Zandy Forbes’ biotech company MeiraGTx has developed targeted gene therapy methods to treat more than just genetic disorders.

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form ...

Development strategies designed to reduce the unknowns so that cell and gene therapy innovators can develop robust processes ...

Gene mutations in acute myeloid leukemia (AML) cells can guide treatment options, and machine learning can rapidly guess the ...

The joint venture with generative AI firm Hologen hands MeiraGTx $200 million up front as well as other financial perks — a “transformative” deal, according to the company’s CEO.

In a groundbreaking advancement for families grappling with the challenges of Dravet syndrome, a rare and life-altering form of epilepsy, scientists have developed a new gene replacement therapy in ...

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely ...

Because his drug was a gene therapy, public grant money and private ... The CEO, John Carbona, then 54, had run medical device and equipment companies, but he had no background in drug development.

Eleven children who were legally blind at birth all gained visual acuity after receiving MeiraGTx's investigational gene therapy, data that are prompting the biotech to seek accelerated approval ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

“Gene therapy is we collect the cells through stem cell collection, which requires you being placed on a large machine for collection, where you receive mobilized," she said. "This helps push ...