Osiris Therapeutics announced that it received marketing authorization from Health Canada to market its stem cell therapy, Prochymal, for the treatment of graft-versus-host disease in children.
Osiris Therapeutics announced that it received marketing authorization from Health Canada to market its stem-cell therapy, Prochymal (remestemcel-L), for the treatment of graft-versus-host disease in children who fail to respond to steroids. Prochymal is a manufactured mesenchymal stem cell therapy derived from the bone marrow of healthy adult donors.
According to the company press release, Health Canada’s marketing authorization marks the world’s first approval of a manufactured stem-cell product, and the first approved treatment for graft-versus-host disease. The product was approved under Health Canada’s Notice of Compliance with conditions pathway, which is an accelerated approval pathway for products that address unmet medical conditions and which have shown a favorable risk/benefit profile in clinical trials. Under this pathway, further clinical trials will be required to verify the anticipated benefit.
Graft-versus-host disease is a complication resulting from bone marrow transplantation in which immune cells contained in the transplant recognize the recipient of the graft as foreign and mount an immune response. Severe graft-versus-host disease is fatal in as much as 80% of cases. Steroids are the first-line treatment, but have a success rate of only 30–50%. According to the company website, Prochymal is currently in Phase III trials in the US for treatment of graft-versus-host disease and Crohn’s disease and has been awarded both Orphan Drug and fast track status from FDA.
Harnessing mRNA as a Readout to Develop Robust BioPotency Assays
December 12th 2024Transcriptional activity within a cell can be used to evaluate cell response to a ligand or promoter activity within a transgene or plasmid within a cell. Catalent has developed a relative potency bioassay using real-time quantitative reverse transcription (RT-qPCR) in a duplex format to assess relative transcription activity in cells treated with ligands or transgenic vectors. The assay utilizes two fluorescent dyes with minimally overlapping emission spectra that allow real-time monitoring of the gene expression of both target and normalizer genes. The assay does not require purification of the mRNA produced by the cells once lysis has occurred. Normalizing the qPCR cycle thresholds (CT) of the target transcript to the reference transcript allows response curve to be generated and compared to a reference standard. The generation of a four-parameter fit curve analysis from raw qPCR cycle threshold data allows for comparison of relative potency and assessment of suitability based on curve parallelism. The assay platform has been used by Catalent to qualify a repeatable, accurate, linear, and specific bioassay for assessing relative potency.
The Solution Lies with SOLBIOTE™: Achieving Sustainability, a Growing Focus in Biopharma
October 28th 2024The nexus between biopharmaceuticals and sustainability is seemingly far apart, however, it is increasingly recognized as an inevitable challenge. It is encouraged to take a sustainable approach to reducing the environmental impact of the production and supply of medicines while improving people's health; delivering the well-being of people and the planet. Yosuke Shimojo (Technical Value Support Section Manager, Nagase Viita) will unveil how SOLBIOTE™, a portfolio of injectable-grade saccharide excipients, would be a key for the biopharmaceutical development and achieving sustainability for a better future of the industry.
Exploring New and Improved Analytical Methods for Traditional and Unique Modalities
December 12th 2024Biophysical characterization is critical to understand the make-up and behaviors of biologic therapies and vaccines, both early in development and throughout the manufacturing scale-up process. As biologics become more complex in structure, and as scientists improve their understanding of the effects of structure on stability, efficacy, safety, etc., there is a need to develop new and improved analytical methods to characterize biologic products. During this presentation, experts will discuss the latest challenges in biophysical characterization and will present solutions to overcome these challenges.