Trials and Triumphs of Gene Therapy: A Compendium

THE COWEN INSIGHT

Over the past several years, investment in the gene therapy sector has been robust. The data from 2020 indicate there is no sign of stopping. While technology and clinical programs in the field have advanced rapidly over the past five years, we expect the next five years will yield even more answers regarding the promise of gene therapy approaches to potentially cure disease.

Gene Therapy Programs Continue To Expand In Number And Scope

Gene therapies deliver targeted genetic material to patients in order to treat or prevent disease. While the field has progressed over decades, investment and progress in the sector has grown rapidly over the past five years. 

Our analysis indicates that gene therapy companies raised nearly $4B in proceeds during 2020 alone. Investment in the sector is largely due to the wide potential implications for advancements in the field. We have identified 284 gene therapy programs currently underway at public and private companies across more than 130 different disease indications. Successful developments in the field are set to address substantial markets.

What Do We Know And Where Do We Go?

The gene therapy industry has made great strides, marked notably by the U.S. approvals of Luxturna in 2017 and Zolgensma in 2019. These programs demonstrated:

1. Genetic material could successfully be delivered to various tissue sites and confer benefit
2. The FDA was willing to grant approvals for such transformative therapies
3. Rigorous gene therapy programs may often be the target of lucrative M&A transactions

The field has taken lessons from the development of these and other therapies to enhance aspects of clinical trial design, manufacturing, administration, and the applicability of the technology at a rapid pace.

Despite the advances, much remains to be clarified. How will the FDA treat disease indications with an acceptable standard of care vs. orphan indications with no effective treatments? How can the durability or degree of protein production be amplified across targeted tissues? How will these therapies be paid for? How near is the holy grail of effectively re-dosing patients? We expect that continued innovation over the next five years will help to answer several of these key questions.

Focus For Outperformance In The Gene Therapy Sector

While success in gene therapy programs is never guaranteed, we believe certain company attributes tend to highlight a solid organization including:

1. Robust pre-clinical investigation in applicable non-human primate models
2. Early alignment on manufacturing requirements for clinical trial material
3. A clear plan to manage AEs and early immune responses
4. An understanding of the appropriate patient population to investigate

Our report details 50 publicly-traded companies and 20 private companies that have a gene therapy asset in the pipeline.