Technology
BGTF-027 Our Candidate for BCM
AAV.7m8 Platform
Intravitreal (IVT) Gene Therapy for the Treatment of Inherited Retinal Diseases (IRDs)
IVT Injection
- Utilizes current standard of care route of administration for biologics
- Routine in-office procedure
- IVT delivery enables pan-retinal transduction
- No mechanical disruption of the retina
7m8 adeno-associated virus (AAV.7m8) capsid1-4
- Engineered variant of AAV2 with a 10-amino-acid (LALGETTRPA) loop insertion
- Modification allows the vector to bypass the inner limiting membrane (ILM) to efficiently transduce and deliver transgenes to target retinal cells
Single IVT administration of BGTF-027 (Formerly ADVM-062) effectively transduces gerbil cone photoreceptors and produces de novo response to long wavelength light
ADVM-062 is expected to expand retinal sensitivity of dichromatic gerbil to red light
ERG responses to red light stimulus (660 nm) at increasing light intensities
Localization of human L-opsin in gerbil cones detected using ADVM-062.myc
Use Of BGTF-027 (Formerly ADVM-062) myc In NHPs To Enumerate Transduced Foveal Cones
IVT -administered BGTF-027 Drives Expression Of Human L-opsin mRNA In A Cone-specific Manner In NHP Retinas
- ISH detected mRNA in NHP eye dosed with IVT-delivered ADVM-062 at 2.1E10 vg/eye
- Vector DNA signal has been reduced by DNAse treatment
- Red: ADVM-062 mRNA
- Purple: Nuclei
