Akouos IPO Presentation Deck
The High Transduction Efficiency of AAVAnc80 Coupled with Local Delivery to the Inner
Ear Compartment Allows for a Dual Vector Approach for Larger Transgenes
ITR
25
ITA
الے
promoter 5 GFP cDNA
promoter
5' GFP cDNA
SD ITR
ITR SA
Transduction
ITR
t
ITR SA
DNA sequence alignment
Recombination
Transcription
Splicing
polyA TR
3º GFP cDNA H
3* GFP cDNA
GFP mRNA
Translation
polyA ITR
Percentage of Inner Hair Cells Expressing Transgene Threel
Weeks Following Administration of Dual AAVAnc80 Vectors
Inner Hair Cell Transduction (%)
100
80-
60
40-
20-
0-
7.2E11 vg
0.06
Monkey 1
Monkey 2
Monkey 3
Monkey 4
0.5
4
32
Cochlear Frequency Position (kHz)
AKOUOS
Functional protein
Ability to deliver transgenes that are larger than 5 kb in size creates the
potential for broader treatment of genetically-driven hearing loss
CONFIDENTIALView entire presentation