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AAV Process Development

Development and optimisation of an upstream manufacturing process for IKAR-001: a bicistronic AAV8 gene therapy for geographic atrophy

IKAR-001 Preclinical Data

Efficacy of a bicistronic AAV8-PEDF/sCD46 gene therapy in a mouse sodium iodate model of GA and in human iPCS-RPE cells.

IKAR-001 NHP Evaluation

Non-human primate evaluation of a dual-pathway gene therapy (IKAR-001) expressing PEDF and anti-complement sCD46 for the treatment of geographic atrophy.

Company Overview

A bicistronic AAV platform for dual-pathway modulation in AMD: Platform development and manufacturing readiness

CD46 as a Complement Modulator

CD46 is a potent complement cofactor as part of a clinical strategy for Geographic Atrophy

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From Science to Sight