Rare Daily Staff

Iveric Bio said it exercised its option for an exclusive global license 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 rare inherited retinal disease Leber Congenital Amaurosis.

The gene therapy is for the most common type of Leber congenetic amaurosis type 10 due to mutations to the CEP290 gene. Mutations in CEP290 lead to severe bilateral vision loss in early childhood, incapacitating affected individuals.

Iveric Bio is also expanding its gene therapy portfolio through 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).

USH2A-related IRDs are 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.

“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,” said Glenn Sblendorio, CEO and president of Iveric Bio.

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 biotech’s plans to move the program forward. 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.

“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,” said Kourous Rezaei, chief medical officer of Iveric.

Photo: Glenn Sblendorio, CEO and president of Iveric Bio

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