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Iveric bio Advances LCA10 Minigene Programme and Expansion of Orphan Gene Therapy Portfolio with USH2A-Related Inherited Retinal Diseases Programme.

M2 PHARMA-July 24, 2019-Iveric bio Advances LCA10 Minigene Programme and Expansion of Orphan Gene Therapy Portfolio with USH2A-Related Inherited Retinal Diseases Programme

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- US-based biotechnology company Iveric bio, Inc. (NASDAQ: ISEE) 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 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, the company said.

Iveric bio's collaboration with UMass Medical School and its Horae Gene Therapy Center, utilising the minigene therapy approach, has resulted in additional research data that supports the company's plans to move the programme 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 commercialise novel AAV gene therapy product candidates utilising a mutation independent minigene therapy approach for the treatment of vision loss in USH2A-related inherited retinal diseases.

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

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

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.

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.

USH2A-related inherited retinal diseases 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 characterised 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 USH2Amutations.

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.

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Date:Jul 24, 2019
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