Pequliar is the most advanced artificial intelligence for designing ASOs for rare genetic disorders.
Trained on all existing knowledge, benchmarked against FDA-approved ASOs, and being used in pilots in leading hospitals. Design takes minutes and costs $399.
Between 300 and 500 million people live with a rare genetic disease. Most of these conditions do not even have a name, and no one is developing a cure for them — they are each considered too rare to be worth it.
Yet for the great majority of these cases a route to treatment already exists. It is called an antisense oligonucleotide — a small, tailored molecule that quiets, corrects, or works around one specific error in one specific gene. Fifteen are FDA-approved, and roughly a hundred patients have already been treated with an ASO made for their mutation alone.
In 2017 doing this for one child took a year and $1.5 million. A regulatory framework has since been built to fast-track these treatments: today the same path can take three months and cost on the order of $10,000. What remained scarce was the knowledge to design an ASO that is safe and effective for a mutation nobody has studied.
That is what Pequliar is. It is trained on 500,000 rare mutations and on the available scientific, medical, and pharmacological literature on ASOs; it has been benchmarked against FDA-approved ASO drugs; and it has devised new classes of ASO for mutations long considered intractable, including single-base substitutions and deletions of repetitive codons. It is in use today in pilots at leading hospitals in Israel.
What you receive is a report: the designed ASOs themselves, and a clear path forward.
The first conversation is free, because the most useful thing Pequliar can tell some families is that this particular road is not the right one.
Tell us the gene and the change. You get an amenability read — plain-language, with the reasoning — before any payment.
Reference mRNA retrieved and annotated, candidates generated across eight mechanisms, thermodynamics scored, off-targets screened against the human transcriptome.
Explaining to you and a physician what the next steps are.
See a real example →A child in Boston had a Batten-disease mutation that existed nowhere else in the world. Her physicians designed a molecule for her alone, tested it, and had it authorized by the FDA. Mutation to first dose: ten months. Since then the n-Lorem Foundation has turned that one-off act of care into a route others can walk.
Built by Dr. Ido Bachelet — pharmacologist and synthetic biologist, with twenty years in nucleic acid therapeutics.
Postdoctoral work at MIT and in the George Church lab at Harvard Medical School. Founder of five biotechnology companies.
If it looks amenable, a full design report is $399. If it doesn’t, Pequliar will tell you plainly and you’ll have lost nothing.
Start the conversation →