NeuBase lays off 60% of employees as it swaps out antisense focus for gene editing tech endorsed by George Church
17 Oct 2022
OligonucleotideExecutive ChangeAcquisition
Geneticist and serial entrepreneur Dietrich Stephan had hoped that his antisense oligonucleotide platform at NeuBase Therapeutics could expand the field that Ionis pioneered to new areas. But in the end, he concluded now is not the right time to explore it.
NeuBase has decided to shift its whole focus to gene editing, deferring preclinical activities for antisense programs targeting myotonic dystrophy type 1, Huntington’s disease and KRAS-mutant cancers.
Preview
Source: Endpts
Sandra Rojas-Caro
As a result, the biotech is letting 60% of its staffers go. Sandra Rojas-Caro, head of R&D and chief medical officer, put in her resignation.
The antisense tech, dubbed PATrOL, promised to overcome limitations of previous gene silencing techniques and create genetic medicines that can be delivered to more types of tissues, are more tolerable and selective, and also relatively cheap to manufacture.
“While we continue to believe PATrOL has the ability to deliver best-in-class treatments for rare and common diseases, we have decided to reprioritize Company resources by limiting future investment in our DM1, HD, and KRAS programs and pursue collaborative initiatives, including partnerships, for these programs,” Stephan said in a statement. “We believe our differentiated gene editing capabilities will allow us to develop the next generation of therapies to address various high-value genetic mutations.”
He cited “current development timelines for the pipeline and the tightening of the capital markets for small biotech companies over the past year” as the reason to pivot, a decision that the board believes is best for shareholder value.
Preview
Source: Endpts
Founded in 2019, NeuBase made its way to Nasdaq the same year through a reverse merger with struggling eye drug developer Ohr Pharma. It also broadened its suite of tools by buying scaffold technologies from Vera Therapeutics, formerly TruCode Gene Repair.
Despite a period of going up, though, shares $NBSE have been plumetting and are now trading at $0.28.
Harvard’s George Church, who was recruited to chair NeuBase’s gene editing advisory board, offered his thumbs-up for the tech as an “ideal” solution for in vivo editing applications:
This technology potentially allows for extremely precise targeting of a mutation in the genome, which is likely to reduce or eliminate off-target edits, and it works through the recruitment of high fidelity human DNA repair enzymes to correct a mutation as opposed to the use of bacterial-derived CRISPR/Cas enzymes. There is likely low toxicity due to a lack of double-stranded breaks and a low immunogenicity profile, with the potential for repeat dosing to compensate for tissue turnover and requisite editing efficiencies for clinical benefit.
After accounting for $0.5 million in restructuring costs, NeuBase says it now has a runway into the second quarter of 2024 to test out its new strategy.
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