pp-255 Injection
Dossier type
US CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Lebers hereditary optic atrophy
Clinical stage: Phase 3
Modality: AAV gene therapy
Mechanism & Target: Adeno-associated virus based gene therapy
Route & form: Subretinal / intravitreal injection
Differentiation: Designed to treat Leber’s Hereditary Optic Neuropathy (LHON) with ND4-mutation.
Manufacturer #39670
A late-stage clinical biopharmaceutical company focused on ophthalmology. Developing a gene therapy therapeutic targeting ophthalmology with global development focus. Pipeline positioned for regulatory advancement in key markets.
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