Healthcare Industry News:  hemophilia A 


 News Release - June 23, 2014

Bayer HealthCare and Dimension Therapeutics to Develop Novel Gene Therapy for Hemophilia A

LEVERKUSEN, Germany, June 23, 2014 -- (Healthcare Sales & Marketing Network) -- Bayer HealthCare (Bayer) and Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, have entered into a collaboration for the development and commercialization of a novel gene therapy for the treatment of hemophilia A.

"Bayer is a worldwide leader in the treatment of hemophilia A and we are highly committed to advancing innovative treatment options for patients with hemophilia A," said Prof. Dr. Andreas Busch, Member of the Bayer HealthCare Executive Committee and Head of Global Drug Discovery. "We are excited to partner with Dimension Therapeutics to jointly harness the power of gene therapy to drive the development of new long-term options in treating this disease."

"Currently available replacement therapies for hemophilia A are often administered intravenously multiple times a week and may be required for life, depending on the severity of a patient's disease," said Thomas R. Beck, M.D., chief executive officer of Dimension Therapeutics. "Gene therapy offers the potential to transform the treatment of hemophilia by inserting a correct version of the faulty gene responsible for the disease. We are proud to partner with Bayer, a leader in the treatment of hemophilia A, to develop a therapy with the potential to significantly change the treatment landscape."

Under the terms of the agreement, Dimension Therapeutics will receive an upfront payment of $20 million and will be eligible for potential development and commercialization milestone payments of up to $232 million. Dimension Therapeutics will be responsible for all pre-clinical development activities and the Phase I/IIa clinical trial, with funding from Bayer. Depending on the results of the Phase I/IIa clinical trial, Bayer will conduct the confirmatory Phase III trial, make all regulatory submissions, and will have worldwide rights to commercialize the potential future product for the treatment of hemophilia A. Dimension Therapeutics is eligible to receive tiered royalties based on product sales.

Dimension Therapeutics' AAV vector technology allows for systemic intravenous administration of the clotting factor gene in vivo, which has been shown in preclinical studies to target the liver resulting in long lasting expression of FVIII protein at therapeutic levels. Dimension Therapeutics' vectors are enabled by REGENX Biosciences' proprietary NAVĀ® technology.

About hemophilia A

hemophilia A, also known as factor VIII deficiency or classic hemophilia, is a largely inherited bleeding disorder in which one of the proteins needed to form blood clots in the body is missing or reduced. hemophilia A, the most common type of hemophilia, is caused by a deficient or defective blood coagulation protein, known as factor VIII. hemophilia A is characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs.

About Hematology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer Hematology includes an approved treatment for hemophilia A and compounds in development for hemophilia A, sickle cell anemia, and other blood and bleeding disorders. Together, these compounds reflect the company's commitment to research and development in these disease states.

About Dimension Therapeutics

Dimension Therapeutics is a gene therapy company focused on developing novel therapies to treat rare diseases. Formed in 2013, the Dimension team comprises biotech industry veterans and renowned thought leaders in gene therapy and rare diseases. The company is focused on building its adeno-associated virus (AAV) therapeutic capabilities and advancing multiple gene therapy programs in rare diseases, and is advancing a wholly-owned hemophilia B program towards clinical development. Dimension's partnerships with REGENX Biosciences and the University of Pennsylvania provide Dimension with exclusive gene therapy intellectual property and preferred access to multiple best-in-class AAV vectors based on REGENX's NAVĀ® technology. Dimension has been funded by Fidelity Biosciences and OrbiMed Advisors. For more information, please visit

About Bayer HealthCare

The Bayer Group is a global enterprise with core competencies in the fields of health care, agriculture and high-tech materials. Bayer HealthCare, a subgroup of Bayer AG with annual sales of EUR 18.9 billion (2013), is one of the world's leading, innovative companies in the healthcare and medical products industry and is based in Leverkusen, Germany. The company combines the global activities of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals divisions. Bayer HealthCare's aim is to discover, develop, manufacture and market products that will improve human and animal health worldwide. Bayer HealthCare has a global workforce of 56,000 employees (Dec 31, 2013) and is represented in more than 100 countries. More information is available at

Forward-Looking Statements

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

Source: Bayer HealthCare

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