Bio-Techne's Simple Western? Technology Utilized in Recent Fda Approval of Zevaskyn Cell-Based Gene Therapy

Published: 2025-07-14 07:50:46 ET TECH

Published on 07/14/2025 at 07:50

Bio-Techne Corporation announced that its Simple Western? Technology played a key role in supporting the FDA approval of ZEVASKYN? (prademagene zamikeracel), the first autologous cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), developed by Abeona Therapeutics®.

Throughout clinical development, Simple Western enabled precise identification and quantification of Collagen VII, a complex therapeutic target critical to ZEVASKYN's potency, supporting lot release testing for the viral vector and the cell therapy in a GMP setting. Abeona selected Simple Western over competing methods like traditional western blot for its speed, reproducibility, picogram-level sensitivity, and minimal sample volume requirements. The platform effectively detected both the trimeric and monomeric forms of Collagen VII, addressing the analytical challenges posed by the protein's size and structure and supporting the regulatory requirements for potency and consistency.

RDEB is a rare and debilitating genetic skin disorder caused by mutations in the COL7A1 gene, which leads to the misfolding of Collagen VII, a protein essential for skin integrity. Patients with RDEB suffer from severe blistering, chronic wounds, scarring, and are at significantly increased risk of life-threatening complications such as squamous cell carcinoma. The current standard of care focuses on symptomatic management, with no durable therapies available until now.

The Simple Western platform provided quantifiable, GMP-compliant data to ensure consistency of each autologous ZEVASKYN product lot, overcoming key challenges in analytical standardization, limited sample availability, and complex assay development. Abeona's final assay design was optimized through rigorous antibody selection, assay optimization, and use of an appropriate reference standard - enabled by Simple Western's automated and high-throughput capabilities.