🔍 They are developing innovative biomanufacturing methods to cut costs significantly. Collaborations with industry leaders focus on improving yields and efficiencies using AI and new delivery vectors.
💡 The goal? Transform CGTs into affordable solutions for all patients in need.
Introduction:
The article discusses Généthon’s initiative to enhance the affordability and efficiency of cell and gene therapies (CGTs) through innovative biomanufacturing processes. Recognizing the high costs associated with CGTs, particularly for rare diseases, Généthon emphasizes the need for cost-effective solutions that can democratize access to these life-saving treatments. The article highlights strategic collaborations and novel technologies aimed at reducing manufacturing expenses while improving therapeutic efficacy.
- Généthon aims to make CGTs affordable by decreasing manufacturing costs, which can currently reach millions of dollars.
- Innovative biomanufacturing methods are being developed to improve clinical and production efficiency, with a focus on rare and ultra-rare diseases.
- Collaborations with companies and academic institutions are essential, including partnerships with Eukarÿs and Thales to enhance protein production and utilize AI in bioprocessing models.
- Généthon’s upcoming Phase III trial for a microdystrophin gene therapy for Duchenne muscular dystrophy aims to provide a more effective treatment option at a lower dose.
- The company is researching advanced delivery vectors, including AI-designed capsids and immune modulators, to improve treatment specificity and expand gene therapy access to seropositive patients.
Conclusion:
Généthon’s focus on innovative biomanufacturing and collaborations signals a transformative approach to making CGTs more accessible and affordable. By leveraging AI and advanced vector technologies, the organization aims to not only improve manufacturing processes but also enhance treatment efficacy, ultimately addressing the pressing challenge of high costs in the realm of rare diseases. Future developments may significantly shift the landscape of CGT affordability and availability, offering hope to patients with limited treatment options.
