Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

a technology of polynucleotide and retinal cone, which is applied in the field ofviral-based gene therapy of retinal disorders, can solve problems such as the risk of creating additional damage to retinal tissu

Pending Publication Date: 2021-12-16
UNIV OF WASHINGTON +1
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The patent is about a way to treat eye conditions caused by problems with cone photoreceptors (the cells that help with color and vision) by injecting a special virus carrying a therapeutic gene into the eye. This method has shown promise in animals with experimentally induced cone dysfunction or death.

Problems solved by technology

However, these vectors must be administered by subretinal injection, a procedure that disrupts the structure of the retina and carries with it a risk of creating additional damage to retinal tissue that is often already damaged by the disorder being treated.

Method used

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  • Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
  • Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
  • Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

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Background

[0174]New therapies are needed for the treatment of many cone photoreceptor associated disorders, including macular dystrophies such as cone-rod dystrophy, cone dystrophy, Stargardt macular dystrophy, and achromatopsia; color vision disorders such as protan, deutan, and tritan defects; and vision disorders of the central macula such as age-related macular degeneration, macular telangiectasia, retinitis pigmentosa, diabetic retinopathy, retinal vein occlusions, glaucoma, Sorsby's fundus dystrophy, adult vitelliform macular dystrophy, Best's disease, and X-linked retinoschisis. As these vision disorders are associated with a loss of function and / or viability of the cone photoreceptors, it is hypothesized that these disorders may be treatable by delivering a therapeutic gene to cone photoreceptors to rescue cone viability and function.

[0175]To that end, the polynucleotide cassette “pMNTC” was designed in which enhancer, promoter, 5′UTR, intron, Kozak, and polyadenylation sequ...

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Abstract

Methods and compositions are provided for intravitreally delivering a polynucleotide to cone photoreceptors. Aspects of the methods include injecting a recombinant adeno-associated virus comprising a polynucleotide of interest into the vitreous of the eye. These methods and compositions find particular use in treating ocular disorders associated with cone dysfunction and / or death.

Description

CROSS REFERENCE TO RELATED APPLICATIONS[0001]This application is a continuation application of U.S. patent application Ser. No. 15 / 554,664, filed Aug. 30, 2017, which is a U.S. National Phase Application of International Patent Application No. PCT / US2016 / 020482, filed Mar. 2, 2016; which claims the benefit under 35 U.S.C. § 119(e) of U.S. Provisional Application No. 62 / 127,194, filed on Mar. 2, 2015, and U.S. Provisional Application No. 62 / 134,466, filed on Mar. 17, 2015; each of which is incorporated by reference herein in its entirety.STATEMENT REGARDING SEQUENCE LISTING[0002]The Sequence Listing associated with this application is provided in text format in lieu of a paper copy, and is hereby incorporated by reference into the specification. The name of the text file containing the Sequence Listing is AVBI_006_04 US_ST25.txt. The text file is 74 KB, was created on May 5, 2021, and is being submitted electronically via EFS-Web.FIELD OF THE INVENTION[0003]This invention pertains to...

Claims

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Application Information

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Patent Type & Authority Applications(United States)
IPC IPC(8): C07K14/005A61K48/00C12N15/86C12N15/861
CPCC07K14/005A61K48/0066A61K48/0075C12N7/00C12N15/861C12N2750/14122C12N2750/14143C12N15/86A61P27/02C12N2750/14121
Inventor CHALBERG, JR., THOMAS W.NEITZ, JAYNEITZ, MAUREEN
Owner UNIV OF WASHINGTON
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