Translational Applications of MiniPDX: An In Vivo Organoid Assay in New Drug R&D

Therapeutic discovery and development rely on numerous analytical techniques in order to best position candidate drugs for commercial success. Different analytical technologies are utilized for their respective strengths in maximizing understanding of candidate properties.

Dr. Limon begins this webinar with an introduction to synaptic dysfunction in human disorders, including autism, schizophrenia, major depressive disorder, and AD. All of these conditions have multifactorial etiologies and also involve changes in dendritic spine number over time that are associated with the development of symptoms. Establishing animal models for such complex disorders is inherently difficult, and even in cases where such models do exist, they fail to recapitulate all of the characteristics of the relevant human condition. Further, in some cases, such as non-demented individuals with AD pathology (as revealed on postmortem examination), no clinical evidence of underlying disease pathology is detectable. Indeed, as Dr. Limon demonstrates, a reduction in cortical thickness (as much as one third) with age may be observed without accompanying cognitive impairment, at least in part due to brain reorganization and plasticity.

As gene therapies and gene-modified cell therapies show increasing promise, the need for innovative and proficient viral vector manufacturing continues to grow. Concurrently, increased regulatory guidance governing the manufacturing and testing of viral vectors adds complexity and increases the timelines to successfully produce high-quality virus ready for clinical use.

Interleukin-11 (IL-11) is an interesting and unique pleiotropic cytokine, originally characterized as a hematopoietic cytokine with thrombopoietic activity.