“Moving our LCA10 program forward and expanding our pipeline with the addition of a minigene research program for USH2A further validates our commitment to develop innovative and life changing gene therapies for patients with orphan inherited retinal diseases,” stated Glenn P. This is a group of orphan IRDs that includes Usher syndrome type 2A and USH2A-associated autosomal recessive nonsyndromatic retinitis pigmentosa. The Company also announced today that it is expanding its gene therapy portfolio by entering into a sponsored research agreement with UMass Medical School and an exclusive option agreement for rights to develop and commercialize novel AAV gene therapy product candidates utilizing a mutation independent minigene therapy approach for the treatment of vision loss in USH2A-related inherited retinal diseases (IRDs). IVERIC bio’s collaboration with UMass Medical School and its Horae Gene Therapy Center, utilizing the minigene therapy approach, has resulted in additional research data that supports the Company’s plans to move the program forward. (Nasdaq: ISEE) today announced that the Company has exercised its option and entered into an exclusive global license agreement with the University of Massachusetts Medical School for rights to develop and commercialize mutation independent novel adeno-associated virus (AAV) gene therapy product candidates for the treatment of Leber Congenital Amaurosis type 10 (LCA10) due to mutations to the CEP290 gene, the most common type of LCA. NEW YORK-( BUSINESS WIRE)- IVERIC bio, Inc.
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