HORAMA Signs Exclusive License Agreement with Leiden University Medical Center Targeting CRB1 Gene Mutations to Treat Inherited Retinal Dystrophies

Further expansion of HORAMA gene therapy portfolio to treat Inherited Retinal Dystrophies to lead to second clinical program by 2023

Paris (France) and Leiden (Netherlands), March 18, 2020 – HORAMA SA, a French biotechnology company focusing on gene therapy for the treatment of rare genetic diseases in ophthalmology, announced today an exclusive licensing agreement with the Leiden University Medical Center (LUMC) for global rights to a gene therapy program to treat the Inherited Retinal Dystrophy associated with pathogenic CRB1 gene mutations, a rare but devastating ophthalmic condition leading to blindness

We are excited to enter into this agreement with the LUMC, a leading academic institution with highly recognised scientific leaders in the field of gene therapy such as Jan Wijnholds, to expand our leadership in gene therapy. This collaboration enables us to expand our pipeline of gene therapy treatments for ophthalmic conditions for which there is a high unmet medical need”, commented Christine Placet, CEO of HORAMA.

Our studies in the last 20 years resulted in the development of a platform for candidate gene therapy medicines for children with pathogenic CRB1 mutations. The main obstacle to test our novel innovative medicine gene therapy products in clinical studies was the high costs of the clinical development phase. We are, therefore, excited about this research agreement with HORAMA team, which is a global expert in this field”, commented Jan Wijnholds, LUMC.

Under the agreement, HORAMA will receive an exclusive worldwide license to certain patent rights and know-how for the drug candidate (referenced as HORA-001). In return for these rights, LUMC will receive an undisclosed upfront payment, milestone payments and royalties on net sales of products. HORAMA shall be responsible to bring the gene therapy to market with completion of the non-clinical and clinical studies. Based on current timelines, and subject to regulatory review, HORAMA expects initiating a Phase I/II clinical study with HORA-001 in 2023.

Per the agreement, the parties have entered into a non-clinical development agreement with Leiden University Medical Center (LUMC), led by Dr. Jan Wijnholds, Team Leader and permanent staff member at the LUMC Department of Ophthalmology.

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