It’s time.
Dedicated to advancing novel treatments for patients living with severe, genetic disorders associated with the liver.
It’s time.
Dedicated to advancing novel treatments for patients living with severe, genetic disorders associated with the liver.
It’s time.
Dedicated to advancing novel treatments for patients living with severe, genetic disorders associated with the liver.

Portfolio of gene therapy treatment

Dimension’s programs address significant unmet medical needs for patients living with hemophilia and rare diseases associated with the liver. The Company’s lead program in hemophilia B is currently in phase I/II clinical testing. The company is also advancing programs through preclinical testing to address hemophilia A in collaboration with Bayer HealthCare and for patients living with ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia (GSDIa). Additional programs addressing citrullinemia type 1, phenylketonuria, and Wilson disease in candidate selection continue to broaden and expand our liver-directed rare disease platform.

Dimension’s therapies use the biology of the parvovirus (adeno-associated virus (AAV)) to carry the missing gene in the body to the target cell with the aim of correcting the defect and providing meaningful clinical benefit to the lives of patients. This platform has demonstrated early validation and compelling preclinical in vivo proof of concept across multiple disease states in leading academic research centers.  / Learn More /

GENE THERAPY

Dimension’s approach selectively targets the replacement of missing intact genes to treat diverse rare diseases.

AAV Platform
Selectively targeting liver cells to deliver missing intact genes
Pipeline
Addressing severe, rare genetic disorders
About Us
Thought leaders with expertise in orphan products

Featured Program

Glycogen Storage Disease Type Ia (GSDIa)

GSD Type Ia (GSDIa) is the most common, genetically inherited glycogen storage disease. Individuals with GSDIa have a defective gene for the enzyme glucose-6-phosphatase, resulting in the inability to regulate blood sugar (glucose).

NEWS
December 5, 2016
Dimension Therapeutics Announces Presentation at the 58th American Society of Hematology (ASH) Annual Meeting
November 29, 2016
Dimension Therapeutics Receives Positive Opinion for European Orphan Drug Designation of DTX401 for the Treatment of Glycogen Storage Disease Type Ia (GSDIa)
November 10, 2016
Dimension Therapeutics Reports Recent Corporate Progress and Third Quarter 2016 Financial Results
November 3, 2016
Dimension Therapeutics Announces Presentation at the Upcoming 58th American Society of Hematology (ASH) Annual Meeting