Technology Bundle ID

Creation and Use of Kinetin Derivatives for Treating RNA Missplicing Diseases Such as Familial Dysautonomia

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Lead Inventors
Susan Slaugenhuapt (Massachusetts General Hospital)
Juan Marugan (NCATS)
This technology includes the creation and use of compounds, including kinetin derivatives, that improve mRNA splicing in a cell for the treatment of disorders associated with misspliced mRNA, including familial dysautonomia (FD). FD, the best-known and most common member of a group of congenital sensory and autonomic neuropathies, affects neuronal development and is associated with progressive neuronal degeneration. This disease is caused by mutations in the splicing of intron 20 of the IKMKAP gene that results in a unique pattern of tissue-specific exon skipping.
Commercial Applications
Further clinical work with the mRNA splicing compounds may establish these therapeutics as definitive treatments for familial dysautonomia (FD).
Competitive Advantages
There are no current treatments of familial dysautonomia (FD) and the kinetin derivatives described in this technology could be the first-in-class.

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