Paris, July 11, 2018 – Horama, a French biotech company based in Paris and Nantes, which specialises in gene therapy for the treatment of rare, inherited retinal diseases, announces today that the company initiated its Phase I/II clinical trial for HORA-PDE6B last November and enrolled its first patients. This study, conducted within the Ophthalmology Clinic of Nantes University Hospital (France), aims to evaluate the safety and efficacy of HORA-PDE6B in the treatment of retinitis pigmentosa caused by mutations in the PDE6B gene.
This retinitis pigmentosa is linked to biallelic mutations of the PDE6B gene leading to a lack of expression of the PDE6β protein. The subsequent pathology causes gradual and irreversible blindness.
It is one of the most common retinal dystrophies but is currently untreatable.
The HORA-PDE6B drug candidate replaces the defective gene with a non-mutated copy of the PDE6B gene, to express a functional PDE6β protein. HORA-PDE6B is administered unilaterally in the subretinal space.
The Phase I/II trial, which was authorized by the French National Agency for Medicines and Health Products Safety (ANSM) in October 2017, is an open-label, monocentric and dose-escalation study. It will include 12 volunteer patients over 18 years old with PDE6B retinitis pigmentosa due to a mutation in the PDE6B gene without any other syndromic manifestations. These 12 patients will be divided into 3 successive cohorts integrating 3, 3 and 6 patients, respectively.
“The initiation of our Phase I/II clinical trial with HORA-PDE6B for the treatment of retinitis pigmentosa is a major milestone for Horama. We look forward to the opportunity of providing patients with a potential treatment and responding to a medical need for which there is, to date, no response “, commented Christine Placet, CEO of Horama.
Retinitis pigmentosa (RP) is an inherited retinal dystrophy characterised by progressive loss of photoreceptors and/or dysfunction of retinal pigment epithelium, associated with pigment deposits visible in the fundus.
Children with RP develop night blindness. Subsequently, their peripheral vision gradually decreases. By the age of 40, patients experience central vision loss. Among the various cases of RP, PDE6B gene mutation is one of the most common genetic forms.
HORA-PDE6B is a recombinant adeno-associated viral (rAAV) vector developed for the treatment of retinitis pigmentosa due to a mutation in the PDE6B gene. This gene replacement therapy provides an unmutated copy of the human PDE6B gene to replace the defective gene, in order to induce the expression of a functional PDE6β protein in the rod outer segment. HORA-PDE6B is administered as a sterile suspension of viral particles, injected directly into the subretinal space. This triggers the expression of the transgene in the rods (where the PDE6β subunit is expressed) as well as in the cones.
Horama is a clinical-stage biotech company that develops gene-therapy treatments, based on recombinant adeno-associated virus (rAAV) vectors, targeting rare inherited retinal diseases. Horama was founded in 2014 by a team of academic researchers who conducted, in 2011, one of the first clinical trials of gene therapy applied to ophthalmology in France.
Caroline Carmagnol / Aurore Gangloff / Elise Guyot
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