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Learn more about the four Pillar 2 programs

Pillar 2 of the FAST Roadmap to a Cure 2.0 consists of therapeutic programs that focus on “turning on dad’s gene” by activating the silent paternal copy of the UBE3A gene in neurons.

Currently, we have 4 programs in the pipeline under Pillar 2:

  • Antisense Oligonucleotides/Spherical Nucleic Acids (ASO/SNA)
    • Uses a combination of synthetic RNA and DNA to bind to the RNA of the UBE3A-ATS (UBE3A antisense transcript), which is responsible for silencing the paternal copy of the UBE3A gene in neurons
  • Clustered regularly interspaced short palindromic repeats (CRISPR)
    • Consists of two components, the CAS enzyme that can cut DNA (CAS or CRISPR associated protein 9) or RNA (CAS13) and a guide RNA (gRNA) that can recognize specific sequences of DNA or RNA to cut. This technology is often delivered through an AAV carrier, but new technology is advancing to offer viral-free options 
  • Artificial Transcription Factors / Zinc Fingers (ATF)
    • A protein that binds to the UBE3A-ATS resulting in unsilencing of the paternal UBE3A gene 
  • Short-hairpin RNA/micro-RNA (shRNA)
    • Pieces of RNA that are delivered in an AAV virus that bind to the UBE3A-ATS and activate the silent paternal copy of the UBE3A gene in neurons

Within Pillar 2, we have 3 active human clinical trials, all of which are within the ASO program!