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Transgenomic, Raptor Collaborate on Genetic Testing for Potential Leigh Syndrome Drug

NEW YORK (GenomeWeb News) – Transgenomic today announced an agreement to provide genetic testing services for Raptor Pharmaceuticals' clinical trial evaluating its drug called RP103 as a potential treatment for Leigh syndrome and other inherited mitochondrial disorders.

The deal will use mitochondrial gene tests that Transgenomic already has commercially available, a spokesperson from the company told GenomeWeb Daily News in an e-mail, noting the Mitochondrial Whole Genome Analysis with Haplotyping test. Testing will be conducted in Transgenomic's CLIA-certified laboratory, using its Surveyor Nuclease and Wave System technologies and sequencing, along with long-range PCR to detect large genetic deletions.

She added that while the agreement is not for the development of a "'formal companion diagnostic,' this is a good example of how Transgenomic works with pharmaceutical companies to employ genetic testing solutions for product candidates in development."

The company declined to disclose financial and other terms of the deal.

Leigh syndrome is a neurological disorder characterized by progressive loss of mental and movement abilities. It typically arises in the first year of life and results in death within a few years, usually from respiratory failure, Transgenomic said.

Raptor filed an investigational new drug application with the US Food and Drug Administration in December for the clinical development of RP103 as a treatment of Leigh syndrome. RP103 is marketed in the US as Procysbi (cysteamine bitartrate) for managing nephropathic cystinosis in adults and children six years of age and older.