CEVEC Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors

• ELEVECTA(R) takes viral vector manufacturing to the next level to serve the rapidly growing market of gene therapy applications
• Growing serum-free in suspension, ELEVECTA(R) producer cell lines enable fully scalable high-performance AAV vector production based on standardized processes widely used in industry for monoclonal antibody production
• With all components stably integrated into one cell, the ELEVECTA(R) technology provides large scale AAV production without expensive cGMP-grade plasmids and complex transient transfection steps

Cologne, Germany, April 28^th, 2020

CEVEC Pharmaceuticals GmbH (CEVEC), the leading provider of high-performance cell technology for the manufacturing of advanced biotherapeutics from research to production scale, today announced the launch of the ELEVECTA(R) platform, a unique technology to manufacture AAV (Adeno-associated viral) vectors at large scale. With the launch, CEVEC is the first and only provider on the market to offer AAV gene therapy vector manufacturing technology on the basis of fully stable producer cell lines.

The patent-protected technology has been proven by CEVEC in pilot projects with several partners and is now commercially available for Pharma and Biotech companies running gene therapy programs from late research to all clinical phases.

ELEVECTA(R) takes AAV vector manufacturing to the next level

With the ELEVECTA(R) technology, CEVEC has taken a unique approach based on producer cell lines, which have all necessary elements for AAV production stably integrated in one cell. Up to now, manufacturing of AAV - the most widely used vector for in vivo gene therapy - has required expensive transfection reagents and cGMP-grade plasmids. ELEVECTA(R) now overcomes these limitations and enables efficient, high-performance AAV production from one cell line in consistent quality. The technology can easily be implemented in bioprocess development and large-scale GMP manufacturing facilities running standard suspension bioreactor equipment widely used in industry for monoclonal antibody and recombinant protein production.

"The launch of ELEVECTA(R) forms a milestone on the way into a new era of viral vector manufacturing as it provides a solution for one of the major challenges in gene therapy development. By closing the production gap and providing the necessary quantities of viral vectors, it paves the way to address more common indications such as Alzheimer's, Parkinson's, or Rheumatoid Arthritis as well as therapies that require larger doses," said Nicole Faust, CEO of CEVEC Pharmaceuticals. "With this launch, we provide pharmaceutical and biotechnology companies with the opportunity to benefit right from the beginning from what we believe is the future for viral vector production. The superior features in terms of scalability, production efficiency and robustness of our stable producer cell lines position CEVEC at the leading edge in the rapidly growing field of gene therapy vector manufacturing."

ELEVECTA(R) closes the production gap in gene therapy viral vector production

Gene therapy is considered to be the most effective and often only treatment option for many severe and life-threatening diseases. Moreover, in a growing number of indications, gene therapies for the first time offer the opportunity to cure a disease. In addition, as gene therapy is moving from rare and ultra-rare to more common indications with larger patient numbers and systemic treatments, larger amounts of vector material are required. Consequently, production processes are needed that are fit for this purpose, enabling the manufacture of the necessary quantities of viral vectors with high yield and consistent quality.

ELEVECTA(R) - A concept to match the customers' needs

CEVEC's newly launched ELEVECTA(R) technology overcomes the limitations of existing manufacturing methods such as restricted scalability, time-consuming and cost-intensive material sourcing and complex production processes. ELEVECTA(R) technology is based on a patent-protected, fully documented human suspension cell line developed and optimized by CEVEC.

The ELEVECTA(R) technology works for any combination of serotype-specific capsid and therapeutic gene of interest. The stable integration of these components into the cell results in cell lines designed to produce highly functional AAV vectors. Custom-made ELEVECTA(R) Producer Cell Lines are available as research cell banks or as fully tested cGMP Master Cell Banks for manufacturing of clinical and commercial material.

CEVEC launches the technology in various formats matching the needs of customers in different development phases, with focus on gene therapy programs from late research to any clinical phases. CEVEC offers cell line development services to match the specific needs of gene therapy programs and various license packages from research and development to commercial manufacturing.

Further information:

To get a deeper insight into the ELEVECTA(R) technology register for a on May 5th, at 5:00 p.m. CEST (11:00 a.m. EST) featuring CEVEC's CSO, Dr. Silke Wissing, talking about "Stable AAV producer cell lines: elevating vector manufacturing" and/or visit the web page.

In addition, you can meet CEVEC's management at the taking place from May 12 - 15, 2020. During this event, interested parties can listen to the ELEVECTA(R) poster presentations and visit the CEVEC virtual booth. Please request a virtual meeting through the ASGCT webpage or directly contact CEVEC under .

About CEVEC:

CEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced bio-therapeutics from R&D to manufacturing scale. The company's product portfolio comprises platform technologies for gene therapy viral vectors (AAV, Adenoviral vectors, Lentiviral vectors,), vaccines and complex recombinant proteins. With the ELEVECTA(R) Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any expensive transfection reagents and cGMP plasmids. CEVEC's CAP(R) Technology based on human suspension cells is the ideal production platform for RCA-free Adenoviral vectors, Lentiviral vectors, Oncolytic viruses, Viral vaccines and exosomes. With the CAP-Go(R) Technology CEVEC provides a solution to the increasing need for recombinant production of complex and highly glycosylated protein molecules, including laminins, coagulation factors and plasma proteins.

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