Dimension Therapeutics Embarks on Gene Therapy for Two Rare Metabolic Disease: Will Your Child Benefit?

Patients and their parents celebrate another win in the fight against two rare diseases with Dimension Therapeutics, Inc. Ornithine Transcarbamylase (OTC) deficiency and Glycogen Storage Disease Type Ia (GSDIa) will both be the focus of a developing study to replace the missing or faulty gene, effectively correction the underlying defect to restore metabolic function.

These genetically inherited inborn errors of metabolism, usually present in the neonatal period or infancy, and can have grave effects on patients of all ages. For severe forms, there are few or no effective treatment options other than liver transplantation, leaving patients with significant unmet needs.

Dimension is developing novel gene therapy approaches to replace the missing or faulty gene, correcting the underlying defect to restore metabolic function.

“In an important step for both patients and for Dimension, we are launching our first two liver-associated, metabolic disease programs to address rare disorders with the greatest needs and fewest treatment options, other than liver transplant. Transplants are often associated with significant risks both during the procedure and over the long term”

“In an important step for both patients and for Dimension, we are launching our first two liver-associated, metabolic disease programs to address rare disorders with the greatest needs and fewest treatment options, other than liver transplant. Transplants are often associated with significant risks both during the procedure and over the long term,” said Dimension’s CEO Annalisa Jenkins, MBBS, MRCP. “Our mission – working in collaboration with patient advocacy communities and with the world’s leading clinical investigators and advisors – is to rapidly and responsibly develop new therapies that can offer hope and better health to those patients in the greatest need.”

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