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company profile

 

NeuBase Therapeutics, Inc., a pre-clinical-stage biopharmaceutical company, focuses on the development of therapies to treat rare genetic diseases and cancers caused by mutant genes. The company’s proprietary modular peptide-nucleic acid antisense oligo (PATrOL) platform focuses on addressing Huntington’s disease (HD) and myotonic dystrophy, as well as other genetic disorders and cancer. Its lead programs include NT0100, a PATrOL enabled anti-gene for Huntington’s disease; and NT0200, a PATrOL enabled anti-gene for myotonic dystrophy type 1. NeuBase Therapeutics, Inc. is headquartered in Pittsburgh, Pennsylvania.

Sector
:
Healthcare
Industry
:
Biotechnology
Exchange
:
Nasdaq
Ticker
:
NBSE

vista's key points

  • Neubase is led by Chairman & CEO, Founder Dietrich A. Stephan, Ph.D. an industry veteran having had a career in academia and in industry. Stephan served as Deputy Director for Discovery Research and chair of the neurogenomics division at TGen, and served as professor and chairman of the Department of Human Genetics at the University of Pittsburgh/UPMC.
  • NeuBase is advancing an entirely new class of medicines to address many currently undruggable diseases.
  • NeuBase's ultra-precision genetic medicines have demonstrated in proof-of-concept preclinical studies treatment tolerability, selective engagement of different disease-driving mutations at the DNA or RNA level, and broad tissue distribution after systemic administration.
  • NeuBase has validated their ability to Drug the Genome™ to address both rare and common diseases without the limitations of early precision genetic medicine technologies.
  • NueBase’s delivery shuttle enables pharmacology across programs in multiple tissues, including in the brain and muscle, after subcutaneous administration.
  • As of September 30, 2021, the Company had cash and cash equivalents of approximately $52.9 million, compared with approximately $32.0 million as of September 30, 2020. NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the first quarter of CY2023.