In the last few decades, gene therapy has gone through significant developments, but still it has to go far for widespread application in healthcare. Cellular and gene therapy faces multiple regulatory hurdles and fails to comply with the guidelines. Understanding the regulatory framework and prominent healthcare models is vital for any clinical development.
Challenges faced by cellular and gene therapy
Though cellular and gene therapy has been emerging as a revolutionary solution to genetic disorders, and these groundbreaking therapies have proven their potential for lifesaving capacities around the world, they still have miles to go in overcoming the stringent barriers.
- Lack of sufficient non-clinical models and efficacy is a rising concern over clinical development of cellular and gene therapy. The animal model selection appropriately before clinical trials on humans is a challenging factor. The non-clinical program must be designed and planned before human trials.
- The lack of an established regulatory framework for advanced therapies acts as a potential barrier. The commercialization of products faces difficulty due to the existence of separate regulatory frameworks in different countries, and there are significant differences among them. The regulatory framework universally should support the predictability, flexibility, and efficiency of therapies, considering patient safety.
- Inclusion of key elements such as a new innovative framework for regulating gene therapy, advanced clinical trial design, and post-market confirmatory research studies are the crucial factors to make the implementation of gene therapies on a global scale.
- Cellular and gene therapy face major challenges regarding the quality, efficacy, and safety of data to predict the benefit-to-risk ratio. The small patient population for rare diseases creates the hindrance of large-scale applications.
- Gene therapies face some long term risk of immunogenicity in the cases where anti-drug antibodies create complications in the treatment. Moreover, the chances of tumorigenicity also raise concern over the application of cellular and gene therapy.
- The critical quality attributes (CQAs) of gene therapy products also play as critical challenges, and many times they are not well-established before application. The gene therapy products and their clinical outcomes are often unpredictable and suffer from efficacy.
- Capacity constraint is also a factor behind the implementation. The high cost of goods, the need for long-term investment, and, moreover, the long duration of research and development have a substantial impact on the clinical development of cellular and gene therapy.
Cellular and gene therapy has gone through significant development in recent years, but there is a need for a global regulatory framework to support the evaluation and regulation of gene therapy products. Organizations like Allucent are helping move these treatments forward so they can reach more people around the world. Gene therapies can be a new hope for patients suffering from debilitating diseases.
Frequently Asked Questions
What are the major challenges faced by gene therapy development?
Lack of efficacy of therapies, quality attributes, immunogenicity, high cost of development, and global regulatory framework are the major hurdles in gene therapy.
What is gene therapy?
Gene therapy is a treatment procedure that fixes the faulty genes with healthy genes to cure the disease.
What are some examples of gene therapy?
Spinal muscular atrophy (SMA) and Luxturna are some examples of approved gene therapies.
