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

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.

At HORAMA, we believe in gene therapy to treat a broad range of inherited disorders. Our focus is on Inherited Retinal Dystrophies with our lead clinical program targeting patients with PDE6B gene mutations, a condition which leads to progressive vision loss in children and adults ultimately leading to legal blindness.
Our team is pushing the boundaries of gene therapy by advancing next generation delivery platforms that will improve effectiveness and coverage of gene transfer to address multiple diseases. For more information, please go to: www.horama.fr.

Gene therapy market (source: FiorMarkets and Grand View Research, Inc)
Gene therapy is being developed with an aim to treat rare conditions with limited or no treatment options. Genetic disorders occur due to gene mutations, which can result in incorrect protein synthesis. Gene therapy is used to introduce a healthy gene into cells to allow the synthesis of a functional protein. Growing awareness and acceptance of gene therapy for various disease treatments are favouring market growth. The global gene therapy market is estimated to reach $5.5 billion by 2026, while the global ophthalmology market is projected to grow to $43 billion by 2026 (April 2019 report issued by Grand View Research, Inc.).

Inherited Retinal Dystrophies
Inherited Retinal Dystrophies (IRD) represent a diverse group of progressive visually debilitating diseases that can lead to blindness. In patients with an IRD, mutations in genes, which are critical to retinal function, lead to progressive, direct or indirect photoreceptor cell death and associated visual function losses. IRDs are a genetically heterogeneous group of diseases, with over 260 genes identified to date, IRDs associated with pathogenic CRB1 gene mutations are among this heterogeneous group, similar to the autosomal recessive
IRD associated with pathogenic PDE6B gene mutations.

About CRB1
CRB1 gene mutations are a major cause of early onset and delayed onset IRD. Proteins such as CRB1 and CRB2 are essential in the retina to maintain adhesion between photoreceptors and Müller glial cells. Loss of CRB function results in loss of photoreceptors and causes blindness.