Newcomer NeuBase Therapeutics is re-engineering antisense oligos to access a broader range of targets, while simultaneously solving the modality’s dosing and delivery issues.
With three approvals since the start of 2016, antisense oligonucleotides (ASOs) have started to hit their stride, yet first-generation technologies are limited on multiple fronts.
Most ASOs only target linear RNA, which means they fail to bind the more complex structures often assumed by mutant, disease-causing RNAs. And ASOs don’t cross the blood-brain barrier, which has meant that Biogen Inc.’s spinal muscular atrophy therapy Spinraza nusinersen must be delivered intrathecally.
NeuBase aims to address those issues, plus several others, with its Peptide-nucleic acid AnTisense OLigonucleotide, or PATrOL, platform.
Founded by serial entrepreneur and CEO Dietrich Stephan, NeuBase Therapeutics Inc. made its trading debut on NASDAQ through a reverse merger on July 18, just 11 months after launching.
Stephan told BioCentury the biotech took advantage of an RNA-hungry market to eschew the more traditional VC financing route, enabling it to hand pick its board without restrictions. It attracted former Global Head of Innovation at Johnson & Johnson and current Vividion Therapeutics Inc. CEO Diego Miralles, as well as Franklyn Prendergast, who served on the board of Eli Lilly and Co. from 1995-2017 (see SideBar: “NeuBase Takes Control”).
On July 30, the company added more big names to its roster when former National Cancer Institute Director Samuel Broder and serial entrepreneur George Church, a professor of genetics at Harvard Medical School, joined its SAB.
A key differentiator of NeuBase’s PATrOL