Généthon is on a mission to make cell and gene therapies (CGTs) both safe and affordable, addressing a critical barrier for patient access. The exorbitant costs associated with CGTs often deter payers, particularly for rare diseases where commercial viability is limited. As Frédéric Revah, PhD, CEO of Généthon, highlights, approximately 85% of rare diseases are deemed too rare for traditional pharmaceutical companies to pursue profitably.
With a focus on rare and ultra-rare diseases, Généthon recognizes that reducing manufacturing costs is essential for delivering these life-saving therapies to patients. Current manufacturing expenses can reach millions of dollars, a figure they aim to lower to tens of thousands. The organization is leveraging innovative biomanufacturing methods, collaborating with industry and academic partners to explore new technologies that enhance production efficiency.
As Généthon advances its pipeline, including a Phase III trial for a microdystrophin gene therapy for Duchenne muscular dystrophy, the implications of these efforts could reshape the landscape of CGT accessibility. By prioritizing cost-effective production and efficacy, Généthon is positioning itself as a leader in making transformative therapies more attainable for those in need.
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