AGTC CORPORACIÓN DE TECNOLOGÍA GENÉTICA ANUNCIA LA INVESTIGACIÓN DE UNA NUEVA DROGA PARA LA FASE I/II DE LA TERAPIA GÉNICA PARA EL TRATAMIENTO DE LA ACROMATOPSIA LIGADO A LA MUTACIÓN DEL GEN CNGA3
LA ACROMATOPSIA ES UNA ENFERMEDAD CONGÉNITA HEREDITARIA QUE DA UN RESULTADO DE REDUCCIÓN DE VISIÓN, SENSIBILIDAD A LA LUZ E INHABILITACIÓN PARA VER LOS COLORES.
AGTC Files IND Application to Study Second Achromatopsia Drug Candidate
OCTOBER 21, 2016: BY JOAN MCKENNA
AGTC Applied Genetic Technologies Corporation (AGTC) announced Oct. 19 that is has filed an investigational new drug application with the US FDA to conduct a Phase I/II clinical trial of its gene therapy for the treatment of achromatopsia caused by mutations in the CNGA3 gene.
Achromatopsia is an inherited retinal disease resulting in reduced visual acuity, extreme light sensitivity, and the inability to see color.
The adeno-associated virus-based gene therapy already is in Phase I/II study for achromatopsia involving mutations in the CNGB3 gene.
The Gainesville, Florida, and Cambridge, Massachusetts, firm also is developing therapies for X-linked retinoschisis, X-linked retinitis pigmentosa, wet age-related macular degeneration, and blue cone monochromacy.