Pharmaceutical Technology - May 2021

Pharmaceutical Technology - Biologics and Sterile Drug Manufacturing - May 2021

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26 Pharmaceutical Technology BIOLOGICS AND STERILE DRUG MANUFACTURING EBOOK 2021 P h a r mTe c h . c o m Analytics G ene therapy was first described in the early 1970s (1), and nearly 50 years later, FDA approved the first one for use in patients: an adeno-associated vector (AAV)-mediated therapy that treats biallelic RPE65 mutation-associated retinal dystrophy, a condition that causes pro- gressive blindness (2). More gene therapies will soon follow, as nearly 450 gene therapy clinical trials are underway (3). FDA is expected to approve 10 to 20 new cell and gene therapies by 2025 for diseases ranging from hemophilia to spinal muscular atrophy (4). By 2030, Massachusetts Institute of Technology researchers ex- pect 20 to 60 cell and gene therapy products will reach the market, benefitting a total of 350,000 patients (5). The accelerated pace of gene therapies is a sign of enhancements in our understanding of genetics and viral engineering as well as innovation in manufacturing processes. Despite this progress, gene therapy development is not a perfect science, and manufactur- ers must be careful to monitor the quality of their products before they reach patients. Take viral vector-mediated gene therapies, for example. Like the first gene therapy to be approved, most biopharmaceutical manufacturers are expected to rely on AAV vectors as the deliv- ery vehicle for their gene therapies. This stems from the fact that these vectors can carry a wide variety of nucleic acid sequences, and low immunogenicity and mutagenicity due to their episomal nature (6). Unfortunately, as AAVs are grown in cell culture, they are prone to contamination from microbes and host-cell components such as nucleic acids. These contaminants could Using ddPCR to Detect Mycoplasma Contamination Mark White The sensitivity of droplet digital PCR helps manufacturers detect Mycoplasma contamination in AAV-based gene therapy products. Mark White, PhD, is associate director, Biopharma Product Marketing, at Bio-Rad Laboratories. K ATERYNA _KON - STOCK.ADOBE.COM

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