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Merck, Pfizer, Eli Lilly, Enlight Biosciences, RainDance Technologies, Znomics, Oregon Health & Science University, Sangamo BioScience, Sigma-Aldrich, Roche, National Cancer Institute, Wellcome Trust Sanger Institute

Merck, Pfizer, Lilly Form New Company to Speed Development of Drug-Discovery Tools
Merck, Pfizer, and Eli Lilly said this week that they have partnered with PureTech Ventures to create a new firm called Enlight Biosciences that will focus on the development of new drug-discovery technologies.
The three pharmas and PureTech have ponied up a total of $39 million that Enlight will direct toward “breakthrough technologies that can fundamentally alter drug discovery and development.”
Enlight is focused on “pre-competitive” technology that will “connect preclinical research, clinical development, and medical practice,” according to a company statement.
The company noted that despite the recent emergence of “vital enabling technologies” such as PCR, PET, RNAi, and gene microarrays, “over the last few years most traditional life science investors have focused their funding on late-stage therapeutic programs.”
As a result, Enlight said, “important technologies that could be of great strategic impact to the pharmaceutical industry are not being commercialized.”
Enlight said it has already begun technology-development programs in the areas of molecular imaging, biologics, and drug delivery, and is planning to launch programs in other areas.
The company’s website lists a range of “areas of interest” under the categories of imaging and biomarkers, safety and toxicology, predictive models, chemistry and biochemistry, synthesis and production, formulation and delivery, and biologic platforms.
In the “biomarker” category, for example, Enlight is interested in developing “systems biology/multi-marker patterns, protein/antibody arrays, label-free binding assay methods, [and] platforms for patient segmentation.”
Rod MacKenzie, senior vice president of Pfizer Global R&D, said in a statement that the effort should help develop “breakthrough technologies” that will enable innovation in biopharmaceutical science, which he described as “an urgent priority” for drug-makers.

RainDance Technologies Forms French Subsidiary for Euro Partnering
RainDance Technologies has formed a subsidiary in France that will focus on developing research partnerships in Europe, the company said this week.
The new company, Raindance Technologies France SARL, will be based in Strasbourg at the Louis Pasteur University’s Institute of Science and Supramolecular Engineering. Andrew Griffiths, a RainDance founder, is director of the institute’s Chemical Biology Laboratory.
The subsidiary is expected to start operations at the beginning of September, RainDance said.
RainDance was founded in 2004 and is based in Lexington, Mass. It is developing an analytical platform that produces picoliter-volume droplets that can contain a single molecule, reaction, or cell, and are each the “functional equivalent of an individual test tube,” the company said.
The company plans to focus its initial application on human genome resequencing research.
The new subsidiary is expected to help RainDance develop collaborations with European groups studying genomics, molecular biology, and drug discovery, RainDance CEO, Chris McNary, said in a statement.

Znomics Inks Drug-Discovery Collaboration with OHSU
Zebrafish-screening firm Znomics said this week that it is collaborating with Oregon Health & Science University to develop pre-clinical compounds to treat diseases such as rheumatoid arthritis, asthma, and inflammatory bowel syndrome.
The company said it will be collaborating with Thomas Scanlan, director of OHSU’s chemical biology program, on the project. Scanlan has been a member of Znomics' scientific advisory board since December 2007.
Under the terms of the agreement, Znomics will fund the program and will have the option to exclusively license the rights to the discoveries.
The company said that the collaboration meets its objective of establishing three de novo drug discovery programs this year, and that it plans to have three pre-clinical lead compounds in different disease areas in 2010.
In addition to the disease areas covered in the OHSU collaboration, the firm has a drug discovery program in obesity and a collaborative drug discovery program with the University of Utah that it signed earlier this year to develop compounds for the treatment of T-cell diseases, including leukemia, lymphoma, and autoimmune disorders (see CBA News, 5/16/08).

Roche Licenses Sangamo's ZFN Technology to Produce Cell Lines, Transgenic Animals
Sangamo BioSciences and Sigma-Aldrich said this week that Roche has licensed Sangamo’s zinc finger nuclease technology to develop cell lines and transgenic animals that have targeted modifications in specific genes.
Zinc finger nucleases are engineered forms of zinc finger DNA-binding proteins that can be used to help modify target genes in organisms. Sigma-Aldrich is the exclusive licensee of Sangamo’s ZFN technology for research reagents.
Under the agreement, Roche will receive a non-exclusive, worldwide research license to Sangamo’s ZFN technology in exchange for research maintenance fees and research event payments, and it will conduct research with both Sangamo and Sigma-Aldrich.
Roche also has an option to obtain an exclusive, worldwide license for the commercial use of ZFN-generated transgenic animals in the production of therapeutic and diagnostic products.
If it exercises this option, it will pay an additional license fee, as well as payments on clinical milestones and royalties from the sale of therapeutic and diagnostic products.

