ADENO-ASSOCIATED VIRUS (AAV)

STABLE AAV PRODUCTION

The ELEVECTA® Producer Cell Lines are custom-made for your specific AAV vector by stably incorporating the sequences of the serotype-specific capsid and the gene of interest into the genome of the cell. It is the perfect cell line for reliable AAV production in suspension bioreactors at all scales and formats. Available as research cell bank or, optionally, as fully tested GMP Master Cell Bank for the manufacturing of clinical and commercial material.

All Producer Cell Lines are derived from the monoclonal ELEVECTA® Alpha Cell Line which provides helper functions, replicase and further genetic elements necessary to generate stable ELEVECTA® Producer Cell Lines and to produce AAV vector material at consistent yield and quality.

THE TAILOR-MADE PRODUCER CELL LINE

The stable ELEVECTA® Producer Cell Line reaches titers up to 10E14 vg/l during a typical 10 L single use bioreactor run. Single step scale-up from 15 ml to 10 L stirred tank bioreactors have been demonstrated without any product loss showing the robustness of the process.

Cloning from a polyclonal pool to a monoclonal single cell often results in a 10-fold vg/mL titer increase.

AAV production from stable producer cells

A Titer comparison of stable AAV production, polyclonal pool and single cell clones, in 15 mL parallel reactors (Sartorius ambr®) and 10 L suspension bioreactor runs.

B Cryo TEM picture from AAV particles derived from ELEVECTA® Producer Cell Lines display an approximate size of 22 nm. Full particles are indicated by a black arrow, empty particles by a white arrow.

Example of producer cell lines displaying high level expression of serotype-specific capsid and other elements necessary to produce AAV vector material.

High-level productivity over time was proven with single cell clones, producing AAV at approximately the same titers over 75 cell doublings.

PROVEN STABILITY AND TIGHT CONTROL OF AAV PRODUCTION

A Serotype specific capsid, rep and helper functions as well as the the gene or interest, expressed by the Stable Producer Cell

B Expression of vg/L of a single cell clone across different passages with and without selection

© CEVEC Pharmaceuticals GmbH 2020