Drug intelligence / Profile preview

AAV-CFIco

Development stage
Preclinical
Lead developer
Adverum Biotechnologies
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Ophthalmic, Intravitreal
01

Overview

AAV-CFIco is an investigational gene therapy developed by Adverum Biotechnologies for the treatment of geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD). The therapy utilizes a proprietary adeno-associated virus (AAV) vector platform, including the 7m8 and LSV1 capsids, designed for efficient intravitreal delivery to the retina. AAV-CFIco delivers a codon-optimized (co) transgene encoding human complement Factor I (CFI), a key regulatory serine protease that inhibits the alternative complement pathway. By increasing local expression of CFI, the therapy aims to modulate the overactive complement cascade, thereby reducing retinal cell death and slowing the progression of GA.

Other names
AAV7m8 CFI gene therapyAAV-7m8 CFI gene therapyAAV 7m8 CFI gene therapyComplement Factor I gene therapy
02

Targets

CFI (Complement factor I)

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