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Neubase Therapeutics

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vista's key points

  • Led by Dietrich A. Stephan, Ph.D. Chairman & CEO, Founder 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.
  • NeuBase is accelerating the genetic revolution using its proprietary Peptide-nucleic acid AnTisense OLigo (PATrOL™) platform.
  • Modular and highly specific, NeuBase's PATrOL platform has the potential to significantly improve upon current gene modulation technologies by combining the specificity of nucleotide engagement with the intracellular penetration and broad organ distribution capabilities of small molecule therapeutics.
  • Initial focusing on neurological & neuromuscular diseases i.e. Huntington's Disease (HD) and Myotonic Dystrophy (DM1).
  • NeuBase recently announced positive in vitro and in vivo preclinical data for its PATrOL™-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1).

vista's key points

  • Led by Dietrich A. Stephan, Ph.D. Chairman & CEO, Founder 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.
  • NeuBase is accelerating the genetic revolution using its proprietary Peptide-nucleic acid AnTisense OLigo (PATrOL™) platform.
  • Modular and highly specific, NeuBase's PATrOL platform has the potential to significantly improve upon current gene modulation technologies by combining the specificity of nucleotide engagement with the intracellular penetration and broad organ distribution capabilities of small molecule therapeutics.
  • Initial focusing on neurological & neuromuscular diseases i.e. Huntington's Disease (HD) and Myotonic Dystrophy (DM1).
  • NeuBase recently announced positive in vitro and in vivo preclinical data for its PATrOL™-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1).

company profile

 

vista's view

As NeuBase states and we agree, the world is at a fundamental inflection point in the history of the pharmaceutical industry where, as a result of the Human Genome Project, we now has the ability to leverage nature’s own information encoding system (the genetic code) to “digitally” encode our drugs...

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