NeuBase Announces Formation of Gene Editing Advisory Board

PITTSBURGH, May 16, 2023 (GLOBE NEWSWIRE) — NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company developing Stealth Editors™ to perform in vivo gene editing without triggering the immune system, today announced the formation of a gene editing advisory board comprised of distinguished leaders of the scientific community who will support the development of the differentiated gene editing capabilities of the Company’s PATrOL™ platform. In addition, the Company announced the appointments of Jeffrey Kindler, J.D. and John Maraganore, Ph.D. as strategy and business advisors to the Company.

“We are honored to bring together this distinguished and talented group of gene editing advisors, with whom we will meet on a regular basis, to support our development of next generation therapies capable of addressing various high-value genetic mutations,” stated Dietrich A. Stephan, Ph.D., Founder and Chief Executive Officer of NeuBase. “We continue to advance the development of our gene editing technology and plan on identifying initial programs targeting prevalent mutations that cannot be edited by base editors. Our technology leverages the differentiated gene editing capabilities of our PATrOL™ platform, which promises the unique ability to achieve precise repair of a diverse set of mutations in the genome without the need to deliver nucleases, such as modified bacterial CRISPR/Cas enzymes. We look forward to several data announcements supporting the development of our gene editing platform at various scientific conferences throughout the remainder of 2023, including oral presentations scheduled to take place at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting taking place on May 16-20, 2023.”

The members of the gene editing advisory board include:

George Church, Ph.D. (Chairperson)

Professor Church is a pioneer of genome engineering, DNA sequencing and synthetic biology. He brings significant expertise both in genetics and the biotechnology industry to the NeuBase gene editing advisory board. He has co-founded 24 biotechnology companies, authored over 500 papers and 140 patent publications. George Church is a professor of genetics and professor of health sciences and technology at Massachusetts Institute of Technology. He is the director of the Center for Genomically Engineered Organs (CGEO) and the Lipper Center for Computational Genetics. Professor Church is a member of the National Academy of Sciences (2011) and the National Academy of Engineering (2012), and he has received the Franklin Institute’s Bower Award for Achievement in Science (2011). He holds a Ph.D. in biochemistry and molecular biology.

James Coull, Ph.D.

Dr. Coull has 35 years of experience as a successful entrepreneur and builder of life science companies. He began his career at Millipore Corporation where he supervised teams of scientists working on the development of new methods and chemistries for synthesis and analysis of DNA, peptides and proteins. He then co-founded and was Vice President R&D at Boston Probes, Inc., where he spearheaded the development of its core PNA technology resulting in probe-based tests to identify pathogenic micro-organisms, genetic mutations and cancers, and which was acquired by Applied Biosystems. Dr. Coull has served as CTO of Ensemble Discovery, CTO and VP Business Development of AdvanDx, and CEO of PNA Innovations. He is currently a scientific and business consultant to various private and public life science companies. A pioneer of many innovative techniques, Jim is an inventor who is listed on more than 40 issued US patents and has published numerous peer-reviewed scientific articles. He holds a BSc in Chemistry from Colby College (1980) and received his Ph.D. in Biochemistry from Purdue University (1986).

Steven Dowdy, Ph.D.

Dr. Dowdy is a cancer biologist, specializing in the development of RNA therapeutics and in understanding cell cycle controls. He is a professor of cellular and molecular medicine at the University of California San Diego, School of Medicine, where his research focuses on the delivery of RNA therapeutics into cells, including the chemistry of developing endosomal escape technologies. Dr. Dowdy is a past member of the board of directors of the Oligonucleotide Therapeutics Society and consults extensively for biotechnology and pharmaceutical companies.

Peter Nielsen, Ph.D.

Dr. Nielsen is a leading expert in gene targeting, RNA interference and chemical replication and translation and was one of the inventors of PNAs in 1991. He is currently a professor at the University of Copenhagen, where his lab focuses on PNAs in regard to drug discovery, gene targeting, antisense principles, cellular and in vivo delivery, and administration of biopharmaceuticals. He is the co-author of more than 400 scientific papers and reviews, editor of 6 books, inventor on over 20 patents and patent applications, and he serves on the advisory board of four scientific journals, one of which he is section editor. In addition to his esteemed academic career, Dr. Nielsen is the co-founder of two biotechnology companies: PNA Diagnostics A/S, which was acquired by Boehringer Mannheim, and Pantheco A/S, which merged to form Santaris A/S and was subsequently acquired by Roche. Dr. Nielsen is a member of EMBO and the Danish Academy of Technical Sciences. Dr. Nielsen has been recognized for his seminal contributions to science by earning the Novo Nordic Foundation Prize (1997), the Lundbeck Foundation Prize (1997), and the InstitutCurie Jeanne Loubaresse Prize (Paris/France) (2002).

Eriks Rozners, Ph.D.

