Local company leads the way in gene therapy - Cleveland 19 News Cleveland, OH

Local company leads the way in gene therapy

(Source: WOIO) (Source: WOIO)
CLEVELAND, OH (WOIO) -

Local company Abeona Therapeutics is on the forefront of gene therapy.

In just three years, the Carnegie Avenue start-up has raised over $100 million. They recently went public and are now traded on the Nasdaq Exchange.

Abeona's mission is to give patients with a genetic deficit a functioning copy of the gene, using a virus for delivery.

“We've taken a virus, we've scooped out the bad stuff and put in correct normal copy of the gene so the virus is a delivery vehicle," said Timothy Miller, founder, president and CEO. "It's very Star Trek."

Abeona is currently working on three clinic trials, one of which involves their work on Sanfilippo Syndrome. Sanfilippo Syndrome has profound effects on children's central nervous systems, causing neurocognitive decline, speech loss and loss of mobility. Most kids who suffer from it don't live past 18 years old.

The Abeona trial involves the only virus in gene therapy that can cross into the central nervous system. They are the only bio-tech in the world using a virus that is given in a single intravenous injection, allowing the therapy to target the entire body.

“The amount of virus we're giving, for example, when a person sneezes they release maybe 100,000 particles in the room, you can get infected by that," said Miller. "So to give the amount of virus that we're giving you'd have to sneeze ten million times a day for five days in a row."

Abeona announced that the first high-dose patient was enrolled in the trial on Feb. 1. This announcement follows six months of sustained success in low dose patients.

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