MUMBAI, India, Aug. 10 -- Intellectual Property India has published a patent application (202511066980 A) filed by Indian Institute Of Technology Kanpur on July 14, 2025, for Recombinant Adeno-Associated Virus (aav)-Based Pharmaceutical Compositions With Codon- Optimized Rpe65 And Mcl-1 Transgenes.
Inventors include Jayandharan Giridhara Rao; Rishabh Chandna; and Aheria Dey.
The application for the patent was published on July 31, 2026, under issue no. 31/2026.
Abstract: The present invention relates to recombinant adeno-associated virus (AAV)-based pharmaceutical compositions comprising codon-optimized human RPE65 and codon- optimized human MCL-1 transgenes. The pharmaceutical composition comprises a first recombinant AAV vector comprising a codon-optimized human RPE65 transgene operably linked to a cytomegalovirus (CMV) promoter and packaged in an AAV2K665Q mutant capsid, and a second recombinant AAV vector comprising a codon-optimized human MCL-1 transgene operably linked to a human RPE65 promoter and packaged in an AAV2K105Q mutant capsid. The recombinant AAV vectors are self-complementary AAV vectors formulated in a vector genome ratio of 4:3 for subretinal delivery. The invention further provides recombinant AAV vectors and kits comprising the pharmaceutical composition. Experimental evaluation demonstrated enhanced retinal transgene expression, reduced apoptosis, preservation of retinal morphology, and improved retinal function. FIG. 1
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