Gene editing biotech NeuBase Therapeutics is stopping program development and looking for a sale after its board of directors voted to close up shop and seek strategic alternatives.
After a review of the company’s business, the board decided this was the best path forward to maximize shareholder value, according to a Thursday press release. The board will now consider strategic alternatives, which could include an acquisition, merger, business combination or other transaction.
There's no guarantee these efforts will result in a transaction, the company said. Even if NeuBase does strike a deal, there's no guarantee of any benefit to shareholders.
There is no timeline for completion of the evaluation process and NeuBase will not communicate further updates until necessary.
Pittsburgh-based NeuBase set out to develop a technology called Stealth Editors, which conduct in vivo gene editing without triggering the immune system using a non-viral delivery mechanism. The goal was to develop a new wave of gene editing therapies that could potentially be redosed.
The biotech's pipeline included five programs in blood diseases such as sickle cell and beta thalassemia, Huntington's disease and KRAS-mutated cancers. All of its programs were preclinical.
In June, NeuBase showcased preclinical data that suggested that redosing was in fact possible, at least in nonhuman primates. The data showed no innate or acquired immune responses after repeated dosing of the Stealth Editors.
At the time, CEO Dietrich Stephan, Ph.D., noted these adverse events have been well documented with existing gene therapy technology. Such immune responses can cause serious health issues or even death, Stephan pointed out.
"We are beginning to see reports of immune responses after delivery of gene editing solutions, possibly due to viral vector-based delivery or the expression of bacterial nucleases in vivo. For these reasons, we are on a mission to develop the next generation of editors that are non-immunogenic,” Stephan said.
The biotech’s technology aimed to conduct the gene edit by harnessing the cell’s DNA repair mechanism.
But at the same time, NeuBase was fighting to keep the company rolling. A $5 million registered direct offering and private placement executed in June obviously did not go far enough. The funds were to be used for working capital and general corporate purposes.