grant from the U.S. National Institutes of Health to develop its resQ-RNA technology for quantitative real-time PCR of fragmented RNA samples.
 
Somagenics’ resQ-RNA method now allows analysis of RNA fragments as short as 23 nucleotides, whereas other methods require a minimum of about 100 nucleotides. As a result, resQ-RNA reportedly allows RNA quantification in older FFPE samples, which represent a resource for biomarker discovery that, due to greater fragmentation, has been inaccessible until now.
 
“resQ-RNA has great potential to help transform huge archives of biopsy specimens that currently languish in hospital collections into resources that researchers can analyze. Because these specimens are associated with patient data, that analysis can lead to discovery of biomarkers linked to disease progression and treatment response,” said Brian Johnston, CEO of Somagenics. “Such biomarkers are valuable tools for determining patient prognosis, choosing optimal treatment plans and assessing progress.”
 

Multiplex synthesis platform for DNA assembly
 
CAMBRIDGE, Mass.—Early February saw Gen9 announce it has developed a novel multiplex DNA assembly approach which scales the manufacture of synthetic DNA by nearly two orders of magnitude. Gen9 has applied this technology to long-length DNA construction, building 50 gene-length DNA constructs simultaneously in a single reaction. The multiplexing technology will be applied to Gen9’s BioFab next-generation DNA synthesis platform over the next 12 months.
 
“Global DNA synthesis capacity is currently limited to hundreds of millions of base pairs of DNA per year. Gen9’s multiplex technology has the potential to scale that to billions of base pairs and to make possible the idea of realizing a $1,000 synthetic genome,” said Joseph Jacobson, co-founder of Gen9 and an associate professor at the Massachusetts Institute of Technology.
 
“Our BioFab platform makes manual, time-consuming PCR cloning methods for working with DNA obsolete. This enables scientists to get to their answers faster by designing, building and testing many sequences at once, rather than serially,” said Devin Leake, vice president of R&D at Gen9.
 
Code: E031630

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