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AAN 2026 | Applications of GHF-201 in lysosomal and glycogen storage diseases

Or Kakhlon, PhD, Hadassah University Medical Center, Jerusalem, Israel, discusses preclinical evaluation of GHF-201 across multiple lysosomal and glycogen storage disease models, including Pompe disease, Gaucher disease, mucopolysaccharidosis type 1, and glycogen storage disorders. He highlights encouraging efficacy findings and the potential relevance of the compound for disorders with neurodegenerative components. This interview took place at the 78th American Academy of Neurology (AAN) Annual Meeting in Chicago, IL.

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Transcript

So as a matter of fact, we have tested GHF2-1 in five mouse models of storage diseases. We tested it in Pompe disease, in mucopolysaccharidosis type 1, or Hurler disorder, and in Gaucher disease. All are done in vivo in mouse models of the respective disorders. In all of them, we have no safety issues and very good efficacy results in many parameters. I don’t have time to elaborate too much, but for instance, in MPS-1, just as an example, in MPS-1, mucopolysaccharidosis type 1, there’s an improvement of the joint deterioration, which was never done before by any other drug, just as an example...

So as a matter of fact, we have tested GHF2-1 in five mouse models of storage diseases. We tested it in Pompe disease, in mucopolysaccharidosis type 1, or Hurler disorder, and in Gaucher disease. All are done in vivo in mouse models of the respective disorders. In all of them, we have no safety issues and very good efficacy results in many parameters. I don’t have time to elaborate too much, but for instance, in MPS-1, just as an example, in MPS-1, mucopolysaccharidosis type 1, there’s an improvement of the joint deterioration, which was never done before by any other drug, just as an example. But since you asked about neurodegeneration, these diseases are not typical neurodegenerative disorders, but they have components of neurodegeneration. So especially in MPS1, there is neurodegeneration and neuroinflammation because of the postural static deficiency. In Pompe disease, also, it was described that there is a neurodegenerative component, even though it’s a myopathy, it’s a muscle disorder. And also in Gaucher, type 1 and 3, there is a neuropathic component. So while not being bona fide neurodegenerative disorders, they have neurodegenerative components, okay? So these are the lysosomal storage disorders. We also have very good results in glycogen storage disorders, which were published. Glycogen storage disease type 1A, GSD1A, and type 3. And these are published to show efficacy of this drug as well. So it actually degrades the inclusions, glycogen inclusions, which cause the pathology.

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