Computationally designed antisense oligonucleotides for upregulating haploinsufficient genes.
For a target gene, our pipeline designs and ranks antisense oligonucleotide candidates across four
upregulation mechanisms: splice-switching at poison/cassette exons (TANGO), alternative splice-site
extensions, AntagoNAT gapmers against natural antisense transcripts, and cis-element blockers. Each
candidate is scored on medicinal-chemistry quality, target-site accessibility, specificity
(off-target alignment), and brain expression, then assembled into a diversified screening plate with
an automated discovery briefing. Select a featured disease gene below. Looking for small molecules
instead? See the repurposing explorer and
drug design dashboard.
Discovery briefing generated by LLM from the pipeline output: scoring, NMD positioning, conservation, microwalk structure, and a portfolio screening recommendation.
Candidate landscape
Each point is a designed ASO candidate, positioned by genomic coordinate and ranked by MedChem score, colored by mechanism. Clusters mark the targetable regulatory regions the pipeline found. Click a mechanism in the legend below to toggle it.
Targets by mechanism and score
Each bar is a biological target; height is the best MedChem score in that target. Click a mechanism to filter.
Full target table
Safety profile (GC vs off-targets)
Green zone: 40-60% GC, ≤10 genomic off-targets. Candidates inside the box have the best thermodynamic and specificity properties.
Screening plate
96 wells
Have a haploinsufficiency target?
We design, rank, and de-risk ASO candidates for upregulation targets, including ultrarare disorders
that are underserved by industry. Tell us your gene and we will run the pipeline and share a shortlist.
References (tools used in the upstream ASO design pipeline)
Colombo M, et al. NMDtxDB: data-driven identification and annotation of human NMD target transcripts. RNA 2024;30(10):1277-1290.
GTEx Consortium. The GTEx Consortium atlas of genetic regulatory effects across human tissues. Science 2020;369(6509):1318-1330.
Pollard KS, et al. Detection of nonneutral substitution rates on mammalian phylogenies. Genome Res 2010;20(1):110-121.
Bernhart SH, et al. Local RNA base pairing probabilities in large sequences. Bioinformatics 2006;22(5):614-615.
Langmead B, Salzberg SL. Fast gapped-read alignment with Bowtie 2. Nat Methods 2012;9(4):357-359.