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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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Our unique gene therapy platform enables high-efficiency, cost-effective approaches to gene therapy. This allows us a two-fold approach to treatment of rare genetic diseases:

  1. Targeted, disease-specific gene therapy therapeutics with an initial focus on neuromuscular diseases
  2. A broad-based, “gene-agnostic” platform approach pursuing multiple rare genetic diseases with a single gene therapy product

ERAD Therapeutics Inc. has also developed a novel biomolecule called mCT to rescue mutated, misfolded proteins from destruction by Endoplasmic Reticulum Associated Degradation (ERAD). This unique biomolecular platform employs a modified, inactivated cholera toxin (mCT) that has had its cytotoxic activity eliminated by genetic manipulation of the DNA. mCT can enter all cells in the body, and is capable of crossing the blood brain barrier.

mCT has been demonstrated to rescue mutated, misfolded, partially functional proteins in multiple rare genetic diseases, including cystic fibrosis and several lysosomal storage diseases, by interfering with their destruction. These accomplishments give us a “gene-agnostic” approach to treatment of numerous rare and very rare genetic diseases with unmet clinical needs. mCT received Orphan Drug Designation for Gaucher Disease in 2017

Recent developments enable us to take a gene therapy approach for mCT (mCT-GT) offering new hope to many patients, families and stakeholders.

ERAD Therapeutics, Inc. is currently developing our proprietary disease-specific gene therapy for targeted diseases and our broad-based mCT-gene therapy for delivery to a broad spectrum of diseases, including across the blood brain barrier (BBB) and into CNS tissues.

1/ Our novel gene therapy vector is initially being developed for targeted gene therapy in neuromuscular diseases (TBA).

2/ Many proteins that harbor a single amino acid mutation, though slightly mis-folded, still retain activity. Normally these mis-folded proteins are destroyed by the endoplasmic reticulum (Endoplasmic Reticulum-Associated Degradation aka ERAD). Our mCT-GT candidate is under initial development in certain lysosomal storage diseases, but has the capability of treating many rare genetic diseases based on successes in pre-clinical development in a variety of diseases. Our mCT-GT used in this approach is expected to have the ability to reversibly interfere with the ERAD process, allowing the mis-folded, but functional protein, to transit to its site of action where it can restore normal or near-normal cellular activity in multiple diseases creating a therapeutic platform.
 
 

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