A Pittsburgh biotech startup that’s working to find cures for fatal neurodegenerative diseases has raised $2.25 million in investment capital as the company begins testing compounds that one day could be therapies.
Helexva Inc. has received $2.25 million from UPMC Enterprises, the health care giant’s investment arm, and the nonprofit Focused Ultrasound Foundation in Charlottesville, Va., said Helexva CEO Robert M. Friedlander, who also chairs the Department of Neurological Surgery at the University of Pittsburgh School of Medicine. Huntington’s disease, which is among the diseases Helexva is studying, has been a research focus for Dr. Friedlander, a UPMC neurosurgeon, for more than 25 years.
“This is a very, very powerful technology to cure incurable diseases,” said Dr. Friedlander, a native of Caracas, Venezuela and former Harvard Medical School professor. “At this point, all the wheels are in motion.”
Huntington’s disease affects about 30,000 people in the U.S. with another 200,000 at risk of becoming sick with the disorder. Current treatments can only manage symptoms, which include depression and uncontrolled movement of the arms, legs, face and upper body. There is no cure.
Treatments that Helexva is working on target genetic mutations that cause Huntington’s and at least eight other similar conditions, then inactivate the responsible genes. The compounds being tested as therapies are being made by a contract laboratory, Dr. Friedlander said, which is “fickle chemistry to make.”
Helexva was founded in 2023 and employs five to six part-timers. Helexva has no corporate address and is operated out of Dr. Friedlander’s lab, he said. The company has not generated revenue.
Biotech startups are notoriously cash-hungry and high-risk ventures that often take years to bring a product to market. The Pittsburgh market may make it harder still for entrepreneurs because of a dearth of venture capital in the city.
Helexva, for example, is using technology that was licensed from NeuBase Therapeutics Inc., a publicly held South Oakland outfit whose board voted in January to dissolve the company. A market freeze in investment capital ultimately forced the company out of business, said former CEO Dietrich A. Stephan, who believes the city still has the bones to become a hub for life science startups, much like Silicon Valley or Boston.
“It’s absolutely possible to build sophisticated biotech companies here in Pittsburgh,” said Mr. Stephan, a human geneticist and serial entrepreneur. “All of that’s possible.”
NeuBase was founded in 2009 and went public in 2019 with a $35 million valuation through a reverse merger with Ohr Pharmaceuticals. The company attracted $100 million in investment capital before a big freeze in biotech investment starting in early 2021.
“All the air went out of the biotech market,” he said. “We simply couldn’t raise more capital.”
In late December, NeuBase terminated a licensing agreement with Carnegie Mellon University for technology used in silencing mutated genes. A week later, NeuBase closed its South Oakland office.
Kris B. Mamula: kmamula@post-gazette.com
First Published: March 1, 2024, 8:24 p.m.
Updated: March 2, 2024, 8:28 p.m.
