The economic downfall of gene therapies for rare diseases threatens access to life-saving treatments.
Story Highlights
- Gene therapies for rare diseases are often abandoned due to prohibitive costs.
- The high-profile withdrawal of treatments leaves many patients without options.
- Funding for gene therapy development has seen a significant decline since 2021.
- Advocacy groups call for innovative funding models to sustain access.
Economic Challenges in Gene Therapy Development
Gene therapies for rare diseases face daunting economic challenges, often leading to their discontinuation. Despite scientific breakthroughs, the cost of manufacturing and commercializing these therapies for small patient populations is unsustainable. Pharmaceutical companies find it difficult to recoup expenses, resulting in the withdrawal of treatments even after they receive approval. This economic barrier leaves many patients without access to potentially life-saving therapies.
Over the years, the initial enthusiasm for gene therapies, fueled by early successes, has waned due to economic realities. The approval of treatments for conditions like spinal muscular atrophy initially inspired optimism. However, the financial burden of maintaining these therapies for ultra-rare diseases has proven too great. Companies have been forced to pull products from the market, as seen with bluebird bio’s withdrawal of Zynteglo in Europe, highlighting the need for sustainable solutions.
A new #MachineLearning approach is helping to estimate the disease risk of rare genetic variants, researchers report in Science.
According to the findings, the method produces more precise, individualized measures of penetrance than traditional methods and could potentially… pic.twitter.com/AWyLGPBY68
— Science Magazine (@ScienceMagazine) September 6, 2025
Decline in Funding and Investment
Since 2021, funding for cell and gene therapies has plummeted by over 80%, with venture capital and private equity investments reaching historic lows. Major pharmaceutical companies are scaling back research and development for rare diseases, citing unsustainable economics and poor returns on investment. This downturn threatens to stall innovation in treating rare diseases, leaving a significant gap in the availability of therapies for patients in dire need.
Advocacy groups and industry experts are calling for new funding models and partnerships to address these challenges. Proposals include venture philanthropy and innovative payment schemes to incentivize the development and commercialization of gene therapies. Without systemic change, many promising treatments will never reach the patients who need them, and those that do may be withdrawn after approval due to economic pressures.
Watch: Advancing the Development of Gene Therapy for Rare Diseases Center for Individualized Medicine 2024
Impact on Patients and the Industry
The consequences of the economic barriers to gene therapy development are profound, affecting both patients and the pharmaceutical industry. In the short term, patients lose access to potentially curative therapies, while companies face financial losses and reputational risks. In the long term, the lack of innovation in rare disease treatment could erode trust in the pharmaceutical industry and regulatory system, as unmet medical needs persist.
Patients with rare and ultra-rare diseases, along with their families, are directly impacted by these developments. Additionally, small biotech firms and academic researchers face significant challenges as R&D efforts shift toward more profitable areas. There is growing pressure on policymakers to reform orphan drug incentives and reimbursement models to ensure those in need have access to life-saving treatments.
Sources:
Advanced Therapies Week 2025 Panel Discussion
Applied Clinical Trials Online, “The Hard Truth About Rare Disease and Gene Therapy Drug Development”
Labiotech, “Cell and gene therapy funding: Trends and challenges in 2025”
ASGCT Policy Statement, “Ensuring patient access to gene therapies for rare diseases”
