EPR Podcast 24 – Developing modifier gene therapy – Ocugen

Podcast: Play in new window | Download

Ocugen is utilising the modifier gene therapy technology approach to treat retinal diseases. The therapy can be used to treat both inherited disorders, such as retinitis pigmentosa (RP), Leber congenital amaurosis, and Stargardt disease, but also multifactorial diseases like dry age-related macular degeneration and angiographic atrophy.

The company’s lead candidate in modifier gene therapy, OCU400, is in Phase III trials for treatment of RP and Phase I/II for treatment of Leber congenital amaurosis.

In this episode, Dr Neena Haider, Faculty member at Harvard Medical School and founder of biotech company Shifa Precision and Dr Arun Upadhyay CSO of Ocugen discuss the development of modifier gene therapy.

What is a modifier gene therapy?

Neena starts by discussing the complexity of retinal diseases where multiple genes are involved.

About two to 3,000 genes – so about 10 percent of the genes in the genome – are involved in helping us see, Neena explains. “If there are mutations… in any one of these, it impacts how the retina functions.”

“If we look at retinitis pigmentosa, it’s known as a rare disease – affecting one in 4,000. However, there’s over 200 different roadmaps to get to this disease, meaning either different mutations in known genes or hundreds of different genes,” Neena says.

Modifier genes, which are “normal genes that modulate clinical outcomes by interacting with disease-causing genes,” have been identified and used to suppress or treat or attenuate disease.

Arun and Neena discuss the benefits and challenges of the modifier gene therapy approach, including the manufacturing considerations. 

Future trends in gene therapy 

The speakers also share their predictions for the gene therapy space. They anticipate a trend towards more gene agnostic approaches, combination therapeutics and alternative delivery technologies.

“What I see is the gene therapy approach is going to expand into the non-genetic area, primarily focused on metabolic disorders, neurological disorders, and other chronic disorders,” says Arun.

Neena adds: “We have already started on [an] upward trend. What I predict is that [the gene therapy] space is going to really explode in the most fascinating way … so we’re going to see better, more effective and sustained therapeutics.”