The LogicBio team is passionate, driven and fiercely committed to delivering on the promise of genetic medicine to transform the lives of children with rare genetic diseases. We recognize that our mission is best achieved by working together with the patient communities that we are focused on. By understanding the unmet needs of patients and the challenges faced in daily disease management, we are able to do our jobs with purpose. By listening and learning from advocates, patients, and caregivers, we can better understand how these diseases affect the lives of patients and their families. And by collaborating with advocacy organizations throughout the drug development process, we can achieve our shared goals more efficiently.

“Every day, patients and their families are on our minds driving a steadfast commitment to address their high unmet needs.”
Fred Chereau, President & CEO of LogicBio.

Our mission is to provide the first disease-modifying therapy for patients living with MMA. We selected MMA as the first indication for our gene-editing platform because of the clear unmet need that exists today.

About MMA

Methylmalonic acidemia, or MMA, is a rare and life-threatening genetic disorder, affecting 1 in 25,000 to 50,000 newborns. In the most common form of MMA, a mutation in a gene called MUT prevents the body from properly processing certain fats and proteins. As a result, toxic metabolites accumulate in the liver, in muscle tissue and in the brain. Symptoms include vomiting, lethargy, seizures, developmental delays and organ damage.

There is no cure for MMA. To manage the symptoms, patients go on a severely restrictive, low-protein, high-calorie diet, often through a feeding tube. Some individuals may undergo a liver transplant. Even so, they often experience life-threatening metabolic crises that can cause permanent neurocognitive damage.

Community Resources

The Organic Acidemia Association (OAA) is the world’s leading source for support and information about the metabolic disorders known as organic acidemias, which include propionic acidemia and methylmalonic acidemia. The group is a volunteer-run, nonprofit organization whose mission is to empower families and health care professionals with knowledge about organic acidemia disorders. OAA supports early intervention through expanded newborn screening, solicits contributions and distributes funding that supports research toward improved treatment and eventual cures.

Additional Resources

Our Clinical Trials

Our Expanded Access Policy

LogicBio Therapeutics is committed to developing safe and effective treatments for patients worldwide. Enrolling in our clinical trials is the primary way for patients to access our investigational therapies. View our Expanded Access Policy.

Contact Us

The LogicBio team is committed to serving the communities affected by the disorders for which we are developing treatments.

If you have questions or would be willing to share your story with us, please send us an email: