#  Gene Therapy Platform Developed by Neena Haider, PhD, Granted FDA Orphan Drug Designation  

 





February 27, 2019

 

 

OCU400, a novel gene therapy platform developed by [Neena Haider, PhD](/neenahaider), Harvard Medical School Associate Professor of Ophthalmology and Associate Scientist at the Schepens Eye Research Institute of Mass. Eye and Ear, and licensed to biopharmaceutical company Ocugen, Inc., has been [granted orphan drug designation](https://www.prnewswire.com/news-releases/ocugen-granted-fda-orphan-drug-designation-for-ocu400-gene-therapy-for-the-treatment-of-nr2e3-mutation-associated-retinal-degenerative-disease-300795509.html?tc=eml_cleartime) by the U.S. Food and Drug Administration (FDA).   
The gene therapy has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited degenerative retinal disease. Consisting of a functional copy of the nuclear hormone receptor (NHR) gene NR2E3, OCU400 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, stabilizing cells and potentially rescuing photoreceptor degeneration. Ocugen plans to initiate a Phase 1/2a clinical study for OCU400 by 2020.



 

 

 



 

 See also:- [ Neena Haider, PhD ](/news-categories/neenahaider)
- [ Gene Therapy ](/news-categories/gene-therapy)
- [ Retina ](/news-categories/retina)
- [ Retinal Degenerations ](/news-categories/retinal-degenerations)
 
 

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