Bio-Techne's Innovative Technology Facilitates FDA Approval of ZEVASKYN Gene Therapy

Bio-Techne's Role in FDA Approval of ZEVASKYN Gene Therapy

Bio-Techne Corporation, a prominent player in the life sciences field, recently announced a significant achievement in medical innovation. Their cutting-edge technology, known as Simple Western™, has been instrumental in the FDA approval of ZEVASKYN™ (prademagene zamikeracel), the world's first autologous cell-based gene therapy aimed at treating patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).

The FDA's endorsement of ZEVASKYN is a monumental milestone for patients with RDEB, a rare genetic skin disorder resulting from mutations in the COL7A1 gene. This condition causes severe skin blistering and chronic wounds, which greatly diminish the quality of life for those afflicted. Until now, treatment options were limited to symptomatic management, making this new therapy a groundbreaking advancement.

Key Features of Simple Western Technology

Simple Western™ technology has outshone traditional methods by offering rapid results, reproducibility, and sensitivity at the picogram level, all while requiring significantly lower sample volumes. This platform facilitated the precise identification and quantification of Collagen VII, a vital therapeutic target needed to demonstrate ZEVASKYN's efficacy. By providing accurate potency data, Simple Western supported the stringent lot release testing for both the viral vector and the cell therapy within a Good Manufacturing Practice (GMP) environment.

The meticulous development process undertaken by Abeona Therapeutics, the creator of ZEVASKYN, highlighted the need for an effective quality control solution. They experienced analytical challenges due to Collagen VII's complex size and structure. Simple Western proved to be the best solution, allowing the identification of both trimeric and monomeric forms of the protein, thereby meeting the regulatory requirements essential for establishing potency and consistency.

Implications for Patients and the Healthcare Field

RDEB patients face numerous complications, including chronic wounds and a heightened risk of scarring and life-threatening conditions. With ZEVASKYN, these patients now have access to a treatment option designed to create lasting positive effects rather than merely managing symptoms. This landmark approval marks a new era in cell-based gene therapies and signifies hope for individuals grappling with rare diseases.

Will Geist, President of Bio-Techne's Protein Sciences Segment, expressed pride in having their technology contribute significantly to this initiative. He emphasized that the collaboration with Abeona Therapeutics has allowed both companies to tackle the complexities associated with advanced therapeutics effectively.

Dr. Ann Durbin, Senior Director of Quality Control at Abeona, further elaborated on their experiences with Simple Western. She noted that the technology was unparalleled in terms of reproducibility and GMP compliance, which was crucial for advancing ZEVASKYN through the rigorous stages of clinical development and regulatory approval.

Bio-Techne's Contribution to the Life Sciences Industry

Bio-Techne serves a crucial role in the life sciences sector, offering innovative tools and bioactive reagents that aid research and diagnostic efforts globally. Their diverse product portfolio, including the Simple Western™ technology, empowers researchers and clinicians in drug discovery and precise clinical testing.

In fiscal 2024, Bio-Techne reported approximately $1.2 billion in net sales, with a dedicated workforce of around 3,100 employees committed to improving healthcare outcomes through scientific advancements. The company continues to forge partnerships with various organizations to further enhance their capabilities and contribute to the global understanding of diseases and medical conditions.

As new therapies like ZEVASKYN become available, the landscape of patient care for rare conditions will undoubtedly evolve, offering hope and better outcomes for many. Bio-Techne's strategic initiatives and innovative technologies are paving the way for advancements that will shape the future of medical treatments and enhance the quality of life for patients around the world.