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 commercialisation of a novel gene therapy for the treatment of Haemophilia A.

“Bayer is a worldwide leader in the treatment of Haemophilia A and we are highly committed to advancing innovative treatment options for patients with Haemophilia 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 Haemophilia 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 Haemophilia 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 commercialisation 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 commercialise the potential future product for the treatment of Haemophilia 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 NAV technology.

Haemophilia 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. Haemophilia A, the most common type of haemophilia, is caused by a deficient or defective blood coagulation protein, known as factor VIII. Haemophilia A is characterised by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs.

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer Hematology includes an approved treatment for Haemophilia A and compounds in development for Haemophilia 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.

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 haemophilia B programme 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 NAV technology. Dimension has been funded by Fidelity Biosciences and OrbiMed Advisors.

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.