Cellular and gene therapies are expanding beyond their initial focus on rare genetic diseases and hematologic cancers toward a broader range of conditions. Key areas of innovation include more precise gene-editing technologies, improved gene-delivery platforms, next-generation CAR-T and other engineered cell therapies, and the development of scalable or “off-the-shelf” treatments. Over the next several years, the field is expected to continue expanding into oncology and autoimmune diseases, while also focusing on improving safety, manufacturing capacity, affordability, long-term durability, and patient access.

The results of PHSL’s August poll on the greatest focus for cellular and gene therapy over the next 5 years indicate that oncology is expected to remain the leading area of focus, followed by other rare genetic diseases. Respondents also believe there will be continued interest in expanding successful approaches to treatment of hemophilia and blood disorders and neurological disorders.

Where do you expect the greatest focus in cellular and gene therapy over the next 3–5 years?

Oncology: 43%

Other rare genetic diseases: 29%

Hemophilia and blood disorders: 14%

Neurological disorders: 14%

Autoimmune diseases: 0%

Cardiovascular disease: 0%