This post first appeared as an issue of the a16z Bio Newsletter. Subscribe to stay on top of the latest trends in bio and healthcare.
IN THIS EDITION:
Like everything in 2020, the ARM Cell & Gene Meeting on the Mesa was a little bit different this year. While the Mesa was swapped for home offices, closets, and garages, the incredible science and energy driving these modalities forward remained constant. My high-level summary of the meeting is below.
The Big Themes
The entire industry ecosystem must evolve, and fast.
Many panel discussions centered around the systemic challenges standing between cell & gene therapies and patients, including: (1) regulatory approvals, (2) market access, pricing and reimbursement, (3) manufacturing and supply chain, and (4) care delivery. Scaling models used by first movers (e.g., Zolgensma, Luxturna, Yescarta, Kymriah) is neither operationally nor economically sustainable. Payor hurdles are particularly steep, with high front-loaded costs and no guarantee that the efficacy of one-time treatments will last. The industry has toyed with value-based contracts that spread payor costs, but agreements developed thus far are too complicated and expensive to implement for more than a handful of drugs.
How can we derisk “one and done” modalities for new indications and patient populations?
One-time, curative treatments for rare, debilitating conditions have been the first areas of focus. But while “one and done” treatments sound good, it isn’t ideal for most patients or most conditions. Cutting-edge research may enable us to: (1) branch out from simpler, monogenic diseases and (2) expand into patient populations beyond the most severe, while maintaining appropriate risk-benefit and cost efficiency requirements.
The blistering pace of scientific progress continues.
The immense challenges we face in delivering cell & gene therapies are countered by the astonishing scientific progress driving improvements in safety, efficacy, flexibility, and scalability of these platforms. Four key areas moving the scientific needle are: (1) viral vector and novel gene delivery mechanisms, (2) allogeneic cell therapy production, (3) the gene editing toolbox, and (4) immune cloaking. These innovations continuously bring us closer to getting transformative therapies to the patients that need them.
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