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Rare Disease Start-Up: Dimension Therapeutics’ Gene Therapy For Hemophilia A

October 28, 2014

This Blog Post reviews the new start-up, Dimension Therapeutics and the company’s first gene therapy program for Hemophilia A.

The creation of Dimension Therapeutics (DT), a new Cambridge MA-based start-up gene therapy company, focusing on the development of treatments for rare diseases, is announced in October 2013. Fidelity Biosciences and REGENX Bioscience announce the creation of DT. The new start-up company will develop and commercialize novel Adeno-Associated Virus (AAV) gene therapy products for multiple rare disease indications.  During gene therapy, an AAV delivers a working “healthy” gene into the body to replace the faulty or missing targeted gene.

Fidelity Biosciences provides the initial venture capital for the new start-up. REGENX Biosciences is an AAV gene therapy company that is providing its proprietary NAV vector technology platform. DT acquires access to REGENX Biosciences’ NAV vector technology and rights to the company’s product programs in multiple rare disease indications. Dimension has now raised $30M with funding from Fidelity Biosciences and OrbiMed.

DT’s lead gene therapy program will be for Hemophilia A. Hemophilia is a rare bleeding disorder that is usually inherited and most often occurs in males. Hemophilia is characterized by producing little or no clotting factor. The clotting factor is a protein required for normal blood clotting. When a hemophilic patient has an injury, a life threatening bleeding can occur. The two types of Hemophilia are Hemophilia A (Factor VIII Deficiency), the most common type, and Hemophilia B (Factor IX).

The main treatment option for Hemophilia is replacement therapy – concentrates of clotting factor are administered intravenously to help replace the clotting factor that is either low or missing in the patient. Gene therapy for Hemophilia corrects the faulty gene that causes it. DT’s AAV vector technology allows the “administration of the gene therapy in vivo, which has been shown in previous proof-of-concept studies in hemophilia to trigger the liver to create the clotting factor. This approach may enable patients to live without replacement therapy infusions for an extended period of time.”

In June 2014, DT and Bayer HealthCare announce a collaboration to develop and commercialize gene therapy for the treatment of Hemophilia A. DT will receive an upfront payment of $20 million and will be eligible for potential milestone payments of up to $232 million. DT is responsible for pre-clinical development and the Phase I/IIa clinical trial, with funding from Bayer. Depending on results of the Phase I/IIa clinical trial, Bayer will conduct the confirmatory Phase III trial, will do the regulatory submissions, and will have worldwide rights to commercialize the future potential product.

In September 2014, FierceBiotech, names DT as one of 2014’s “Fierce 15 biotechnology companies”.

Please Note: “Gene Therapy” National Institute of Health [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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