​

Patient of Monroe Carell Jr. Children's Hospital at Vanderbilt first in world to receive new investigational gene editing therapy

A 9-year-old patient of Monroe Carell Jr. Children’s Hospital at Vanderbilt is the first in the world to receive an investigational gene editing therapy for Methylmalonic Acidemia (MMA), a rare genetic disorder diagnosed at birth.

On May 29, Eddie Axelson, of Clarksville, Tennessee, received LogicBio Therapeutics’ investigational single-administration targeted gene-editing therapy, hLB-001, which seeks to correct MMA, an inborn metabolism disorder in which the body cannot properly process protein from food.

Axelson received the dose of hLB-001, administered via IV infusion, as part of LogicBio’s SUNRISE multicenter clinical trial. The study will assess the safety, tolerability and effectiveness of this potential gene editing therapy for MMA.

Read More...

Tweets by vumcpeds