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23andMe, MondoBiotech Collaborate to Study Genetic Risk for Rare Diseases

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Consumer genomics firm 23andMe and Switzerland-based MondoBiotech are collaborating on a global research project to uncover the genetic underpinnings of rare diseases, the partners announced last week.

Details of the collaboration are sparse, but the partners said they hope their research will lead to the development of genetic tests that gauge patients’ predisposition to rare disease and help target individuals most likely to respond to drugs made by MondoBiotech.

“Ultimately, the hope is to improve on the diagnostics that can benefit the global community,” 23andMe Co-founder Linda Avey told Pharmacogenomics Reporter last week. “There are people in countries that are affected by these rare diseases but are never diagnosed.”

The research will focus on diseases whose genetic basis is not well understood, such as pulmonary arterial hypertension, sarcoidosis, and pulmonary fibrosis. MondoBiotech will enroll patients for the clinical trials and fund the studies, including the cost of genotyping with 23andMe’s Personal Genome Service. 23andMe will collect genetic data on patients and coordinate genome-wide association studies.

If the alliance points to the need for companion diagnostic tests for certain MondoBiotech drugs, it is unclear what development strategy MondoBiotech will take. The company said it plans to pursue clearance from the US Food and Drug Administration for any diagnostic developed under the collaboration.

MondoBiotech has a product pipeline of more than 300 peptides associated with 600 rare diseases. Currently, the company is advancing 19 peptides through clinical development.

The most advanced candidate is in Phase III studies for idiopathic pulmonary fibrosis. MondoBiotech also has candidates for pulmonary hypertension, acute lung injury, sarcoidosis, and pulmonary fibrosis in Phase II development.

After developing these drugs, MondoBiotech plans to license the products to larger drug companies for marketing. On it website, MondoBiotech said six of its treatments have garnered orphan drug status in Europe and in the US, and that the firm has licensed seven products to BiogenIdec, InterMune, and United Therapeutics/LungRx.

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The deal between 23andMe and MondoBiotech, disclosed last week during the World Economic Forum in Davos, Switzerland, is the personal genomic shop’s first publicly announced research collaboration with a pharma or biotech company. However, since its inception in 2007, 23andMe has required customers to make their genetic data available to 23andMe for research use.

“By your participation in the 23andMe service you contribute your genetic information to our research effort to study various aspects of human genetics in an attempt to better understand the human genome,” 23andMe states in its Terms of Service. “In addition, our service enables you to contribute other personal information towards research as well.”

To organize its research efforts, 23andMe last year launched a separate pharmacogenomics research division. Called 23andWe, its aim is to improve the quality of patient data that enters pharmacogenomic-based clinical trials organized by 23andMe; advance the development of tailored drugs; and encourage regulators and medical groups to use genetic information in their medical decision-making [see PGx Reporter 06-04-2008].

So far, 23andMe has maintained that its service is for educational and research purposes, and the genetic information it gleans cannot be used for diagnostic or medical purposes.

“Genetic discoveries that we report have not, for the most part, been clinically validated, and the technology the laboratory, [which] uses the same technology used by the research community, has also not yet been validated for clinical utility,” 23andMe notes on its website.

“The Services Content is not to be, and is not intended to be, used for any diagnostic purpose … and is not intended to be used, by you or any other person, to diagnose, cure, treat, mitigate, or prevent a disease or other impairment or condition, or to ascertain your health,” the company states.

In 2007, 23andMe was one of several firms to launch direct-to-consumer genotyping services with the promise of gleaning their risk for developing complex and sometimes fatal, diseases. But as these types of services multiplied, doctor and patient groups became increasingly concerned that existing federal regulations for these services are potentially confusing and the clinical tools being used are not clinically valid or useful [see PGx Reporter 05-07-2008].

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If the collaboration with MondoBiotech leads to genetic tests that can help guide treatment, it may generate clinical validation and clinical utility data for 23andMe’s service.

Meanwhile, 23andMe competitor Navigenics is also establishing partnerships to add clinical validity and utility to its testing service. Last week, Navigenics announced the launch of a stripped down version of its testing service that it plans to market to doctors as a tool to incorporate into patients’ annual health checkup (see related article, this issue).

Last year, 23andMe dropped the price of its genotyping service from $999 to $399, citing advancements in the Illumina genotyping platform [see PGx Reporter 09-12-2008]. Avey noted that clinical trial participants will get the same services as its paying customers.