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Adverum Biotech (ADVM) pre-clinical data on 2 gene therapy candidates for the treatment of Wet AMD

September 16, 2016 9:06am

Data shows it might be possible to achieve the same degree of CNV inhibition using an intra-vitreally delivered novel vector with an optimized AAV capsid, expression cassette, and anti-VEGF cDNA as the standard-of-care

Pre-clinical data demonstrated that a single intravitreal administration of ADVM-022 or ADVM-032 each had comparable efficacy in reducing grade IV CNV lesions to an intravitreal injection of standard-of-care anti-VEGF proteins.

To date additional pharmacokinetic studies of the vitreous and retinal tissue have shown durable anti-VEGF protein expression with therapeutic protein levels at least 20 weeks after a single intravitreal administration.

ADVM-022 & ADVM-032 both utilize a vector designed to allow for intravitreal rather than surgical sub-retinal delivery to potentially treat wet AMD as well as other retinal conditions associated with VEGF over-expression.

The Bottom Line: Observations at 26 weeks show a favorable safety profile.

Minimal vitreous inflammation not requiring steroid treatment was noted four to six weeks after injection but was resolved by week eight and did not recur.
No retinal structural changes or consequences of inflammation were detected on optical coherence tomography at 12 weeks after injection.

ADVM closed at $3.76 which was UP +$0.14 – looks like the upside was already built in?

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