Trials and Triumphs of Gene Editing: A Compendium

THE COWEN INSIGHT

While offering curative potential, much remains to be known about the long-term clinical activity and safety of gene editing therapies. We expect answers to these complex questions will continue to be a key investor focus as programs advance.

While the clinical and regulatory progress of gene therapies may inform the road ahead for gene editing, permanent genomic changes will likely yield distinct advantages and challenges. Advancements in editing efficiency, the characterization of on – and off -targeted editing, and pre -conditioning regimens are likely focus areas in the years ahead. We have identified nearly 190 gene editing programs across broad therapeutic areas.

The Pre-Clinical and Clinical Gene Editing Landscape Is Beginning To Heat Up

Gene editing technologies function to disrupt, correct, replace, or insert genetic material in patient cells in order to treat or prevent disease. Investment and progress in the sector has been rapid advancing toward the clinic and the public market. This is especially true following the discovery of CRISPR/Cas with novel iterations and approaches to editing being developed in the lab.

Our analysis indicates that gene editing companies raised ~$3.5B in proceeds thus far in 2021 alone vs. ~$250MM five years ago. We have identified nearly 190 gene editing-related programs currently underway at public and private companies across diverse therapeutic indications highlighting successful developments in the field are set to address substantial markets.

What We Know About Gene Editing

The gene editing landscape continues to make rapid strides, and we have now seen initial clinical data from key gene editing players. These programs have demonstrated the potential of gene editing through both ex vivo and in vivo approaches across diverse disease indications. The field is quickly translating findings in the lab into patients while also taking lessons from the development paths of gene and cell therapies to enhance aspects of clinical trial design, manufacturing, and administration.

Where Do We Go?

Despite the advances, much remains to be clarified. How will companies manage screening for off-target effects and how closely will the FDA monitor these requirements? Is full gene replacement necessary for maximum efficacy or are disruption techniques sufficient? What length of efficacy and safety follow-up will regulatory authorities, patients, and physicians require ahead of use? What are the ideal pre-treatment regimens for each agent and indication? To cut or not to cut? We expect that continued innovation over the next several years will help to answer several of these key questions.

We outline current company progress and investor sentiment on these topics through a comprehensive analysis of the landscape and survey work.

Focus for Outperformance in the Gene Editing Sector

While gene editing companies are largely still in the process of finding their stride, we believe key company attributes tend to highlight a solid organization. This includes robust methods for detecting accurate editing and data packages supporting a lack of off-target effects, early and open communication with the FDA, innovative approaches to delivery or pre-conditioning, and evidence of reproducible editing efficiency in pre-clinical models. Our report details advancements at over 40 public and private companies that leverage the gene editing toolbox for therapeutic development or discovery.