Paris , October 11, 2017 – Horama, a French biotech co mpany based in Paris and Nantes, which specialises in gene therapy for the treatment of rare , inherited retinal diseases , announces today that it has been authori s ed by the French National Agency for Medicines and Health Products Safety (ANSM) to launch a Phase I/II clinical trial for HORA – PDE6B, in the treatment of a retinitis pigmentosa. The main objective of this clinical study, which will be performed in th e Ophtha lmol ogy Clinic of Nantes U niversity H ospital , is to assess the safety and effic a cy of a unilateral subretinal injection of the product in patients with a retinitis pigmentosa caused by biallelic mutations in the PDE6B gene, which lead to a defective expres sion of the PDE6β protein (PDE6beta).
This monocentric , open – label, dose – esca lation , Phase I/II study will be performed o n 12 patients 18 years old or older, suffering from a retinitis pigmentosa caused by mutations in the PDE6B gene , with no other syndromic manifestations.
“ Being granted an authori s ation by the ANSM to proceed with our clinical study is an important milestone for Horama. We are pleased to initiate this Phase I/II trial to confirm the safety of HORA – PDE6B , administered via unilateral subretinal injection, for the treatment of this form of retinitis pigmentosa. This pathology, one of the most widespread among retinal dystrophies , still has no treatment” , commented Christine Placet, CEO of Horama.
Retinitis pigmentosa (RP) is an inherited retinal dystrophy characteri s ed by progressive loss of photoreceptors and/or dysfunction of retinal pigment epithelium, associated with pigment deposits visi ble in the fundus. Children with RP develop night blindness. Subsequently , the ir peripheral vision gradually decreases. By the age of 40, patients experience central vision loss . Among the various cases of RP, PDE6B 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 a s a sterile suspension of viral particles, injec ted 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 treatment s, 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 f irst clinical trials of gene therapy applied to ophthalmology in France , including Philippe Moullier, Christian Hamel (1955 – 2017) and Vasiliki Kalatzis , from the French National Institute of Health and Medical Research ( INSERM ) and Michel Weber and Guylène Le Meur, from the Nantes U niversity H ospital in France .