A New Biotech Hopes to Bring Us the Next Generation of Rare Metabolic Disorder Drugs

metabolic disorders chaperones gain thx 1

Barcelona-based biotech, Minoryx, has spun out Gain Therapeutics to develop the next generation of drugs for rare metabolic disorders like GM1 gangliosidosis.

Gain Therapeutics is a new biotech that will focus on developing new pharmacological chaperones for the treatment of rare diseases. It was set up by Minoryx, a biotech that has so far raised over €24M for therapies against genetic metabolic diseases, and a group of Swiss investors based in Lugano, Switzerland, including TiVenture – no financial data for the deal was disclosed. Gain Therapeutics is looking to apply its technology to a specific group of metabolic disorders called lysosomal storage disorders, which cause the severe and progressive degeneration of the nervous system.

SEE-Tx, or Site-directed Enzyme Enhancement Therapy, will help to develop the next generation of non-competitive pharmacological chaperones. These small molecules rescue mutated proteins, which no longer function correctly and cause disease. The hope is that this technology will improve the treatment of rare metabolic disorders. Gain Therapeutics has its hands full, with the biotech working on 5 programs that are at various stages of development.

The technology has been developed by Minoryx, which is also working on the clinical development of its lead candidate, MIN-102, which passed a Phase I trial for the treatment of X-linked Adrenoleukodystrophy (X-ALD) and received Orphan Drug Designation from the FDA last year. According to Marc Martinell, Minoryx’s co-founder and CEO, the decision to spin out will “allow Minoryx to focus on its clinical candidate, MIN-102, which is now in phase II/III clinical trials for the treatment of X-linked adrenoleukodystrophy and in preclinical development for other orphan CNS diseases.”

TiVenture’s managing partner, Lorenzo Leoni, will take interim charge of Gain Therapeutics and he is excited to build on the work done by the team at Minoryx. “SEE-Tx has already identified molecules that have the potential to become novel therapies for the treatment of several devastating rare diseases… We will invest in the further development of these molecules that should lead to the identification of clinical candidates in at least two independent programs.”

A number of biotechs are looking at lysosomal storage disorders, which affect the body’s capacity to break down heparan sulfate, a long chain sugar molecule that builds up in lysosomes to cause the degeneration of the central nervous system. In particular, Sanfilippo A syndrome has received plenty of attention from biotechs like Sobi, which had its investigational new drug application accepted by the FDA, and Lysogene, a company set up by one of our favorite female entrepreneursKaren Aich. This biotech’s gene therapy that replaces the defective sulfamidase enzyme is undergoing a Phase II/III trial.

With a range of different approaches being taken, including gene therapy, enzyme replacement therapy and now pharmacological chaperones, it will be interesting to see who comes out on top. Lysogene is currently leading the field but we will see if Gain Therapeutics’ technology can compete if it can make it through the clinic and onto the market.

Media – vitstudio / shutterstock.com; Minoryx Therapeutics

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