Two paths. One goal: a NIPBL therapy.

Drug repurposing

Searching 40,000+ existing compounds for therapeutic potential.

Our first therapeutic track is focused on drug repurposing: identifying existing compounds that may have the potential to modify the NIPBL condition and improve quality of life.

Through our work with Unravel Biosciences, we began with an N-of-1 analysis for Yuvaan. Unravel’s platform compares patient biology against a large library of 40,000+ existing compounds to identify candidates that may help shift disrupted gene-expression patterns back toward a healthier state. The good news: many of these compounds already have safety data, clinical history, or prior use in humans. That means if we find a promising candidate, the path toward clinical trial may be faster than developing a brand-new drug from scratch.

How it works: Unravel uses RNA sequencing and computational biology to map the molecular signature of the condition, then identifies compounds predicted to reverse or improve that signature.

Antisense oligonucleotides (ASO)

Evaluating approaches for NIPBL upregulation.

Many NIPBL-related conditions are caused by haploinsufficiency, meaning a person has only one working copy of the NIPBL gene and that single copy does not produce enough protein for normal function. Antisense oligonucleotides (ASOs) are small genetic medicines that have traditionally been used to silence or reduce gene expression. However, newer ASO platforms have shown that it is possible to increase expression from a person’s existing gene. We are exploring whether these upregulation approaches could be applied to NIPBL.

How it works: We’re working with multiple biotech solutions to evaluate which platform is best to upregulate NIPBL via ASOs. This includes understanding the gene biology, identifying the appropriate therapeutic strategy, and determining which models, data, and safety considerations are needed before moving into ASO design and testing.