Dr. Rozners is a leading expert in the chemistry and biochemistry of nucleic acids and brings his expertise to NeuBase as the Company is optimizing and developing its PATrOL™ platform. He is a professor and the chairman of the Department of Chemistry at Binghamton University, where his lab focuses on the use of organic chemistry to develop unique model systems and tools for the studies and practical applications of nucleic acid biochemistry. Dr. Rozners received a bachelor’s degree in chemical engineering and a doctorate in organic chemistry from Riga Technical University.

Jeremy Stark, Ph.D.

Jeremy Stark, Ph.D. received his doctorate at the University of Washington/Fred Hutchinson Cancer Research Center working on RNA splicing, and his postdoctoral training was at Memorial Sloan Kettering Cancer Center with Dr. Maria Jasin, where he began his research on chromosomal break repair and gene editing. He has continued this research focus in his own lab at the City of Hope, where he is now professor of cancer genetics and epigenetics. His research group has developed a series of chromosomal break reporter assays that have been used by several labs around the world. His lab was the first to combine the TREX2 exonuclease with a targeted DSB to cause elevated indel frequencies (Bennardo et al. 2009), leading later to a patent on combining TREX2 and CAS9. Focusing on recent discoveries, his group developed a highly specific assay for canonical non-homologous end joining (C-NHEJ): EJ between two Cas9-induced blunt DSBs without insertion/deletion (indel) mutations (No Indel EJ). This hallmark of C-NHEJ enabled defining key distinctions between this pathway and ALT-EJ, characterizing the function of the C-NHEJ factor XLF (Bhargava et al. 2018), and defining the influence of XLF and DNAPKcs on EJ (Cisneros-Aguirre et al. 2022).

The Company’s recently appointed strategy and business advisors include:

Jeffrey Kindler, J.D.

Jeff Kindler, J.D. is a healthcare executive, investor and advisor who has held leadership positions at numerous globally recognized companies, including Pfizer, McDonald’s and General Electric. With more than three decades of experience, Mr. Kindler is a leading expert in pharmaceuticals and other healthcare sectors, retail, corporate legal matters including regulation, litigation, compliance, and crisis management, brand and franchise management, and executive leadership. Mr. Kindler serves as CEO of Centrexion Corporation, a privately held biotherapeutics company. He is a Senior Advisor for Blackstone, a Venture Partner at Artis Ventures, and a principal in and advisor to numerous privately held healthcare firms and sponsors. Mr. Kindler serves on the boards of several publicly and privately held healthcare companies as well as several not-for-profit institutions.

John Maraganore, Ph.D.

Dr. John Maraganore served as the founding CEO and a Director of Alnylam from 2002 to 2021, where he built and led the company from early platform research on RNA interference (“RNAi”) through global approval and commercialization of the first five RNAi therapeutics, creating a new class of medicines. He continues to serve on the Alnylam Scientific Advisory Board. Prior to Alnylam, he held scientific and business leadership roles at Millennium Pharmaceuticals, Inc., at Biogen, Inc., where he invented and led the discovery and development of ANGIOMAX® (bivalirudin), at ZymoGenetics, Inc., and at the Upjohn Company. Dr. Maraganore is currently an advisor to a number of venture and public company investment firms, and a member of the board of directors of multiple publicly traded companies, including Agios Pharmaceuticals, Beam Therapeutics, Kymera Therapeutics, ProKidney Corp., and Takeda Pharmaceuticals. He is on the board of a number of private companies and non-profit organizations, and also serves as a strategic advisor to other public and private biotechnology companies. Finally, Dr. Maraganore is on the Board of the Biotechnology Innovation Organization, or BIO, where he was Chair from 2017-2019 and is Chair Emeritus. Dr. Maraganore received his B.A., M.S. and Ph.D. in biochemistry and molecular biology at the University of Chicago.

About NeuBase Therapeutics
NeuBase is a pre-clinical stage biopharmaceutical company leveraging its peptide-nucleic acid technology to accelerate the genome editing revolution. NeuBase’s Stealth Editing™ technology is a new type of gene editing designed to avoid being identified by the immune system and provide pronounced effects that are safe, delivered with non-viral technologies, and broadly applicable across different mutation types and industries. This in vivo gene editing system seeks to address disease at the base level by recruiting the body’s own editing machinery to correct mutations that cause disease. The Company projects that its technology can potentially address up to ~90% of all known human mutations, including insertions, deletions, transitions, and transversions with a simple non-immunogenic solution. To learn more, visit www.neubasetherapeutics.com.

Use of Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by the use of words such as “will,” “would,” “anticipate,” “expect,” “believe,” “designed,” “plan,” “project,” or “intend,” the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential and prospects of the Company’s proprietary PATrOL™ platform or Stealth Editing™ technology and the Company’s statements regarding potential collaborations. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission (the “SEC”), may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company’s current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company’s plans to research, develop and commercialize any product candidates; the timing of initiation of any clinical trials; the risk that prior data will not be replicated in future studies; the timing of any investigational new drug application or new drug application; the clinical utility, potential benefits and market acceptance of any product candidates; the Company’s commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company’s ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the SEC. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.