Biomay Receives FDA Approval for Cas9 Nuclease Manufacturing, Expanding CRISPR Therapy Supply Chain
Key Insights
Biomay (search) has secured FDA approval for manufacturing Cas9 nuclease (search) at its Vienna headquarters, a critical component for CRISPR (search)-based gene editing therapies including Vertex Pharmaceuticals' CASGEVY®.
The approval follows a December 2024 inspection by FDA's Center for Biologics Evaluation and Research (CBER) with no observations or Form FDA 483 issues, marking Biomay (search)'s second successful FDA inspection.
The Austrian CDMO has established itself as a key player in biopharmaceutical manufacturing, previously partnering with BioNTech to supply DNA templates for COVID-19 (search) mRNA vaccines since early 2020.
Vienna-based Contract Development and Manufacturing Organization (CDMO) Biomay (search) has received approval from the U.S. Food and Drug Administration (FDA) to manufacture, test, and release recombinant Cas9 nuclease (search) from its headquarters site in Vienna, Austria. This approval positions Biomay as a critical supplier in the growing CRISPR (search) gene editing therapeutics market.
The FDA's Center for Biologics Evaluation and Research (CBER) conducted an inspection of Biomay (search)'s headquarters manufacturing facility in Vienna Seestadt in December 2024. The inspection concluded with no observations and no Form FDA 483 being issued, effectively authorizing the site to supply Cas9 nuclease (search) for the United States market.
Cas9 nuclease (search) is an essential component in CRISPR (search)-based gene editing therapies, including CASGEVY® (exagamglogene autotemcel), the groundbreaking therapy developed and launched by Vertex Pharmaceuticals.
Expanding Manufacturing Capabilities
This recent approval marks Biomay (search)'s second successful FDA inspection, following the 2023 approval of the company's downtown Vienna facility. Biomay now operates two independent cGMP manufacturing sites in Vienna – the headquarters in Seestadt and the downtown facility – both with FDA clearance.
The headquarters site is described as a recently constructed, state-of-the-art biomanufacturing facility, reflecting Biomay (search)'s commitment to expanding its production capabilities for advanced therapeutic components.
Established Track Record in Biopharmaceutical Manufacturing
Founded in 1984, Biomay (search) has specialized in the expression of recombinant proteins using E. coli since its inception. The company has evolved to offer comprehensive cGMP services for manufacturing therapeutic proteins, plasmid DNA (pDNA), and messenger RNA (mRNA).
Biomay (search)'s service portfolio encompasses process and analytical development, cell banking, cGMP manufacturing of drug substances, and aseptic filling of drug products.
Previous Collaboration with BioNTech for COVID-19 Vaccine
Biomay (search) previously demonstrated its manufacturing expertise through a partnership with BioNTech SE to support the supply chain for the Pfizer-BioNTech COVID-19 (search) mRNA vaccine. This collaboration, initiated during the clinical development phase of the vaccine in Q1/2020, involved Biomay supplying BioNTech with DNA templates for in-vitro transcription of the mRNA active ingredient.
After becoming a formally qualified supplier for BioNTech, Biomay (search)'s scope of services included process and analytical development, GMP preparation of cell banks, manufacturing of circular DNA plasmid, and preparation of linear DNA templates. Throughout 2021, the company produced and delivered multiple batches of released DNA templates, contributing significantly to mRNA vaccine production during the pandemic.
Strategic Positioning in Advanced Therapeutics
With its expanded FDA-approved manufacturing capabilities, Biomay (search) is strategically positioned to support the growing demand for components essential to advanced therapeutics, particularly in the rapidly evolving field of gene editing.
The approval for Cas9 nuclease (search) manufacturing is particularly significant as CRISPR (search)-based therapies gain momentum in clinical applications, with CASGEVY® representing one of the first commercially available CRISPR gene editing treatments.
As a privately owned and fully integrated CDMO, Biomay (search) continues to strengthen its role in the global biopharmaceutical manufacturing landscape, supporting innovative therapies from development through commercial production.