Newsletter | February 8, 2024

02.08.24 -- Gene Therapy Advancements: Natural History Studies And Rare Disease Milestones

Gene Therapy Natural History Studies: Benefits, Timing, And Execution

A traditional regulatory pathway requires being able to accurately describe the normal clinical course of the disease. This can be difficult with rare diseases because existing academic data is incomplete and/or poorly controlled. Moreover, data rarely exists that is relevant to specific, high-sensitivity endpoints needed for a gene therapy study.


Rare Disease Clinical Endpoints: Ingenuity Meets Practicality

From a scientific research perspective, finding a way to proliferate or silence (as needed) the protein of interest equals success. But once medical scientists become involved, the translational bridge must be constructed — arguably, the hardest step in gene therapy development. How do we apply that science and make it meaningful?


High-Velocity Development: Gene Therapy Vs. Small Molecule

In gene therapy, clinical tasks overlap, and translational medicine digs deeper and takes longer, making the crunch on chemistry, manufacturing, and controls (CMC) personnel, as well as lab personnel, difficult to navigate. Explore how a carefully plotted approach can minimize risk and maximize chances for success.



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