Date: 2014-08-21
Type of information: Development agreement
Compound: automated quantitative endpoint measurements
Company: Sarepta Therapeutics (USA - MA) Flagship Biosciences (USA - CO)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Type agreement: development
Action mechanism:
Disease: Duchenne muscular dystrophy
Details: * On August 21, 2014, Sarepta Therapeutics, a developer of innovative RNA-based therapeutics, and Flagship Biosciences LLC, a leading tissue-based companion diagnostics firm, announced a multi-year, multi-product partnership for the development of automated quantitative endpoint measurements in muscular dystrophy to support the advancement of Sarepta’s Duchenne muscular dystrophy (DMD) drug pipeline, including its lead candidate, eteplirsen. Eteplirsen is designed to address the underlying cause of DMD by enabling the production of a functional internally deleted dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression. Eteplirsen uses Sarepta\'s novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene\'s ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. In order to optimally and efficiently evaluate therapeutic efficacy in patients, the next generation of protocols are being developed to digitally automate and standardize dystrophin measurement in tissue biopsies to speed the process while ensuring consistency. The establishment of these new standardized methods for automated quantitation is being enabled though the proprietary image analysis platform and digital pathology capabilities developed by Flagship.
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