NIH, Sanger Researchers Develop Assay for Assessing BRCA2 Variants
Scientists have developed a functional assay for distinguishing between dangerous and neutral mutations in a breast cancer susceptibility gene.
In a paper appearing online in Nature Medicine this past Sunday, researchers from the National Cancer Institute and the Wellcome Trust Sanger Institute described how they used mouse embryonic stem cells to evaluate the functional implications of 17 BRCA2 sequence variants. They found that this assay could effectively and reliably categorize risky BRCA2 mutations — which can dramatically increase an individual’s risk of breast and ovarian cancer — as well as relatively innocuous mutations.
“[O]ur assay is likely to improve our understanding of unclassified mutations because it allows for analysis of all types of BRCA2 mutations,” senior author Shyam Sharan, head of NCI’s Genetics of Cancer Susceptibility Section, said in a statement.
BRCA1 and BRCA2 mutations, which tend to be inherited in some families, can dramatically increase an individual’s cancer risk. For instance, those carrying a mutation in one of the genes have a 35 percent to 80 percent risk of developing breast cancer by their 70th birthday. In contrast, the average American woman has a 12.3 percent risk of developing breast cancer in that time.
But BRCA1 and BRCA2 sequence variants don’t all confer the same risk. Some are extremely deleterious, while others are neutral or low risk. Distinguishing between these extremes can be tricky, since mutations are often identified through genetic tests of families with a history of breast and ovarian cancer.
“Segregation analysis in cancer-afflicted families provides the most reliable information to distinguish between deleterious and neutral alterations identified in BRCA1 or BRCA2,” Sharan and his colleagues wrote. “However, there is an enormous need for a functional assay to classify variants for which such information is not available, because most mutations are rare, and familial data are often insufficient.”
That also makes it difficult to interpret BRCA1/BRCA2 genetic tests for those carrying unclassified or minor mutations — such as the 1,900 known BRCA1 or BRCA2 variations that don’t disrupt the gene product in an obvious way but may still affect gene function.
For this study, Sharan and his team exploited the properties of mouse embryonic stem cells to develop an assay for systematically testing BRCA2 variants. Specifically, the researchers took advantage of the fact that mouse stem cells need a functional copy of BRCA2 to survive.
The human BRCA2 gene can complement — that is, take over the function of — mouse BRCA2, but only if the human BRCA2 gene is functional. To test the functionality of various BRCA2 sequence variants, the team generated mouse embryonic stem cells that were missing one copy of BRCA2 and one conditional BRCA2 allele.
Then, they added bacterial artificial chromosomes containing individual human BRCA2 variants, inactivated the lone copy of mouse BRCA2, and looked at whether the cells could survive by relying on that human BRCA2 gene.
Variants that could not rescue the BRCA2 function in these mouse embryonic stem cells were classified as deleterious mutations, while those that could were considered neutral mutations. The researchers further characterized the variants using plating efficiency experiments and testing the cells’ sensitivity to DNA-damaging agents.
All told, the researchers tested 17 BRCA2 variants. As expected, mutations with known functional effects could not rescue the BRCA2 function while neutral mutations could. Among the previously unclassified variants, they found examples of both neutral and deleterious variants. Overall, the researchers classified eight of the 17 mutations as deleterious. The remaining nine appear to be either neutral or low risk.
While the researchers emphasized the assay’s promise for evaluating BRCA2 variants, they were quick to note that it will take time before the assay can be used as a clinical tool. “[U]ntil the assay is fully validated,” the authors warned, “caution must be exercised when using these data to make clinical decisions.”
According to a statement from the National Institutes of Health press office, Sharan is interested in collaborating with commercial organizations to develop the diagnostic test.
In the meantime, the authors are optimistic that such assays will eventually be used to characterize not only BRCA2 variants, but also variants in other disease-associated genes.
“We have established that this assay is accurate and is one of the most comprehensive among those available to date, allowing analysis of virtually any type of mutation,” they wrote, “and it may also serve as a model for investigating other human disease-associated genes that result in a phenotype detectable in [embryonic stem] cells.”
— Andrea Anderson

The Scan

Drug Response Variants May Be Distinct in Somatic, Germline Samples

Based on variants from across 21 drug response genes, researchers in The Pharmacogenomics Journal suspect that tumor-only DNA sequences may miss drug response clues found in the germline.

Breast Cancer Risk Gene Candidates Found by Multi-Ancestry Low-Frequency Variant Analysis

Researchers narrowed in on new and known risk gene candidates with variant profiles for almost 83,500 individuals with breast cancer and 59,199 unaffected controls in Genome Medicine.

Health-Related Quality of Life Gets Boost After Microbiome-Based Treatment for Recurrent C. Diff

A secondary analysis of Phase 3 clinical trial data in JAMA Network Open suggests an investigational oral microbiome-based drug may lead to enhanced quality of life measures.

Study Follows Consequences of Early Confirmatory Trials for Accelerated Approval Indications

Time to traditional approval or withdrawal was shorter when confirmatory trials started prior to accelerated approval, though overall regulatory outcomes remained similar, a JAMA study finds.