Exhibit 99.1
NeuBase Reports Business Update and Financial Results for the First Quarter of 2023
Announced oral presentations at the American Society of Gene & Cell Therapy (“ASGCT”) 26th Annual Meeting to take place on May 16-20, 2023
Company to present additional data showcasing gene editing capabilities at scientific conferences throughout remainder of 2023
PITTSBURGH, May 11, 2023 -- 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 reported its financial results for the three-month period ended March 31, 2023 and other recent developments.
“To date in 2023, we have made steady progress advancing the development of our gene editing application based on our PATrOL™ platform. One of the main drivers behind the initial success of this application area, and speed at which we are moving forward, is the ability to leverage our extensive experience in peptide nucleic acids (“PNAs”), the chemistry which forms the core of our platform. PNAs have been shown to engage the double-stranded human genome due to their high binding affinity and exquisite sequence selectivity. This chemistry would allow us to tag a locus in the genome that harbors a mutation with a simple synthetic compound and recruit the cell’s own machinery to repair the mutation. This is the approach behind our in vivo gene editing platform,” stated Dietrich A. Stephan, Ph.D., Founder and Chief Executive Officer of NeuBase. “We believe our Stealth Editors™ can potentially address up to 90% of disease-causing mutations, compared to the estimated approximately 20% addressed by base editing platforms currently in development. In addition, our approach to gene editing offers several benefits that differentiate us from CRISPR/Cas editors, base editors, and prime editors. For example, Stealth Editors™ are non-immunogenic and have exquisite fidelity, maximizing the ability to re-dose should durability wane over time, which is a situation we have seen with gene therapy approaches in the past.”
“Throughout the remainder of the year we look forward to presenting initial ex vivo and in vivo editing results against high-value genetic mutations, together with associated performance metrics, such as fidelity and efficiency. This includes preclinical data in two oral presentations the NeuBase team will provide at the ASGCT 26th Annual Meeting taking place next week. We plan to follow up the presentations with an investor and analyst conference call on May 22, 2023 to discuss these data,” concluded Dr. Stephan.
First Quarter of 2023 and Recent Operating Highlights
| o | The Company is advancing development of the differentiated gene editing capabilities of its PATrOL™ platform and development of Stealth Editors™. |