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RP2 and RPGR Vectors For Treating X-linked Retinitis Pigmentosa

20:00 EDT 31 May 2018 | NIH

X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor dysfunction primarily caused by mutations in RPGR or RP2 gene.

This technology is poised to advance RPGR or RP2 gene therapy to clinical stage using AAV8 or AAV9 vector carrying human full-length RPGR or RP2-coding sequence.  The investigators have performed a wide dose range study over 18-months and found it to preserve rod and/or cone function as evidenced by ERG and/or OCT, optomotor tests. Morphologically, the treatment preserved rod and cone viability, and corrected mistrafficking of cone opsin and/or rhodopsin. The therapeutic effect was also achieved in advanced disease stage. The broad treatment window and long-lasting therapeutic effects make the RPGR and RP2 gene therapy attractive for clinical development.

This technology is available for licensing, or the NEI research team will entertain potential collaborations that will advance this technology through the remaining preclinical stage toward IND development under a CRADA or a license combined with a CRADA project.

IC: 
NEI
NIH Ref. No.: 
E-050-2015
Advantages: 
  • Pre-clinical dose efficacy study done 
  • RPGR and RP2 available in both AAV-8 and AAV-9 vectors. 
  • Preclinical data to show that treatment at advanced age also shows remarkable preservation of retinal structure and function.
Applications: 
  • Use in gene therapy to prevent or cure XLRP
  • Preserving cone and/or rod function, restoring ERG and protein in the retina, increasing photoreceptor numbers, decrease in retinal detachments 
  • Improving quality of life, visual acuity, ability to drive and independent living
Development Status: 

Pre-clinical (in vivo)

Updated On: 
Jun 1, 2018
Provider Classifications: 
Date Published: 
Friday, June 1, 2018
Publications: 
Patent Application: 
USPTO 62/131,661
Licensing Contacts: 
Lead Inventor: 
Inventor IC: 
NEI
NEI
NEI
Inventor Lab URL: 
https://nei.nih.gov/intramural/ocular_gene
https://irp.nih.gov/pi/tiansen-li
https://irp.nih.gov/pi/anand-swaroop
LPM FIrst Name: 
John
LPM Last Name: 
Hewes
Inv Is lead: 
LPM Phone: 
240-276-5515
LPM Suffix: 
Ph.D.
LPM Organization: 
NCI - National Cancer Institute
DTDT Classification: 
Therapeutics
DTDT Description: 
Therapeutics
Pat Filing Date: 
2015-03-11
Publication Link: 
https://www.ncbi.nlm.nih.gov/pubmed/23745007
https://www.ncbi.nlm.nih.gov/pubmed/25422369
https://www.ncbi.nlm.nih.gov/pubmed/26358772
https://www.ncbi.nlm.nih.gov/pubmed/25877300
Publication Caption: 
PMID: 23745007
PMID: 25422369
PMID: 26358772
PMID: 25877300
Publication Title: 

Li L, et al. Ablation of the X-linked retinitis pigmentosa 2 (Rp2) gene in mice results in opsin mislocalization and photoreceptor degeneration 

Zhang H, et al. Mistrafficking of prenylated proteins causes retinitis pigmentosa 2. 

Mookherjee S, et al. Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy

Wu Z, et al. A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration

Collaboration Sought: 
Yes
Institute or Center: 
Collaboration Opportunity: 

Licensing and research collaboration

Related Invention: 
E-164-2014
E-162-2016
E Number Only: 
E-050-2015
Inventor First Name: 
Zhijian
Tiansen
Anand
Inventor Last Name: 
Wu
Li
Swaroop

Original Article: RP2 and RPGR Vectors For Treating X-linked Retinitis Pigmentosa

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