Turning the unthinkable into reality for rare diseases

  • Golden Heart Flower (GHF) is a clinical stage company with a mission to develop a treatment for Adult Polyglucosan Body Disease (APBD)
  • We established our portfolio by performing High Throughput Screening assays on a library of small molecules, using advanced computational techniques we characterized our lead candidates which are now under further investigation.
  • Our core team is highly experienced in drug development and scientific methodologies, integrated with academia collaborations, our aim is bringing to market novel therapeutic candidates for uncurable rare disorders.
  • Our motivation is to stand alongside patients facing rare disorders and to improve their lives where few treatment options exist.

5

Number of assets

GHF-201, GHF-203, GHF-204, GHF-205, GHF-101

34,800

Affected individuals with GSD IV

3400-6400

APBD patients

5

Disorders studied in animal models using GHF-201

APBD, CORI, Von Gierke, Pompe, MPS1

>11

Cumulative patient-years in APBD compassionate use program

3 APBD patients

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