Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy
Latest Information Update: 28 Jan 2022
At a glance
- Drugs Esonadogene imvoparvovec (Primary)
- Indications Leber's hereditary optic atrophy
- Focus Therapeutic Use
- 25 Jan 2022 According to a Neurophth Therapeutics media release, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) granted the orphan drug designation (ODD) for NR082 for the treatment of Leber's hereditary optic neuropathy (LHON) associated with mtND4 mutation, based on compelling IND-enabling data and clinical data of 186 subjects from three investigator-initiated trials (IITs).
- 24 Sep 2020 According to a Neurophth Therapeutics media release,at the 23rd online annual meeting of the American Society for Gene and Cell Therapy (ASGCT) and the online annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), Neurophth presented (in May 2020) the two clinical research data (NCT01267422 and NCT03153293) on the treatment of LHON with NR082 (NFS-01 project of rAAV2-ND4).
- 24 Sep 2020 According to a Neurophth Therapeutics media release, longest follow-up record of gene therapy in the world from this study, has been published in the Scientific Report, EBioMedicine and Ophthalmology journals.