Living bacterial therapy for homocystinuria

Live microbial therapeutics: an enhanced treatment paradigm for classical homocystinuria

NIH-funded research Petri Bio LLC · NIH-11142557

This project is developing a friendly gut bacteria treatment to lower homocysteine levels in people with classical homocystinuria.

Quick facts

Grant typeSbir 2 grant
Study typeNIH-funded research
Funding institutionPetri Bio LLC NIH-funded
Lab location1 site (Los Angeles, United States)
Project IDNIH-11142557 on NIH RePORTER

What this research studies

Researchers are engineering safe gut bacteria to break down methionine in the intestine so less homocysteine builds up in the blood. They searched microbial genomes for methionase enzymes, put promising genes into probiotic strains compatible with the human gut, and are testing activity and safety in the lab and preclinical models. The goal is to create an oral biologic that could reduce reliance on strict low-methionine diets and on betaine supplements that commonly cause unpleasant side effects. If preclinical results are favorable, the company plans steps toward human safety and efficacy testing.

Who could benefit from this research

Good fit: People diagnosed with classical homocystinuria, especially those who do not respond to vitamin B6 and who have elevated homocysteine despite current treatments, would be the main candidates.

Not a fit: People without homocystinuria, those whose HCU is well controlled on existing therapy, or people with severe immune compromise who cannot receive live bacterial products may not benefit or be eligible.

Why it matters

Potential benefit: If successful, this could lower blood homocysteine and make management of homocystinuria easier and better tolerated.

How similar studies have performed: Engineered probiotic approaches have shown promise in other metabolic disorders but using live bacterial therapeutics for homocystinuria is largely novel with limited human data so far.

Where this research is happening

Los Angeles, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.