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
February 7, 2017
Dimension Therapeutics Elects Dr. John A. Hohneker to Its Board of Directors
January 31, 2017
Dimension Announces Interim Topline Results from Ongoing Phase 1/2 Clinical Program for DTX101, Dimension’s Lead AAV Product Candidate in Development for Adult Patients with Moderate/Severe to Severe Hemophilia B
December 13, 2016
Dimension Therapeutics Announces FDA Acceptance of Investigational New Drug Application for DTX301 to Treat OTC Deficiency
December 5, 2016
Dimension Therapeutics Announces Presentation at the 58th American Society of Hematology (ASH) Annual Meeting