Q2 2023 Gene Therapy Report: Promising new treatments on the horizon

The potential for gene therapies to treat – or even effectively cure – debilitating medical conditions continues to expand. Our latest quarterly gene therapy pipeline update includes information about treatments currently under review that may soon gain approval to treat conditions such as:

• Duchenne muscular dystrophy (DMD) – The first gene therapy for treating DMD could be approved later this year.
• Hemophilia A – Hemgenix, a gene therapy to treat severe hemophilia B, was approved in 2022. There may soon be one to treat severe hemophilia A as well. Hemophilia A is much more prevalent – representing 80 percent of overall hemophilia cases.
• Sickle cell disease – There are two gene therapy agents pending approval for the treatment of sickle cell disease. This could mean going from zero gene therapies to treat the condition to having two.

Looking ahead

Going into mid-2024 and beyond, new gene therapies may treat conditions that affect larger populations. Examples include:

• Diabetic peripheral neuropathy, which affects 5 to 11 million adults in the United States
• Neovascular (wet) age-related macular degeneration, which affects about 1 million US adults
• Knee osteoarthritis, affecting 16 million US adults

Keeping a close eye on the gene therapy pipeline is increasingly critical as the potential for gene therapy grows, so that plan sponsors can be proactive in developing strategies to manage access and cost.