Date: 2017-10-11
Type of
information: Clinical trial authorisation
phase: 1-2
Announcement: clinical trial authorization
Company: Horama (France)
Product: HORA – PDE6B
Action
mechanism:
- gene therapy. 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 PDE6? 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 PDE6? subunit is expressed) as well as in the cones.
Disease: retinitis pigmentosa due to a mutation in the PDE6B gene
Therapeutic
area: Rare diseases - Genetic diseases - Ophtalmological diseases
Country: France
Trial
details:
- This monocentric , open – label, dose – escalation , Phase I/II study will be performed on 12 patients 18 years old or older, suffering from a retinitis pigmentosa caused by mutations in the PDE6B gene , with no other syndromic manifestations. (NCT03328130)
Latest
news:
- • On October 11, 2017 , Horama, a French biotech company which specialises in gene therapy for the treatment of rare , inherited retinal diseases , announces that it has been authorised 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 the Ophthalmology Clinic of Nantes University Hospital , is to assess the safety and efficacy 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 PDE6? protein (PDE6beta).
Is
general: Yes
