Nathan Yozwiak, PhD, on Commercial Bottlenecks in Gene Therapy Development
The head of research at Mass General Brigham’s Gene and Cell Therapy Institute discussed how innovative research can be held back by financial considerations.
“How do you create a sustainable model for this? This has come up repeatedly throughout this conference and it's something that we as a field really need to tackle.”
Gene therapy has proven to be a remarkably promising new modality for a wide range of diseases. Although, in some cases, innovative technologies and research can be held back by the costs associated with development and potential financial viability as a commercial product. For example, many gene therapy candidates target very rare diseases, and thus would likely only be administered to a handful of patients each year if they were approved by the FDA.
In a conversation with CGTLive® at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2024, in New Orleans, LA, Nathan Yozwiak, PhD, the head of research at the Gene and Cell Therapy Institute at Mass General Brigham, discussed this phenomena. He pointed out that while scientific breakthroughs are accelerating, commercial and financial models are lagging behind, and that this issue has been discussed often at ASGCT’s meetings.
To address the issue, Yozwiak emphasized the importance of continued innovation in technical aspects of gene therapy, such as improving vectors, utilizing AI for more efficient design, and refining delivery mechanisms. These innovations aim to lower costs and improve efficiency, potentially changing the economic feasibility of rare disease treatments. He noted that he sees the field approaching a major inflection point soon where several big advancements may push the field very far forward.
Yozwiak is especially excited about in vivo gene editing approaches, which allow direct treatment inside the body rather than requiring cells to be removed and reintroduced. He also notes progress in adeno-associated virus (AAV) vectors, particularly in engineering capsids for more targeted and cost-effective delivery. These advancements could replace the current high-dose, systemic approaches, improving safety and precision.
Click here to view more coverage of the 2025 ASGCT Annual Meeting.
Eugenio Galli, MD, PhD, on Reexamining Frailty as a Barrier to CAR-T Treatment
June 16th 2025The hematologist at the Hematology and Stem Cell Transplants Unit of Fondazione Policlinico Universitario A. Gemelli IRCCS in Rome discussed a study on the effect of comorbidities in patients receiving CAR-T.
