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Improving access to gene therapy for rare diseases. | LitMetric

Improving access to gene therapy for rare diseases.

Dis Model Mech

Infection, Immunity and Inflammation Department, UCL Great Ormond Street Institute of Child Health, UCL, London WC1N 1EH, UK.

Published: June 2024

AI Article Synopsis

Article Abstract

Effective gene therapy approaches have been developed for many rare diseases, including inborn errors of immunity and metabolism, haemoglobinopathies and inherited blindness. Despite successful pre-clinical and clinical results, these gene therapies are not widely available, primarily for non-medical reasons. Lack of commercial interest in therapies for ultra-rare diseases, costs of development and complex manufacturing processes required for advanced therapy medicinal products (ATMPs) are some of the main problems that are restricting access. The complexities and costs of navigating the regulatory environments in different jurisdictions for treatments that affect small numbers of patients is a problem unique to ATMPS for rare and ultra-rare diseases. In this Perspective, we outline some of the challenges and potential solutions that, we hope, will improve access to gene therapy for rare diseases.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11051979PMC
http://dx.doi.org/10.1242/dmm.050623DOI Listing

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