Roger Hajjar, MD, on the Commercial Viability of Gene Therapy for Ultra Rare Diseases

This is the third part of an interview with Roger Hajjar, MD. For the first part, click here. For the second part, click here.

“One area that encompasses all of gene and cell therapy is the fact that many of the diseases that are being targeted are rare or ultra rare. The issue of how you can make a viable treatment that may not be commercially viable is one that now is part of our thinking, even though many of us are clinicians or scientists and don't have a lot of focus on the commercialization area.”

Gene and cell therapy research has historically put a large focus on targeting rare and ultra rare diseases. Indeed, gene therapy sometimes constitutes the only modality capable of modifying the underlying disease in rare genetic disorders, and as such is a natural choice for therapeutic development in such indications. Unfortunately, however, the high costs of gene therapy research, testing, and manufacturing, and the relatively smallpatient populations that potentially stand to benefit from rare disease gene therapy products, can often make the development of such therapeutics commercially unviable for biotech companies. As such, therapeutics that may be viable on a technical level are unlikely to become available to patients for financial reasons under current commercialization models.

In the face of this challenge, many scientists and doctors working across the fields of rare disease are seeking ways to overcome this obstacle. Roger Hajjar, MD, the director of the Mass General Brigham’s Gene and Cell Therapy Institute, emphasized the importance of this point in the context of a broader discussion with CGTLive® at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2025, in New Orleans, LA.

Hajjar also shared some of his thoughts about the current state of the field of cell and gene therapy more broadly. He pointed out that the field has had its ups and downs, and may seem to be at a lower point currently, but expressed steadfast optimism about the field’s future.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.