Freeline Therapeutics acquires Munich-based manufacturing technology platform and has appointed Dr Markus Hörer as Chief Technology Officer
London, 17 April 2016 – Freeline Therapeutics (“Freeline”), a biopharmaceutical company focused on the development of gene therapies for bleeding and other debilitating disorders, today announced the acquisition of the Adeno-Associated Virus (AAV) manufacturing platform developed by Rentschler Biotechnologie GmbH (Rentschler), a world leading contract manufacturer of biopharmaceuticals. In conjunction with the acquisition, Freeline has also established operations in Munich to further its manufacturing technology and maintain its leadership position for best in class manufacturing processes for AAV. Dr Markus Hörer, previously Director of Virus-based Biologics at Rentschler, has joined Freeline as founder and head of Freeline GmbH and Freeline’s Chief Technology Officer (CTO).
Freeline, based at the UCL Medical School, Royal Free Hospital, London, was launched in December 2015 with a £25 million Series A financing from Syncona LLP. The Company’s next-generation AAV gene therapy platform; developed by Professor Amit Nathwani, Founder and Chief Scientific Officer of Freeline as well as Professor of Haematology at University College London, builds on the successful haemophilia B phase I/II trial conducted by him with St. Jude Children’s Research Hospital.
Dr Christian Groendahl, CEO of Freeline, said:
“The acquisition of Rentschler’s AAV technology platform and the appointment of Markus Hörer, with over 25 years AAV experience, as our Chief Technology Officer, is key to our strategy of meeting the clinical and commercial supply needs of our pipeline. We will continue to invest in cutting edge manufacturing technology and expertise and look forward to driving our haemophilia B gene therapy programme through the clinic, as well as progressing the potentially life changing therapies for other debilitating disorders in our pipeline.”
Dr Jan Thirkettle, Chief Development Officer of Freeline, commented:
“The acquisition of Rentschler’s AAV manufacturing platform represents our commitment to establishing a global, fully integrated gene therapy company, as we continue to build on our leadership position in the field of AAV-based gene therapy products.”
Dr Markus Hörer, Chief Technology Officer of Freeline added:
“The advancement of gene therapies into clinical trials is currently hampered by the lack of robust quality and scalability needed to manufacture AAV vectors. I’m delighted to be joining the Company on a full time basis as it progresses its gene therapy treatments that have the potential to transform the lives of patients with bleeding disorders and other debilitating diseases.”
Markus Hörer has over 25 years’ experience working in the AAV field, as well as over 18 years’ experience in vaccine and biologics development in research, process and analytical development, quality control, preclinical and business development. Prior to his appointment at Freeline he was responsible for setting up the new Virus-based Biologics business unit at Rentschler GmbH where he focussed on AAV vector development. Prior to that he held various positions at MediGene AG where he was responsible for the development of rAAV vector technology, its successful transfer into a GMP environment and its use for development of autologous Melanoma cell vaccines. After transferring the oncolytic HSV technology from San Diego to Munich, he was in charge of setting up a quality control and assay development division. He was also head of the AAVLP vaccine platform technology, process and preclinical project development. Markus holds a PhD in Biology from the German Cancer Research Center Heidelberg.
Christian Groendahl, Chief Executive Officer
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About Freeline TherapeuticsNotes for Editors:
Freeline Therapeutics is a biopharmaceutical company based at the Royal Free Hospital, London, focused on the development of gene therapies for bleeding and other disorders. Gene therapy has the potential to transform lives for people with severe diseases by providing a long-lasting, safe and reliable source of enzymes to the blood. The Company’s human adapted AAV vector platform is being utilised to advance therapies through the clinic and its shareholders include Syncona LLP and University College London. For further information please visit the Company’s website at: www.freelinetx.com
Syncona LLP was founded in 2012 and operates as a healthcare investment company, taking an active role in identifying, developing and funding technologies with the potential to significantly impact the healthcare market of the future. Syncona can take the long view when necessary, able to concentrate investment into opportunities as technology is validated. Syncona is a subsidiary of the Wellcome Trust. For further information please visit the Company’s website at: www.synconallp.com
About Haemophilia B
Haemophilia B, also called Factor IX (FIX) deficiency, is a genetic disorder caused by missing or defective Factor IX that can result in dangerously low levels of the essential clotting protein. The World Federation of Haemophilia identified over 28,000 males suffering worldwide from haemophilia B in their Annual Global Survey 2013. For those with severe disease marked by FIX levels of less than one percent of normal, scrapes and bumps are medical emergencies. Painful episodes of spontaneous bleeding can result in crippling joint damage early in life and an increased risk of potentially fatal bleeding within the brain.
Current treatment involves replacement therapy where concentrates of clotting factor are administered intravenously on an on-demand or prophylactic basis. This preventive therapy is usually initiated in patients at a young age and may need to be continued for life. It is hampered by the frequency of administration of up to 3 times per week as well as the high associated costs.