Junwei W Sun

US Patent:

20230037283, Feb 2, 2023

Filed:

Nov 10, 2021

Appl. No.:

17/523492

Inventors:

- Philadelphia PA, US
Junwei SUN - Philadelphia PA, US
Vidyullatha VASIREDDY - Wayne PA, US

International Classification:

A61K 48/00
A61K 9/00
A61P 27/02
C12N 15/63
C12Q 1/68
C12N 15/67

Abstract:

Codon optimized nucleic acid sequences for RDH12 are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequence for functional RDH12. Recombinant vectors are provided that express the codon optimized, functional RDH12. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are providing for correcting a non-functional, defective or inadequately expressed native RDH12.

US Patent:

20230009257, Jan 12, 2023

Filed:

Aug 26, 2022

Appl. No.:

17/822533

Inventors:

- Philadelphia PA, US
Junwei Sun - Philadelphia PA, US
Vidyullatha Vasireddy - Wayne PA, US

International Classification:

A61K 48/00
A61P 27/02
A61K 9/00
C12N 15/63
C12N 15/67
C12Q 1/68

Abstract:

Codon optimized nucleic acid sequences for RDH12 are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequence for functional RDH12. Recombinant vectors are provided that express the codon optimized, functional RDH12. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence, deficiency or inappropriate expression of RDH12. Other compositions and methods are providing for correcting a non-functional, defective or inadequately expressed native RDH12.

US Patent:

20210330816, Oct 28, 2021

Filed:

Jul 7, 2021

Appl. No.:

17/369525

Inventors:

- Philadelphia PA, US
Jeannette Bennicelli - Philadelphia PA, US
Junwei Sun - Philadelphia PA, US

International Classification:

A61K 48/00
A61K 9/00
C07K 14/47
C12N 15/86

Abstract:

Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.

US Patent:

20210155938, May 27, 2021

Filed:

Apr 17, 2019

Appl. No.:

17/047496

Inventors:

- Brighton MA, US
- Philadelphia PA, US
Jean BENNETT - Bryn Mawr PA, US
Scott J. DOOLEY - Philadelphia PA, US
Krishna Jawaharlal FISHER - Durham NC, US
Junwei SUN - Philadelphia PA, US

International Classification:

C12N 15/113
C07K 14/705

Abstract:

The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.

US Patent:

20210060176, Mar 4, 2021

Filed:

Nov 16, 2020

Appl. No.:

17/099421

Inventors:

- Philadelphia PA, US
Jean Bennett - Bryn Mawr PA, US
Junwei Sun - Philadelphia PA, US

International Classification:

A61K 48/00
C12N 15/86
C12N 9/02
A61P 27/02
A61K 38/44

Abstract:

Codon optimized nucleic acid sequences for the long form and short form of RdCVF are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequences. Recombinant vectors are provided that express the codon optimized RdCVFL and RdCVF individually, or express two copies of a codon optimized RdCVF or RdCVFL nucleic acid sequence, or both RdCVFL and RdCVF in a single vector or virus. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence or inappropriate expression of RdCVF and RdCVFL.

US Patent:

20200172929, Jun 4, 2020

Filed:

Jun 14, 2018

Appl. No.:

16/621627

Inventors:

- Philadelphia PA, US
Junwei Sun - Philadelphia PA, US
Jeannette Bennicelli - Philadelphia PA, US

International Classification:

C12N 15/86
A61K 48/00
C07K 14/47

Abstract:

Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.

US Patent:

20200093937, Mar 26, 2020

Filed:

May 31, 2018

Appl. No.:

16/615941

Inventors:

- Philadephia PA, US
- Los Angeles CA, US
- Montreal, CA
Jean Bennett - Philadelphia PA, US
Junwei Sun - Philadelphia PA, US
Ji Yun Song - Lower Gwynedd PA, US
Devin McDougald - Philadelphia PA, US

International Classification:

A61K 48/00
A61K 35/761
C12N 9/14
C12N 15/86

Abstract:

Methods and compositions are provided for treatment of peroxisomal biogenesis disorders (PBDs). More particularly, recombinant adeno-associated viruses (rAAV) provided in the form of compositions are used to deliver a nucleic acid encoding human PEX1 to host cells. The rAAVs comprise a AAV capsid, and packaged therein a vector genome comprising an AAV 5′ inverted terminal repeat (ITR) sequence; a promoter; a coding sequence encoding a human PEX1; and an AAV 3′ ITR.

US Patent:

20200061209, Feb 27, 2020

Filed:

Dec 13, 2016

Appl. No.:

16/061530

Inventors:

- Philadelphia PA, US
Jeannette Bennicelli - Philadelphia PA, US
Junwei Sun - Philadelphia PA, US

International Classification:

A61K 48/00
C12N 15/86
C07K 14/47
A61K 9/00

Abstract:

Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.