Pequliar is step one. The rest is a known route with named providers, published costs, and regulatory precedent. Step four is a decision point where stopping is the right answer.
Candidate design, screening, go / no-go decision, large-scale synthesis, release testing, regulatory approval, administration.
Sequences designed, thermodynamics scored, off-targets screened, candidates ranked — a synthesis-ready report in minutes.
Research-grade synthesis in about one to two weeks from vendors such as IDT, Eurogentec, Gene Tools or TriLink.
Screen in patient fibroblasts and cell lines, in a hospital lab or at a contract research provider; dose-response confirms target engagement and potency. Pequliar provides a tailored experiment protocol as part of the report.
Analyse the data and decide whether to proceed. If no candidate produces a positive effect, you can stop here — and that is a legitimate outcome.
Derived from potency, target tissue and route.
Treatment-dose manufacturing.
Identity by MS, purity by HPLC, sterility, endotoxin, appearance, pH and osmolality.
Repeat efficacy testing to confirm manufacturing equivalence, and file a single-patient compassionate-use IND under 21 CFR 312.310.
With IND authorization and confirmed product quality, the treating physician administers the ASO per the approved plan.
As of 2026, 17+ US states explicitly cover individualized ASOs, removing the federal Right to Try Act’s Phase 1 requirement. These laws add state-level protection without overriding federal FDA authority — and they are what turns a year-long programme into a matter of months.
| Drug | Indication | Mechanism | Chemistry |
|---|---|---|---|
| Spinraza (2016) | Spinal muscular atrophy | Splice-switching | 2′-MOE PS |
| Exondys 51 (2016) | Duchenne MD | Exon skipping | PMO |
| Tegsedi (2018) | Hereditary ATTR amyloidosis | Gene silencing (RNase H) | 2′-MOE PS gapmer |
| Qalsody (2023) | ALS (SOD1) | Gene silencing (RNase H) | 2′-MOE PS gapmer |
| Viltepso (2020) | Duchenne MD | Exon skipping | PMO |
| Amondys 45 (2021) | Duchenne MD | Exon skipping | PMO |
| Kynamro (2013) | Homozygous FH | Gene silencing (RNase H) | 2′-MOE PS gapmer |
| Milasen (2018, IND) | CLN7 Batten — single patient | Splice-switching | 2′-MOE PS |
2′-MOE phosphorothioate and PMO backbones account for most of the fifteen FDA-approved ASO drugs, including all eight above. A validated backbone does not mean a novel sequence is clinically tested — each new sequence is a distinct molecular entity.