Process Platforms vs. Custom Process Development for AAVs and Gene Therapies
Gene therapies hold huge promise for patients with a range of monogenic diseases and unmet medical needs. However, they’ve had their fair share of safety concerns – dosing and delivery have been the sources of recent setbacks. Efficient, scalable, and cost-effective manufacturing is another hurdle the industry needs to overcome before commercially viable therapies can be licensed. AAV technology advances are also needed to address yield, material costs, and cycle times in a bid to bring down COGS and ultimately increase financial accessibility by global patient populations.
There is also a cash crunch echoing through the gene therapy industry with layoffs and company closures affecting many promising late-stage advanced therapies from commercializing. This appears to be the sharp end of a wider industry trend; the hype bubble has burst, and the promise of steady, high-valuation cash influxes has gone.
To address issues of cost, time to market and manufacturing complexity, many developers have turned to turnkey manufacturing platforms. The question is whether these platforms provide the right solution for successful CMC development. Use of platforms has come full circle – 5-10 years ago many developers were manufacturing their viral vectors at academic vector core centers. This led to academic processes that were not scalable or suitable for commercial GMP manufacturing, resulting in a big shift for biotech to develop processes that they own and control.”