- CAP®Go provides unique opportunity for CSL to develop highly differentiated recombinant C1 Esterase Inhibitor (C1-IHN) proteins for Hereditary Angioedema (HAE) and other potential indications
- CSL to leverage CEVEC’s expertise in the production of C1-INH based on CAP®Go technology
- Further important step in establishing CAP®Go as the production standard for difficult to express recombinant proteins
Cologne, Germany and Melbourne, Asutralia, May 8, 2018 – CEVEC Pharmaceuticals GmbH (CEVEC), the expert in the production of tailor-made recombinant glycoproteins and gene therapy vectors, and global biotechnology leader CSL Limited (ASX:CSL; USOTC:CSLLY) today announced an exclusive licensing agreement for the development, manufacture and commercialization of recombinant C1 Esterase Inhibitor (C1-INH) proteins for Hereditary Angioedema (HAE) and other potential indications using CEVEC’s proprietary CAP®Go technology.
Under the terms of the agreement, CEVEC grants an exclusive license for the development, manufacture and commercialization of CAP®Go-derived C1-INH proteins to CSL. In addition, CEVEC will provide its expertise in the development and use of CAP®Go cell lines expressing C1-INH, and in optimizing the expression of highly glycosylated and difficult to produce proteins. The licensing agreement was the result of an ongoing collaboration between CSL and CEVEC, which yielded initial data on the significant potential of the CAP®Go technology to enable the development of a differentiated recombinant C1-INH product candidate. Financial details of the agreement have not been disclosed.
Under the agreement, CSL aims to develop CAP®Go-derived C1-INH proteins with tailored glycosylation, leading to an improved half-life and more convenient application. CEVEC’s proprietary CAP-technology may allow for the industrial manufacture of such a protein given the scalable production process established for CAP®Go cells.
“The agreement with CSL is a further endorsement of our unique human expression system with the intention to take our CAP®Go technology to the next level of implementation for the production of difficult to express recombinant proteins,” said Dr. Nico Scheer, Chief Business Officer of CEVEC Pharmaceuticals GmbH. “Its outstanding characteristics in terms of authentic and tailor-made glycosylation capabilities, high yield production of complex proteins and scalability, combined with its IP protection and the complete documentation of the CAP® cells make the CAP®Go technology the ideal choice for industrial scale production of glyco-optimized proteins. We are looking forward to supporting CSL with this exciting project.”
“At CSL, we are patient focused and we are driven by our promise to patients. CEVEC’s CAP®Go technology provides us with the opportunity to develop an innovative and differentiated recombinant and glyco-optimized C1-INH protein for HAE and potential other indications. The potential increase in convenience and quality of life for patients will help us further expand our position in this very important market,” Dr. Andrew Nash Senior Vice President Research, CSL added.
About Hereditary Angioedema (HAE)
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema . HAE attacks can affect many parts of the body and can spread to multiple sites, including the face, abdomen, larynx, and extremities . Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall . HAE attacks that involve the face or throat can result in airway closure, asphyxiation, and, if left untreated, death .
 Kemp JG, et al. Variability of prodromal signs and symptoms associated with hereditary angioedema attacks: A literature review. Allergy Asthma Proc. 2009; 30:492-499.
 Agostoni A, et al. Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase inhibitor deficiency workshop and beyond. J Allergy Clin Immunol. 2004;114(3 Suppl): S51-131.
 Frank MM, et al. Hereditary angioedema. J Allergy Clin Immunol. 2008;121(2): S398-S401.
CSL (ASX:CSL) CSL is a leading global biotechnology company with a dynamic portfolio of life-saving medicines, including those that treat haemophilia and immune deficiencies, as well as vaccines to prevent influenza. For over 100 years, CSL has been driven by its promise to save lives using the latest technologies. Today, CSL — including its two businesses, CSL Behring and Seqirus – provides life-saving products to more than 60 countries and employs nearly 20,000 people. The company’s unique combination of commercial strength, R&D focus and operational excellence enables it to identify, develop and deliver innovations so patients can live life to the fullest.
For more information, please visit: www.csl.com.au.
CEVEC is a center of expertise for the production of biopharmaceuticals using a unique human cell-based expression system.
CAP®Go enables the production of proteins previously out of reachrepresenting a significant proportion of the human proteome that is notoriously difficult to express in conventional cell lines such as CHO. The CAP®Go expression platform comprises a portfolio of glyco-optimized human suspension cell lines for the highly efficient production of a broad range of difficult to express recombinant proteins with authentic human post-translational modifications or on demand tailor made glycosylation patterns.
CAP®GT is a fully scalable manufacturing platform for viral vector production. CEVEC has successfully developed CAP®GT suspension cell-derived viral packaging cell lines which enable better scale-up and competitive production costs when compared to adherent cell culture systems. CAP®GT suspension cell lines grow to high cell densities and show excellent productivity for a broad range of viruses. Gene therapy vectors such as lentivirus (LV), adenovirus (AV) and adeno-associated virus (AAV) can be produced at industrial scale.
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