Perspectives

What do medical market research and health economic outcomes research have in common? How can we combine our work on burden of disease in a way that realizes it fullest potential? What can we learn from each other’s approach?

Review our published works highlighting our projects and research.

Amidst success of in vivo adeno-associated virus (AAV) based gene therapies, the gene therapy community awaits key outcomes from next month’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) meeting discussing toxicities and deaths associated with high titer AAV dosing and potential long-term adverse events. While this discussion evolves, a significant advance for in vivo non-viral gene editing was published in a landmark report by Intellia and Regeneron demonstrating, for the first time, the safety and efficacy of non-viral, lipid nanoparticle (LNP) administered CRISPR-Cas9 gene editing in the human liver. The report demonstrated that NTLA-2001 was efficiently delivered to the liver and knocked out the TTR gene in hepatocytes with a single dose treatment. Accumulations of misfolded transthyretin in systemic tissues is responsible for Amyloidosis (ATTR), which presents clinically with polyneuropathy and/or cardiomyopathy. While chronically administered drugs (tafamidis/Vyndaqel, inotersen/Tegsedi and patisiran/Onpattro) for ATTR have recently been developed, one-time treatment of NTLA-2001 has the potential for lifelong reduction of TTR protein and reversing disease progression.

A global biopharmaceutical company wanted to understand how influential stakeholders within the online and offline landscape for a specific type of cancer are currently engaging and influencing the wider community. Specifically, the team sought to identify who is driving discussions on- and off-line, and what those discussions look like – in order to inform scientific story evolution and optimize communications programming through engagements and activations.