December 2023

• The cell and gene therapy space has seen significant growth in the last 5 years and continues to rapidly evolve
• Currently, approved non-oncology, DNA-based gene therapies are restricted to rare monogenic diseases
• However, as technology becomes more advanced it is likely that gene therapy will have stronger potential to treat more prevalent diseases
• The success of future gene therapies will be dependent on addressing key technical, clinical, and commercial challenges

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Applications of regenerative medicine, including gene therapy, cell-based gene therapy, and cell therapy, vary greatly

Our analysis focuses on non-oncology, DNA-based GTx approaches, including gene editing, gene replacement, and non-oncology gene addition

All approved GRTs are for the treatment of autosomal recessive, monogenic diseases

In the last two years, the number of approved GRTs has doubled; single-dose therapies have an average list price of $3.0M/patient

GRT is the most common and mature approach in the GTx pipeline; a minority of agents employ novel non-viral delivery methods while the majority use classic viral vectors

Gene replacement/editing approaches tend to focus on monogenic diseases with known etiologies; gene addition offers potential in diseases with unknown disease-causing mutations

As the gene therapy space continues to rapidly evolve, addressing technical, clinical, and commercial dynamics will be critical to success

The Authors