Photo: Bryan Goodchild
A new paper published in Molecular Therapy Review co-authored by Terence R. Flotte, MD, the Elisabeth Chair for the Dean of Medicine, executive deputy chancellor, provost and dean of the T.H. Chan School of Medicine, lays out a comprehensive plan for addressing the current market challenges for gene therapy treatments of rare and ultra-rare disorders.
Gene and cell therapies offer transformative potential for patients with rare and ultra-rare diseases. Over the last two decades great strides have been made in moving basic gene therapy discoveries into the clinic. However, an increasing number of pre-and early-stage clinical trials have stalled or been discontinued by the biotech industry, not because of a lack of efficacy or safety, but due to financial and market challenges. According to reporting from Rare Disease Advisor, as many as 100 life-changing gene therapies may have been shelved since 2023 for financial reasons and limited market commercialization potential.
By pooling resources, standardizing processes and coordinating actions across nonprofits, private companies, patient advocacy groups and regulatory agencies, the authors said the gene therapy field can bring cutting-edge gene editing treatments to patients suffering from rare and ultra-rare disease.
“There is an ethical imperative to making transformative gene and cell therapies more accessible for patients who could benefit from them,” said Dr. Flotte, the outgoing president of the American Society of Gene & Cell Therapy. “The next step is to implement strategies and complimentary models to address the different aspects of commercial viability of these early-stage gene therapies.”
There are more than 7,000 known rare diseases and about 250 more are identified annually as more human diseases are genetically characterized and identified. Although the definition of a rare disease differs around the world, the U.S. Food and Drug Administration defines it as a malady that affects fewer than 200,000 people in the United States. Some of the more ultra-rare diseases may afflict only 40 to 50 people worldwide.
In the paper, the authors call for a two-pronged strategy focused on maintaining discontinued gene therapy programs and supporting existing developers of gene and cell therapies for rare and ultra-rare diseases. Among the recommendations are aligning of international regulations to reduce costs and speed development; accelerating regulatory approval and flexibility for rare diseases; reducing manufacturing costs through public infrastructure, nonprofit-led vector production and regulatory changes; implementing alternative financial models that tie payment structures to long-term patient outcomes; and bundling rare disease programs using a platform approach to distribute costs across larger patient populations.
“By working together, collaboratively, across these areas, we can translate scientific breakthroughs into accessible treatments for all patients with rare genetic diseases regardless of the commercial considerations that have historically limited their development,” said Flotte.
Among the co-authors of the paper are colleagues from the American Society of Gene & Cell Therapy. This week, more than 8,000 basic and translational researchers from around the world, including 59 from UMass Chan, came together in Boston for the 2026 annual meeting of the society, which is the largest gathering of cell and gene therapy professionals in the world.
