Iveric bio Announces Successful Advancement of LCA10 Minigene Program and Expansion of Orphan Gene Therapy

Source: Iveric bio

Iveric bio 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. 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.

The company also announced 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). This is a group of orphan IRDs that includes Usher syndrome type 2A and USH2A-associated autosomal recessive nonsyndromatic retinitis pigmentosa.

“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,” Glenn P. Sblendorio, Chief Executive Officer and President of Iveric bio, said in a company news release. “We are very excited about the progress of our collaboration with world-renowned AAV gene therapy scientists at UMass Medical School, Hemant Khanna, PhD, and Guangping Gao, PhD.”

“The results from our LCA10 minigene program are encouraging for the prospect of using AAV vectors for the treatment of retinal diseases caused by gene mutations that exceed the transgene packaging capacity of conventional AAV vectors,” Kourous A. Rezaei, MD, Chief Medical Officer of Iveric bio, said in the news release. “With the addition of the USH2A program to our portfolio, Iveric bio is seeking to address vision loss associated with what we believe are the most common autosomal dominant form (rhodopsin-mediated) and autosomal recessive form (USH2A-related) of retinitis pigmentosa. Retinitis pigmentosa is the most common orphan inherited retinal disease.”

Iveric bio plans to update investors on all of the company’s gene therapy programs at its upcoming Gene Therapy R&D Investor Day scheduled for September 13, 2019.


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