Gene Therapy

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, 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 by the U.S. Food and Drug Administration (FDA).... Read more about Gene Therapy Platform Developed by Neena Haider, PhD, Granted FDA Orphan Drug Designation

Luk Vandenberghe, PhD, Builds Designer Viruses for Improved Gene Therapy

December 19, 2018

Luk Vandenberghe, PhD

In Nature, Luk Vandenberghe, PhD, Harvard Medical School Associate Professor of Ophthalmology and Director of the Grousbeck Gene Therapy Center at Mass. Eye and Ear, discusses increasing the efficiency of gene therapies by manipulating the viral vectors that transport genes into cells. He also talks about taking a nonprofit approach to tackle the economics of developing gene therapy for rare diseases.... Read more about Luk Vandenberghe, PhD, Builds Designer Viruses for Improved Gene Therapy

Scientists, Advocates Team Up to Launch Odylia Therapeutics to Accelerate Gene Therapy Trials for Rare Conditions Causing Blindness

March 7, 2018

New nonprofit organization will bring treatments to clinical trial for people at risk for blindness due to ultra-rare inherited retinal diseases.

Luk Vandenberghe in laboratory

Boston, Mass. — Scientists and advocates today announced the formation of ...

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Luk Vandenberghe

Selecta Biosciences obtains exclusive license to proprietary gene therapy vector from Massachusetts Eye and Ear

May 19, 2016

Selecta is licensing Anc80 from Massachusetts Eye and Ear for a rare genetic disease with options on additional pre-specified indications. By combining Anc80 with Selecta’s investigational drug candidate, SVP-Rapamycin (SEL-110), Selecta is seeking to advance a new gene therapy platform designed to avoid several immunogenicity challenges that limit the development of gene therapies today 

Watertown, Mass. — Selecta Biosciences, Inc., a clinical-stage biopharmaceutical...

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