Baltimore biotech targeting rare diseases raises $4 million

Baltimore biotech targeting rare diseases raises $4 million
<span>A Baltimore biotechnology company has raised $4 million, to help bring its therapeutic treatment targeting rare genetic diseases closer to market.</span>

By   – Reporter, Baltimore Business Journal

A Baltimore biotechnology company has raised $4 million, to help bring its therapeutic treatment targeting rare genetic diseases closer to market.

Elixirgen Therapeutics Inc. filed documents with the U.S. Securities and exchange commission detailing its recent Series A round. The round included one undisclosed investor.

CEO Akihiro Ko said the funding will go toward continued development and clinical trials for Elixirgen's first drug candidate.

Elixirgen's key technology is based on a special protein called ZSCAN4, originally identified in the lab of founder Minoru Ko at the National Institutes of Health. The company is using the protein to create several therapeutic products targeting a range of rare diseases, including bone marrow diseases, aging-related diseases and chromosome diseases.

"The thing about rare disease research is not many people are digging into these kinds of conditions, so people aren’t getting the cures they need," Ko said. His company is aiming to fix that.

It's first drug candidate, EXG 34217, seeks to target inherited bone marrow failure syndromes, or rare disorders known to hinder blood cell production in patients' bone marrow. One such disease, called dyskeratosis congenita, can cause bone marrow failure in up to 80 percent of patients by the age of 30, Ko explained.

Elixirgen had a consultation meeting with the U.S. Food and Drug Administration last year and "got some good feedback," Ko said. The company is finishing up pre-clinical work now, with hopes to begin the first in-human clinical trials for EXG34217 next year.

The company will also continue working on other products in its pipeline, Ko said, including drugs aimed at diseases like Down Syndrome and Alzheimer's.

The company operates out of space in the Johns Hopkins science and technology park in East Baltimore, though it has no association with the University of medical system. It has 15 employees and Ko said the company will continue "reasonable growth," as it moves through clinical trial stages. Ko said the company will also continue seeking new sources of funding for future rounds.

Source: www.bizjournals.com