The Importance of Addressing Gene Therapy's Financial Viability in 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.

Gene and cell therapy modalities are well-posed to tackle certain rare and ultra rare diseases on a technical level. Unfortunately, however, because of high manufacturing costs and the relatively small populations of patients likely to benefit from any given therapy, commercialization of such therapies may be financially unviable.

In an interview with CGTLive® at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2025, in New Orleans, LA, Roger Hajjar, MD, the director of the Mass General Brigham’s Gene and Cell Therapy Institute, brought attention to this issue. Although, he pointed out that a number of scientists and clinicians are now thinking about this aspect during the research and development process, and attempting to find models to overcome this challenge. Hajjar also shared his optimism and enthusiasm regarding the future of the cell and gene therapy field in general.

CGTLive: Looking ahead to the future, are there any major challenges still need to be tackled in the field of cell and gene therapy?

Roger Hajjar, MD: I think 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.

So that's becoming important, and solutions are being brought to these ultra rare diseases, or are at least being planned. I think that's to me exciting because the clinicians and the biologists are coming together with issues of financing and commercialization that are so important for the patients for specific reasons, and I think that's going to be important. Looking forward, we hope to see some models that will benefit the patients mainly, despite the fact that the commercialization may not be realizable.

Is there anything else you want to add?

I just want to share that the gene and cell therapy world has been going through oscillations, a little bit of a roller coaster ride, and we've had some highs recently and now we're maybe a little bit more on the lower side, just because of the environment we're living in. But overall, I feel very optimistic about the field. The treatment modalities that gene and cell therapies bring in are so unique and so precise that they can only go one way, and that's up, so I'm quite optimistic about the field.

This transcript has been edited for clarity.

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