GamowLabs screens all FDA-approved drugs, finds HIV drug raltegravir may raise BUB1B for rare disease

danielmckinn0n · x · 2026-10-29

GamowLabs shared its first drug-repurposing study, done for a child in Hugging Face's Real Kid, Rare Disease hackathon. The team hypothesized the child's MVA stems from biallelic loss-of-function variants in BUB1B and sought FDA-approved drugs that raise BUB1B expression.

Public data fell short, so they ran their own transcriptomic screen of every FDA-approved compound. Several raised BUB1B; the most interesting hit is raltegravir, an HIV integrase inhibitor (+2 log2 fold on BUB1B, DESeq2 adjusted P=0.026), worth follow-up validation.

The authors call it an early proof of concept with a real, scalable method and invite researchers on BUB1B, MVA, or chromosomal instability to collaborate.

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