FDA Approves ZEVASKYN™ Gene Therapy Using Bio-Techne's Simple Western™ Technology

Bio-Techne Corporation has announced that its innovative Simple Western™ Technology has been instrumental in the FDA's approval of ZEVASKYN™ (prademagene zamikeracel). This groundbreaking therapy, developed by Abeona Therapeutics, is the first autologous cell-based gene therapy designed for patients with recessive dystrophic epidermolysis bullosa (RDEB), a severe genetic skin disorder. The Simple Western platform was crucial in accurately identifying and quantifying Collagen VII, a key therapeutic target necessary for ZEVASKYN’s effectiveness, thus ensuring the consistency and potency of the gene therapy. This tool enabled efficient lot release testing for both the viral vector and the cell therapy in a GMP-compliant environment.

Abeona Therapeutics opted for Simple Western over traditional methods such as the western blot due to its superior speed, reproducibility, sensitivity at the picogram level, and minimal sample volume requirements. This technology effectively addressed the analytical challenges posed by Collagen VII’s size and structural complexities by detecting both its trimeric and monomeric forms, meeting regulatory mandates for therapeutic potency and consistency.

RDEB is a rare, debilitating genetic condition characterized by mutations in the COL7A1 gene. This mutation results in the improper folding of Collagen VII, a protein vital for maintaining skin integrity. Those affected by RDEB endure severe blistering, chronic wounds, and scarring, with an increased risk of life-threatening complications like squamous cell carcinoma. Until the advent of ZEVASKYN, treatment largely focused on managing symptoms, with no durable therapies available.

The Simple Western platform provided essential quantitative, GMP-compliant data to ensure the uniformity of each batch of autologous ZEVASKYN, overcoming significant challenges in analytical standardization, limited sample availability, and complex assay development. The final assay design was refined through a rigorous process involving antibody selection, assay optimization, and selecting a suitable reference standard. Simple Western's automated and high-throughput nature facilitated this process.

Will Geist, President of the Protein Sciences Segment at Bio-Techne, emphasized the importance of this advancement, noting, "ZEVASKYN marks a significant milestone for RDEB patients and the field of cell-based gene therapy." He expressed pride in Bio-Techne’s role in aiding Abeona to develop and validate crucial potency assays that were pivotal for the FDA's approval of this first-of-its-kind therapy.

Dr. Ann Durbin, Senior Director of Quality Control at Abeona Therapeutics, highlighted the critical role of potency method development and validation in ZEVASKYN's clinical development and regulatory approval. She described the Simple Western platform as the optimal choice for Abeona’s quality control laboratories due to its reproducibility, time efficiency, and compliance with GMP standards. Abeona collaborated closely with Bio-Techne's ProteinSimple team to overcome challenges related to the large molecular weight of Collagen VII and the need to assess the protein's tertiary structure under non-denaturing conditions. Their efforts led to a validated assay that efficiently quantifies Collagen VII, supporting both the release testing of autologous ZEVASKYN lots and the viral vector's release and stability testing. This collaboration was fundamental to advancing ZEVASKYN as the first approved cell-based gene therapy for RDEB patients.

Bio-Techne Corporation, a global entity in the life sciences sector, provides innovative tools and bioactive reagents to advance research and clinical diagnostics. With an extensive product portfolio, Bio-Techne supports scientific investigations into biological processes and diseases, aiding drug discovery and facilitating precise clinical testing. Abeona Therapeutics, a commercial-stage biopharmaceutical company, is dedicated to developing cell and gene therapies for severe diseases, with ZEVASKYN being its flagship product.