Today, Pittsburgh-based NeuBase Therapeutics, Inc. (Nasdaq: NBSE), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines to drug the genome, announced the execution of a binding agreement to acquire infrastructure, programs and intellectual property for several peptide-nucleic acid (PNA) scaffolds from Vera Therapeutics, formerly known as TruCode Gene Repair, Inc. The technology has demonstrated the ability to resolve disease in genetic models of several human indications. The acquisition was reported to bolster NeuBase’s capabilities and reinforces the Company’s position as a leader in the field of genetic medicine.
“With this acquisition, we enhance our PATrOL™ platform, furthering our unique ability to directly engage and correct malfunctioning genes with exquisite precision to address the root causes of a wide variety of human diseases. These assets extend and refine our PATrOL platform’s capabilities and accelerates, through our Company, to bring the rapidly growing genetic medicines industry toward a single high-impact focal point. We are committed to advancing our pipeline and candidates to the clinic and to exploiting the full potential of PNA technology to continue creating value for our shareholders and importantly, for patients,” stated Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase Therapeutics.
Dr. Stephan is an industry veteran who is considered one of the fathers of the field of precision medicine, having trained with the leadership of the Human Genome Project at the NIH and then going on to lead discovery research at the Translational Genomics Research Institute and serve as professor and chairman of the Department of Human Genetics at the University of Pittsburgh. Stephan has identified the molecular basis of dozens of genetic diseases and published extensively in journals such as Science, the New England Journal of Medicine, Nature Genetics, PNAS and Cell. In parallel, Dr. Stephan has founded or co-founded 14 biotechnology companies and has advised an additional 12 companies. These companies are backed by top-tier investors such as Sequoia Capital, KPCB, Thiel Capital, and Khosla Ventures as well as corporate partners such as Life Technologies, Pfizer, and Mayo Clinic. Dr. Stephan received his Ph.D. from the University of Pittsburgh and his B.S. from Carnegie Mellon University.
Curt Bradshaw, Ph.D., Chief Scientific Officer of NeuBase, added, “By consolidating additional validated technology into our PATrOL platform, we believe NeuBase is positioned to radically transform the landscape of medicine. In vivo activity in a variety of disease indications has been demonstrated with the new scaffolds that we have acquired, and further expands the validated components of our platform to achieve resolution of causality in living systems with target indications such as recently presented in myotonic dystrophy, type 1. In addition to our intellectual property, we believe our in-house expertise in peptide nucleic acids is second to none.”
The transaction is expected to close in the first calendar quarter of 2021. Financial terms were not disclosed.
Key features of NeuBase’s PATrOL-enabled therapies include:
- Modular molecular design
- Biologically and immunologically inert
- Resistant to degradation in the body
- High specificity for target genes while minimizing off-target engagements (OTEs)
- Broad distribution throughout all tissue types
- Ability to use many chemistries for tissue and cell-type specific delivery
- No toxic intramolecular aggregation due to semi-rigid scaffold
- Potential to address dominant and recessive genetic diseases
- Ability to open up and bind to double-stranded nucleic acid targets, including the double-stranded genome
Unlike traditional ASOs which have a sugar scaffold, NeuBase’s PNA therapies are designed with a rigid poly-amide scaffold. The synthetic scaffold enables the PATrOL platform to be truly modular and highly scalable, permitting the rapid development of PATrOL-enabled therapies for many different genetic diseases. The scaffold is first-in-class and its neutral charge, resistance to degradation, lack of immunogenicity and semi-rigid nature collectively confer the ability to produce easy to take, precise and well tolerated therapies.
NeuBase can develop its PATrOL-enabled therapies using multiple different types of nucleic acid bases, including those that are natural or engineered, allowing for increased selectivity and optimization of the therapy for the target disease. NeuBase is the first and only company to successfully create bifacial Janus bases, engineered bases that target double-stranded DNA or RNA by engaging both strands at once.
NeuBase has developed a proprietary chemistry to decorate its candidates, which allows the therapies to be delivered systemically and achieve broad tissue distribution. We also have programs in place that allow more focused delivery to target tissues when that is an advantage for maximizing efficacy.
NeuBase chemists have invented a method that gives NeuBase’s PATrOL-enabled drugs the advantages of small molecule therapies. These PATrOL-enabled drugs have proprietary “linkers” on each end, which enable cooperative binding across the gene target. This self-assembly capability confers the ability to address various mutational types such as large expansions and small single-base mutations.
NeuBase had presented Data throughout 2020 have validated the potential of the PATrOL™ platform to develop highly targeted therapies that increase, decrease or change causal protein function. NeuBase plan to provide updates on their development pipeline, including the myotonic dystrophy type 1 (DM1) and Huntington’s disease (HD) programs, at an R&D day in the first half of CY2021.
NeuBase Therapeutics (NBSE) Stock, Insider and Institutional Ownership
NeuBase has ~141 institutional owners and shareholders that have filed 13D/G or 13F forms with the Securities Exchange Commission (SEC). These institutions hold a total of 8,993,130 shares. NBSE’s largest shareholders include Greenlight Capital Inc (9%), Vanguard Group Inc, BlackRock Inc., Renaissance Technologies Llc, VTSMX – Vanguard Total Stock Market Index Fund Investor Shares, IWM – iShares Russell 2000 ETF, Sio Capital Management, LLC, State Street Corp, VEXMX – Vanguard Extended Market Index Fund Investor Shares, and Geode Capital Management, Llc.
In December, I hosted a FREE Presentation & Q&A Event for our sister organization Tribe Public with the CEO & Founder of NeuBase Therapeutics, Dietrich Stephan, Ph.D., who owns 34.3% of NeuBase’s total shares. He is an industry veteran who is considered one of the fathers of the field of precision medicine. The presentation is titled “Accelerating the Genetic Revolution with a New Class of Synthetic Medicines.” You can view the recording at the Tribe Public Channel.
We expect to see the company to announce an update on its work in Huntington’s Disease and also sign a corporate deal of some kind prior to the end of April 2021 based on recent conversations & expectations of management.