Summary

Cell and gene therapy title covers a wide range of therapeutic approaches for preventing diseases or curing diseases by modifying cells or genes. Startups are leading the field; big pharma owns 15% of launched products (even less for clinical or preclinical stage). More mRNA-based therapies will be in the market in 5-10 years since operations (manufacturing & delivering) will be easier with the pandemic boost. Novel technology with single-shot therapies can be more effective than traditional small molecule drugs. It is the next big thing for pharma, especially in rare diseases that are mostly incurable. It is a platform technology that can be applied to many diseases if the disease metabolism is explored in detail.

Viability (3) Validation and demonstration

Building a platform therapy harbors commercial risk; creating a treatment for a specific disease presents science risk, including the basic understanding of the disease's metabolism. Although there are 22 FDA-approved products on the market, the long-term risks of these therapies are still unknown. Clinical viability mostly depends on the targeted disease, but the infrastructures/platforms are becoming more and more viable with the help of automation of laboratories.

Drivers (5)

Advances in genetic engineering pave the way for curing genetic diseases and even creating designer organisms with the help of recently emerging technologies like CRISPR. COVID-19 is an accelerator for CGT since regulatory bodies prioritize solutions for the pandemic (mRNA vaccine is a CGT product). After the success of Biontech with both mRNA technology and investment model (partnership with big pharma), most startups and pharma are adopting the process (Biontech is replicating its success to fight cancer).

Novelty (5)

CGT promises revolutionary solutions for especially rare diseases because they are incurable and promise single shot therapies rather than months of hospitalization. Genetic modification on humans is one of the most significant endeavors of humanity. Novel CRISPR or mRNA-based solutions are on the rise today. Compared to traditional drugs, CGT is more successful since it directly targets the disease, which results in minor side effects. Big pharma acquires startups for IP.

Diffusion (2)

Cell and gene therapies have

Speed is driven by cost reduction and application against specific diseases, e.g., Covid-19. Forty planned products by 2025, mostly for rare diseases. There is friction mainly on regulatory approvals, reimbursement models, and advanced delivery methods like targeted drug delivery. Ethical debates will slow the adoption of CRISPR therapies, as will public concern. However, not-so-restrictive policies followed in the Asia-Pacific region have increased clinical trials (China accounts for more than half of CGT clinical trials worldwide).

Impact (5+) High certainty

It’s hard to look past an extremely high impact scenario for the broad field of cell and gene therapy. There are many approaches and technologies within the field that will fail, but at some point in the next decade medicine and healthcare will move from generic to personalised. This is up there with some of the greatest improvements in healthcare since germ theory. The field is at the center of new-generation therapeutics. CGT can be adopted for many diseases since it is a platform technology. Its economic impact cannot be projected correctly today, partly because its results will be visible in the long term. However, there will be more than 30 diseases for which CGT is a cure in 10 years frame; it lowers the short-term economic impact. In my view, investment is a must if therapeutics are considered in the portfolio of any company.

Timing (2025-2030) Medium certainty