EX-99.1 2 ocugenincpresentation.htm EX-99.1 ocugenincpresentation

 

 

 

 
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Multifactorial Aging A B C

 
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• • • • OCU400 (AAV encoding NR2E3) Rho-/- RhoP23H+/- Rd7 (NR2E3) Rd1 (PDE6B) Rd16 (CEP290) Adapted from: § Li et al., Nature Gene Therapy, 2021 § McNamee et al., Nature Gene Therapy, 2024 Fundus Imaging Improvement in Retinal Structure OCU400 Untreated Treated Histology Slow/Stop Retina Degeneration OCU400 Untreated Treated Biomarkers: Molecular reset Restoration of Expression of Key Transcription Factors Immunofluorescence (IF) Restoration of Phototransduction Gene Expression OCU400 Untreated Treated RP LCA Preclinical Animal Models: Electroretinography (ERG) Improvement in Photoreceptor Function

 
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-10 -8 -6 -4 -2 0 2 4 6 8 Screening M3 M6 M9 M12 002-007 (treated) 002-007 (untreated) 010-001 (treated) 010-001 (untreated) 003-010 (treated) 003-010 (untreated) ∆ ∆ • • • •

 

 
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0.96 ± 0.25 0.76 ± 0.53 Inset • • •

 

 
-55 -50 -45 -40 -35 -30 -25 -20 -15 -10 -5 0 5 10 SCREENING 3 MONTHS 6 MONTHS LL VA c ha ng e (∆ )f ro m b as el in e Study Duration (Months) 006-001 Treated 006-001 Untreated 007-001 Treated 007-001 Untreated 006-004 Treated 006-004 Untreated ±

 
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M e a n C h a n g e in T o ta l A tr o p h y A re a f ro m B a s el in e (m m 2 ) 84% 0.12 ± 0.36 0.73 ± 0.26

 
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Severe RP only ( blind) 85%

 
% C3 *

 
2023