A new gene therapy delivery device could let hospital pharmacies make personalized nanomedicines to order. This democratized approach to precision medicine, as published in Frontiers in Science, could revolutionize how hospitals treat rare diseases, even in low-resource settings.
Rare diseases affect millions worldwide, yet the one-size-fits-all model of drug development leaves patients with few treatment options. Now a European research project called NANOSPRESSO aims to tip the balance in patients’ favor by boosting access to low-cost bespoke gene and RNA therapies.
The prototype NANOSPRESSO device combines two proven technologies— nucleic acid therapeutics and lipid nanoparticles—into a portable manufacturing unit. Hospital pharmacists could use the unit to prepare sterile, injectable nanomedicines tailored to the specific genetic abnormality causing the patient’s condition, bypassing the need for centralized drug production.