For those interested here's a useful paper with some background on the genetics of Angelman and how Ultragenyx's ASO approach works, and some of its limitations. Basically, they are attempting to use an ASO to release silencing of the normal paternal UBE3A gene to compensate for the disrupted maternal gene. I have some concerns about the practicality of this approach, including the need for repeated and invasive intrathecal administration.
Interestingly, the work in this paper is sponsored by Pfizer who seem to have identified a small molecule candidate to "unsilence" UBE3A. Very early doors though and several years away from even clinical trials.
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