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MISSION STATEMENT

To improve the lives of patients suffering from rare genetic diseases (“RGD or Orphan Diseases”), and the lives of their families, friends and colleagues. Most patients suffering from Orphan Diseases typically have few, if any, treatment options. Our mission is to bring new biotherapeutics that can improve the lives of these patients via a robust research and development program.

OUR SCIENCE

 

ERAD Therapeutics Inc.

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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS

NEUROMUSCULAR DISEASE

Neuromuscular diseases are a large group of disorders that primarily disrupt the normal functioning of muscles and the nerves that control them. This leads to muscle weakness, impaired movement, and often progressive disability.

Examples of neuromuscular diseases are:

  • Muscular Dystrophy including Duchenne, Becker and many more;
  • Charcot- Marie-Tooth Disease and other neuropathies;
  • Myesthenia-gravis and more

GAUCHER DISEASE

Gaucher Disease is the most common Lysosomal Storage Disease, yet it is still considered an Orphan Disease. Currently affecting over 8,000 American’s, it is a genetic disorder resulting in an enzyme deficiency. Type 1 disease usually results in an enlarged liver, enlarged spleen, anemia and bone disease. Without therapy this significantly impacts both the quality and duration of life. Type 3 disease impacts all of the above as well as causing progressive and ultimately fatal neurological deterioration.

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Neuromuscular diseases are a large group of disorders that primarily disrupt the normal functioning of muscles and the nerves that control them, leading to muscle weakness, impaired movement, and sometimes progressive disability. This group of diseases can affect any part of the system responsible for voluntary muscle movement, including muscle fibers, peripheral nerves, motor neurons, the neuromuscular junction, and supporting structures like the plexus and nerve roots.

The main categories of neuromuscular diseases include:

  • Myopathies: Diseases directly affecting muscle fibers, including muscular dystrophies such as Duchenne, Becker, Limb Girdle and many more.
  • Motor neuron diseases: These involve progressive loss of upper or lower motor neurons, like amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy.
  • Neuropathies: Disorders affecting peripheral nerves, such as Charcot-Marie-Tooth disease, diabetic neuropathy, and Guillain-Barre syndrome.
  • Neuromuscular junction disorders: Conditions that impair the transmission between nerves and muscles, like myasthenia gravis and Lambert-Eaton myasthenic syndrome.

Symptoms and Examples

Common symptoms include muscle weakness, cramps, numbness, spasms, and impaired motor coordination. Some notable neuromuscular diseases include ALS, muscular dystrophies (DMD, BMD, FSHD), Pompe disease, myasthenia gravis, and Charcot-Marie-Tooth disease.

 

Cystic fibrosis (CF), a designated Orphan Disease, is a chronic, genetic illness that affects the digestive and respiratory tracts and is usually detected in pre-adolescents. CF results in generalized malnutrition and chronic respiratory infections. Long-term issues include difficulty in breathing and coughing up of mucus as a result of frequent lung infections. This is attributed to a notable thickening of mucus due to the genetic mutation of the CFTR protein.

  • There are currently approximately 33,000 people living with CF in the US and Canada.
  • Medical advances over the years have improved quality of life and survival rates, but, until recently, have done little to address the underlying causes.
  • Recently approved therapeutics and many under development are now serving to address the underlying genetic mutation(s) causing the disease.

FDA Approved Treatments

There are currently two recent therapeutics approved to treat the underlying genetic causes (as opposed to the symptoms) of CF:

  • KALYDECO® is the brand name of ivacaftor. Ivacaftor is what is called a potentiator, a molecule that assists in the improvement of a mis-folded but functional protein and improves its activity at the cell surface. KALYDECO® is approved for the treatment of patients who have the G551 D mutation, in approximately 4-5% of CF patients.
  • ORIKAMBI® is the brand name for a combination therapy of ivacaftor  and lumacacftor. Lumacaftor is a chaperone drug that helps the mutated CFTR protein fold properly so more of it can be transported to the cell surface where it works. 
  • ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor)
  • TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

 

Unique Drug Delivery System

  • Novel, proprietary plasmid for use in gene therapy and biomolecule production
  • Capable of carrying large genetic payloads
  • High efficiency, lower cost production
  • Under development for targeted neuromuscular diseases

Platform technology

  • Modular gene therapy technology adaptable to numerous targets
  • mCT gene therapy, based on temporary blockade of the Endoplasmic Reticulum Associated Degradation (ERAD) pathway
  • Portfolio of patent protected, related but distinct compounds
  • Supported by data in animals and cell lines

Initial focus on:

  • Neuromuscular diseases: (TBA)
  • CF and/or Lysosomal Storage Diseases:
    • Orphan Drug Designation for Gaucher Disease
  • Trans blood-brain-barrier applications accessing neuronal tissues,
  • Intellectual Property protection: key patents issued and pending covering all technologies extending to 2044 and beyond.

Subcategories

Gaucher Disease

Other Lysosomal Storage Diseases