Get Life-Saving Therapeutics to Market, Faster.

Through the use of viral vectors, gene editing (i.e., CRISPR), and other CGTs, biopharmaceutical companies are now treating once-untreatable diseases like Duchenne muscular dystrophy, hemophilia, spinal muscular atrophy, and nearly 7,000 other rare diseases.   Using genetic material to create genomic – gene, cell, and gene editing – treatments, biopharmaceutical companies can create CGT that repairs genetic mutations by introducing functioning genes that replace or inhibit the mutated gene. Compared to traditional remedies like small-molecule drugs, these treatments are more targeted, resulting in minimal unwanted side effects.   Along with the massive potential for targeted medicine, biopharmaceutical companies also must face a large amount of risk, which is why their processes must comply with GMP standards and undergo rigorous audits from health agencies like the FDA. Centralized lab management platforms like CellPort make it easy for biopharmaceutical labs to develop & automate fully traceable, reproducible lab processes – and make satisfying audit requests quick & easy – reducing the time it takes for life-saving products to reach the patients who need them.

For the most productive drug discovery operations, pharmaceutical companies must maintain accurate models of human disease. Historically, mammalian cells, single-cell organisms (e.g., yeast), and immortalized human cancer cell lines have been the primary targets of disease modeling, allowing us to model human disease over time, with certain restrictions.  For example, animal models cannot fully compare to human physiology, whereas immortalized cell lines may be unreliable due to abnormalities or procedural challenges.Recently, due to the development of induced pluripotent stem cells (iPSCs) – terminally differentiated adult cells that have reverted back to an embryonic-like state, and therefore share many characteristics of embryonic stem cells (ESCs) – pharmaceutical companies can scale their use of stem cells for disease modeling purposes.2-D in vitro disease models using iPSCs have proved challenging in a 2D tissue culture environment, due to the sensitive nature of cell maturation and development. Through standardized procedures that improve outcome reproducibility, lab management tools like CellPort are helping pharmaceutical labs improve results, scale manufacturing, reduce costs, and comply with regulations.