Kulika Weizman
Therapeutics has been at the core of systematic healthcare solutions—relying on our survival instinct to tackle sicknesses as they arise.
Therapeutic Treatments Have Advanced Rapidly Over the Years
While our ancestors sought naturally occurring active ingredients to soothe their symptoms, our generations are more familiar with taking prescription drugs based on diagnosed diseases. Their therapeutic properties are functions of active ingredients called Active Pharmaceutical Ingredients (APIs).
Small molecule APIs paved the way and became mainstream, thanks to the scalability nature of chemical syntheses. Then entered biological APIs. While there are still fewer biologics in the market today, they command substantially higher prices. Biologic APIs’ core values are their ability to interact with the system as peptides and proteins, which may be recognized as native biologic molecules such as hormones or antibodies. They are produced using biological systems, resulting in higher production costs.
Biologic APIs Are Good; Gene and Cell Therapies Could Be Great
Despite our reliance on a rather complicated production process, biologic APIs are already produced and commercialized at scale today. Though it unveils the gap in our ability to produce a more advanced mode of therapeutics.
Advanced therapies, specifically gene and cell therapies are the next frontiers. While small molecule and biological APIs may ease symptoms, they rarely address the root cause. This is particularly relevant for chronic diseases, which happen to be responsible for most of the U.S. healthcare spending.
Instead of treating these diseases on a recurring, symptom-based basis, therapeutic genetic materials, or cells may be introduced to patients, targeting internal modification or repairs. The ultimate goal of which is the eradication of these diseases.
Despite their promises, there are still a very limited number of advanced therapies approved by the FDA today, and they do not come cheap. At over a million US dollars per treatment for some, they are far from accessible.
"Having a robust infrastructure for advanced therapy production and improving yield and quality will uniquely contribute to the developers’ ability to expand to metastatic diseases, limit the treatment toxicities, and improve the overall effectiveness"
As with any novel product, the initial phase of market penetration targets those who have the capability and willingness to pay. Though with a few thousand advanced therapies going through clinical trials today, more development should be devoted to how these advanced therapies are made.
Lab Vs. Commercial Scales
Similar to scaling the industrial synthetic biology process, advanced therapy manufacturing faces a challenge between scale-out and scale-up tradeoffs. At a lab scale, they are made on a small scale in stacked trays, but it does not produce enough materials for when the therapies advance to later stage clinical trials, let alone commercially.
One approach is to move away from stacked trays and toward bioreactors. In theory, this would enable a larger suspension volume to allow for higher output. Unfortunately, biology rarely scales linearly, so a substantial amount of work, including regulatory processes, is often required in order to switch between manufacturing methods.
Manufacturing Remains the Next Great Frontier
Delivery and efficacy have been at the center of advanced therapy development. Though with more moving through clinical trials, how they are made in a scalable and cost-effective manner is still largely an untapped opportunity.Having a robust infrastructure for advanced therapy production and improving yield and quality will uniquely contribute to the developers’ ability to expand to metastatic diseases, limit the treatment toxicities, and improve the overall effectiveness. The manufacturing process will become a key component of the therapy products.
