Short Time to Market and Forward Planning Will Enable Cell Therapies to Deliver R&D Pipeline Value.

There is considerable industry excitement about the curative potential of cell and gene therapies, but significant challenges remain in designing cost-effective treatments that are accessible globally. We have taken a modeling-based approach to define the cost and value drivers for cell therapy assets during pharmaceutical drug development. We have created a model development program for a lentiviral modified autologous T cell therapy for Oncology indications.

Host Cell Proteins: The Hidden Side of Biosimilarity Assessment.

One major concern with biosimilars is that small differences compared with reference products might lead to unforeseen immunogenicity, thus affecting patient safety and drug efficacy. Differences could be due to either post-translational modifications of the therapeutic protein and/or to traces of impurities from the manufacturing process. The results presented in this communication illustrate the efforts to assess "biosimilarity" of a biosimilar candidate to a reference product for a specific group of process-related impurities, the host cell proteins (HCP).

Coformulation of a Novel Human α-Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice.

Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the gene that encodes α-galactosidase A and is characterized by pathological accumulation of globotriaosylceramide and globotriaosylsphingosine. Earlier, the authors demonstrated that oral coadministration of the pharmacological chaperone AT1001 (migalastat HCl; 1-deoxygalactonojirimycin HCl) prior to intravenous administration of enzyme replacement therapy improved the pharmacological properties of the enzyme. In this study, the authors investigated the effects of coformulating AT1001 with a proprietary recombinant human α-galactosidase A (ATB100) into a single intravenous formulation.