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A common question I get from my cancer cell therapy clients at Cello Health BioConsulting is why, despite the lion share of clinical data in support of T-cell therapies, it is NK cell therapy candidates (iPSC- and cord-derived) that seem of late to be capturing so much more enthusiasm from investors and Pharma these days?

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.

At the 3rd Annual Emerging Frontiers in Oncology Summit, Clarion Managing Director, Dennis Chang, delivered a talk titled "From 'Precision Medicine' To 'Inclusion Medicine'”.

There is much talk of the old and new normal, but the reality of the future will be a blend of the two, and the patients’ journey with their disease will have a different shape going forward.