Gene therapies (GTx) are a significant breakthrough in the pharmaceutical industry, with the potential to treat genetic disorders by modifying defective genetic material. Luxturna has already restored sight in some patients, highlighting the therapies’ effectiveness. However, these treatments come at a high cost, up to USD 3.5 million per dose for Hemgenix, a hemophilia B infusion. Manufacturing gene therapies requires entirely new modalities that lack the efficiency and cost savings of traditional pharmaceutical production. Additionally, production speeds are currently slow. Despite these challenges, efforts are underway to exploit the technology and reduce costs. This article provides a market overview of GTx, discusses manufacturing challenges, and explores promising technologies to overcome them.
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Gene therapies (GTx) are a significant breakthrough in the pharmaceutical industry, with the potential to treat genetic disorders by modifying defective genetic material. Luxturna has already restored sight in some patients, highlighting the therapies’ effectiveness. However, these treatments come at a high cost, up to USD 3.5 million per dose for Hemgenix, a hemophilia B infusion. Manufacturing gene therapies requires entirely new modalities that lack the efficiency and cost savings of traditional pharmaceutical production. Additionally, production speeds are currently slow. Despite these challenges, efforts are underway to exploit the technology and reduce costs. This article provides a market overview of GTx, discusses manufacturing challenges, and explores promising technologies to overcome them.