IVERIC bio Announces Successful Advancement of LCA10 Minigene Program and Expansion of Orphan Gene Therapy Portfolio with USH2A-Related Inherited Retinal Diseases Program

NEW YORK, NY, USA I July 23, 2019 I IVERIC bio, Inc. (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. 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 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). 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,” stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. “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,” stated Kourous A. Rezaei, MD, Chief Medical Officer of IVERIC bio. “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.

About Minigene Therapy

The use of minigene therapy as a therapeutic strategy seeks to deliver a smaller but functional portion of the larger gene packaged into a standard-size AAV delivery vector commonly used in ocular gene therapy. Research in this evolving area of gene therapy is led by Drs. Hemant Khanna and Guangping Gao in the Horae Gene Therapy Center at UMMS and was described in the 2018 Human Gene Therapy journal article, “Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis” by Wei Zhang, Linjing Li, Qin Su, Guangping Gao, and Hemant Khanna, all of whom are affiliated with the University of Massachusetts Medical School.

About Leber Congenital Amaurosis Type 10

Leber Congenital Amaurosis type 10 (LCA10), the most common type of LCA, is an orphan inherited retinal disease caused by mutations in the CEP290 gene. Mutations in CEP290 lead to severe bilateral vision loss in early childhood, incapacitating affected individuals.

About USH2A-Related Inherited Retinal Diseases

USH2A-related inherited retinal diseases (IRDs) are a group of orphan IRDs associated with mutations to the USH2A gene. The USH2A gene encodes a protein, usherin, which is believed to be important in the development and maintenance of cells in the retina and the inner ear. USH2A-related IRDs include retinitis pigmentosa associated with Usher syndrome type 2A and USH2A-associated autosomal recessive nonsyndromatic retinitis pigmentosa. Usher syndrome type 2a is an autosomal recessive genetic condition characterized by hearing loss from birth and progressive vision loss, due to retinitis pigmentosa, that begins in adolescence or adulthood. USH2A-associated autosomal recessive nonsyndromatic retinitis pigmentosa is a genetic condition that manifests as vision loss without associated hearing loss. The collaborative research program between IVERIC bio and UMMS seeks to develop an AAV deliverable, mutation independent, minigene treatment option for the vision loss associated with USH2A mutations.

About IVERIC bio

IVERIC bio is a biotechnology company with a focus on the discovery and development of novel gene therapy solutions to treat orphan inherited retinal diseases with unmet medical needs. Vision is Our Mission. For more information on the Company’s gene therapy and other programs, please visit www.ivericbio.com.


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