AAV gene therapy programs face long development cycles, raw material variability, and complex scale-up demands.

Summary: Cell and gene therapies continue to expand across oncology, cardiovascular, and CNS diseases, yet production remains limited by talent shortages, complex supply chains, and slow development cycles.

AAV genomic integrity is based on defining the molecular DNA composition of recombinant AAV vector preparations, specifically, the presence of full-length, ITR-to-ITR genomes, and the relative amount of undesirable molecular sub-species encapsidated within AAV particles.

Plasmid DNA is a vital component in the production of viral and nonviral cell and gene therapies (CGT). Catalent's innovative UpTempo℠ Plasmid Platform Process has revolutionized plasmid DNA manufacturing by addressing key challenges and significantly reducing development time.

Summary: Catalent has successfully addressed the challenges associated with plasmid DNA manufacturing by developing robust process and analytical platforms. Catalent’s approach involves a one-process-fits-all single-use (SU) manufacturing process in compliance with current good manufacturing practices (cGMP) standards.

Summary: Catalent offers extensive expertise in the bioproduction of plasmid DNA, from early stages of development to CGMP manufacturing to satisfy clinical and commercial needs.

This year, the long-awaited FDA draft guidance for Platform Technology Designation Program was launched. This critical guidance allows drug developers to leverage Platform Technology data in approved drugs if similar “platforms” are used, accelerating the approval process and therapies to patients. In this webinar, George Buchman will discuss Catalent’s approach to platform processes as well as securing an integrated supply chain.

In this expert roundtable, three experienced professionals from the rapidly evolving gene therapy field delve into the crucial assessment of AAV potency and its profound influence on the industry's progress.

In this expert roundtable, three experienced professionals from the rapidly evolving gene therapy field delve into the crucial assessment of AAV potency and its profound influence on the industry's progress.

Catalent expert George Buchman, Ph.D., offers insights on key challenges with scaling up production for gene therapies, complexities in the supply chain, regulatory considerations and overall reflections on the industry, progress and future.