Our portfolio is a result of high-throuput screening of a diverse library containing lead-like and drug-like compounds to discover small molecule inhibitors of accumulation of insoluble glycogen or polyglucosan bodies, using an APBD patient skin fibroblast cell-based assay. As all Glycogen Storage Diseases (GSDs) share the etiology of excessive normal or malconstructed glycogen, we believe that APBD represents a prototypical GSD and thus development of pharmaceutical inhibitors of glycogen accumulation is likely to benefit most GSDs.

Ex-vivo High-Throughput Screening

  • Cell-based assay* to detect inhibition of polyglucosan accumulation with library of small molecules.
  • Assay readout: Amylase-resistant fluorescence of Periodic Acid Schiff’s reagent (PAS) staining.
  • Use of APBD patient derived fibroblasts for screening Software-based analysis of cell images.
  • Focus on ADMET-compatible (novel and ‘lead-like’) compounds.

* Amylase-resistant fluorescence of Periodic Acid Schiff’s reagent (PAS) staining.

APBD patient fibroblasts

Solmesky et al  (2017)

Healthy control fibroblasts

The selected compounds were further investigated in vitro and in vivo.

In an APBD mouse model (Gbeys/ys), our lead compound, GHF-201 (Compound A), was effective in lowering polyglucosan bodies, improving motility and , carbohydrate burn and lifespan extension.

Furthermore, our extensive cell and in vitro analyses show that GHF-201 targets the lysosomal protein LAMP1 and enhances autophagic flux as well as respiratory and glycolytic ATP production, transforming the adversely excessive glycogen into a useful fuel source.

First In Human – GHF-201 was approved for an Urgent Compassionate Use program in Israel limited to 3 APBD patients.

GHF partnered with LDS to formulate GHF-201 in a highly-bioavailable oral nanodroplet solution, to be administered daily.

Currently, there are 2 APBD patients in this program, under the care of Prof. Alexander Lossos and his team at Hadassah University Hospital-Ein Kerem, Jerusalem Israel. Data from clinical/laboratory tests and patient feedback is being collected by the medical team.

The growing knowledge and evidence about GHF-201 suggest that it may offers a new, safe, and efficacious approach to treat APBD and additional glycogen and lysosomal storage disorders, as well as diseases with greater prevalence.

Research and Development

GHF-201 reduces glycogen in target organs of APBD mouse*

*homozygous for the human mutated GBE gene Gbeys/ys

GHF-201 improves motility in APBD mouse*

*Homozygous for the human mutated GBE gene Gbeys/ys

GHF-201 extends survival in the APBD mouse*

*Homozygous for the human mutated GBE gene Gbeys/ys​

Vaknin et al, 2021

GHF-201 Mechanism of Action is Known*

GHF-201 modulates lysosomal and autophagy genes

LAMP1 has a pivotal role in lysosome integrity and function

*Determined by NPOT technology

Vaknin et al, 2021

GHF-201: From target identification to additional indications

  •  GHF-201 is a novel compound capable of degrading polyglucans and over-accumulated glycogen
  • In vivo proof of concept for use of GHF-201 in treating APBD and other glycogen storage disorders
  •  GHF-201 activates auto-lysosomal catabolism
  • Potential to treat lysosomal storage disorders (LSDs), such as Gaucher disease
  •  GHF-201 reduces nuclear & cellular levels of glycogen


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