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PreClinical

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PreClinical Research For ALS

Pre Clinical Research– SPG302, SLS-005, Ketamine, Macimorelin NU-9, RNA Aptamer, CRISPR gene therapy.

Spinogenix SPG-302 :

Currently Cleared For Phase 1
  • Orally administered small molecule
  • Penetrates blood-brain barrier
  • Regenerates lost contacts (synapses) between neurons

Spinogenix SPG-302 :

Ketamine:

Applied for orphan drug status: FDA
  • Approved as a psychedelic anesthetic and for treatment-resistant depression
  • Reduces over-firing of neurons and glutamate toxicity
  • Increases Dopamine
  • Preserves muscle function

Ketamine:

Macimorelin:

Preclinical research
  • Ghrelin is often reduced in ALS patients
  • Macimorelin mimics Ghrelin
  • Improves survival and motor function in ALS mouse models
  • Neuroprotective & anti-inflammatory

NU-9 Preclinical research

  • Reduces protein misfolding and protein clumping
  • Non-toxic and crosses the blood brain barrier
  • After 60 days of treatment, diseased brain cells look like healthy cells
  • More research needed before clinical trial can be initiated

RNA Aptamer Preclinical research

  • Investigational RNA molecule
  • Halts the death of motor neurons
  • Preserves motor function
  • Binds to glutamate receptors and prevents calcium overload within motor neurons
  • Ghrelin is often reduced in ALS patients
  • Macimorelin mimics Ghrelin
  • Improves survival and motor function in ALS mouse models
  • Neuroprotective & anti-inflammatory

NU-9 Preclinical research

  • Reduces protein misfolding and protein clumping
  • Non-toxic and crosses the blood brain barrier
  • After 60 days of treatment, diseased brain cells look like healthy cells
  • More research needed before clinical trial can be initiated

RNA Aptamer Preclinical research

  • Investigational RNA molecule
  • Halts the death of motor neurons
  • Preserves motor function
  • Binds to glutamate receptors and prevents calcium overload within motor neurons

Macimorelin:

Preclinical research

CRISPR gene therapy:

Preclinical research
  • Uses the gene-editing “scissor” tool CRISPR to delete a genetic mutation
  • Ideally, without the mutated gene, cells cannot make the toxic proteins anymore- and the disease can’t develop
  • Improves survival and motor function in ALS mouse models
  • Neuroprotective & anti-inflammatory

CRISPR gene therapy: