Noticia/ Headline
Un nuevo estudio marca un avance significativo en el desarrollo de una terapia génica para la retinitis pigmentosa ligada al cromosoma X, altas expectativas para iniciar el ensayo clínico fase 3.
A new study marks a significant advance in the development of a gene therapy for X-linked retinitis pigmentosa, high expectations for starting the phase 3 clinical trial.
Resumen
La retinitis pigmentaría ligada al cromosoma x es una enfermedad hereditaria que conduce a una pérdida de visión grave en los hombres jóvenes. Investigadores del Bascom Palmer Eye Institute, del Departamento de Oftalmología de la Facultad de Medicina Miller de la Universidad de Miami, participan en el estudio multicéntrico internacional. “Los resultados de este ensayo inicial no mostraron preocupaciones de seguridad significativas después de la cirugía de terapia génica”, dijo el Dr. Davis. “Se observaron mejoras en el campo visual en algunos pacientes, y estos hallazgos favorables respaldan la amplia investigación de terapia génica de Bascom Palmer”.
Summary
X-linked retinitis pigmentosa a hereditary disease that leads to severe sight loss in young males. Data being presented at the American Society of Retina Specialists (ASRS) 2020 Virtual Annual Meeting. Significant improvements demonstrated after treatment in Phase 1/2 clinical trial. Based on encouraging safety and efficacy profile, MeiraGTx and Janssen expect to progress AAV-RPGR into Phase 3 Lumeos clinical trial. (1)
Researchers at Bascom Palmer Eye Institute, the Department of Ophthalmology at the University of Miami Miller School of Medicine, took part in the international multi-center study and are actively participating in further clinical trials. “The results from this initial trial showed no significant safety concerns after gene therapy surgery,” said Dr. Davis. “Visual field improvements were observed in some patients, and these favorable findings support Bascom Palmer’s wide-ranging gene therapy research.” (2)
Acceso completo a la noticia:
Complete News access:
(2) https://physician-news.umiamihealth.org/new-study-advances-development-of-gene-therapy-for-x-linked-retinitis-pigmentosa/
