A vector from AAVL's directed evolution platform appears effective, amenable to intravitreal delivery based on NHP data, and less immunogenic than AAV2. In line with prior disclosure, Phase IIa baseline characteristics for AAVL's AVA-101 are suggestive of a broad wet AMD population (less severe than Phase I)
AAVL's directed evolution platform features potential intravitreal delivery. A poster presented by AAVL showcased an AAV variant developed in house, called AAV2.5T/7m8. This entails 1) portions of modified AAV2 type (designed through the targeted evolution technology) to target retinal cells and 2) portions of AAV5 (a type less prone to immunogenicity). Preclinical data in non-human primates (intravitreal delivery) revealed superior ability for this "designer vector" to infect retina eye cells. Furthermore, AAV2.5T/7m8 elicited a lesser immune response compared with AAV2. We believe this presentation showcases AAVL's broad capabilities and know-how to develop novel AAV vectors, for more convenient delivery (such as intravitreal in eye disorders). AVA-101 Phase IIa patient characteristics are representative of a milder wet AMD population versus the Phase I trial. A new detail is that all patients were negative for the AAV capsid protein (suggestive of effective delivery of the gene therapy). A poster presentation showcased blinded patient demographics from the Phase IIa study of AVA-101 in wet AMD underway at the Lion's Eye Institute (Australia). Per prior disclosure, median baseline best corrected visual acuity of 63 EDTRS letters (previously disclosed at the R&D Day) was suggestive of a milder population compared to patients treated in the Phase I trial (36.5 letters). These data further support our expectation that AVA-101 activity in the Phase IIa study could be more modest compared to the impressive +8.7 and +6.3 letters gained in the Phase I trial, with potentially no letter gain (maintaining visual actuity) but a likely reduction in injection frequency.
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