HPGCD for Niemann-Pick Type C (NPC) Therapy without Side Effects

Intrathecal HPGCD(2-Hydroxypropyl-γ-cyclodextrin) improving brain abnormalities in NPC patients without side effects

Core Benefit

  • HPGCD is as effective for NPC therapy as the prior candidate intrathecal HPBCD (2-hydroxypropyl-β-cyclodextrin) which has already been in clinical trial.
  • HPGCD is far less toxic than HPBCD which has showed worsening of eye movement and hearing in some patients in clinical trial
  • HPGCD GMP production equipment is near completion

Background and Technology

Niemann‐Pick disease type C (NPC) is a hereditary lysosomal storage disease characterized by abnormal accumulation of free cholesterol and glycolipids associated with mutations in the lipid transporter genes, NPC1 and NPC2. NPC patients suffer from liver and neurological dysfunctions and eventually die due to respiratory and hepatic failure.

We established hepatocyte‐like cells and neural progenitors derived from iPSC lines of NPC patients. We found that HPGCD could reduce the cholesterol accumulation and restore the functional and molecular abnormalities in the NPC patient‐derived cells, and do so more effectively and much less toxically than HPBCD. In addition, NPC model mice showed an improved liver status and prolonged survival with intravenous HPGCD. Soga et al., Stem Cells. 2015 Apr;33(4):1075-88.

A clinical trial of prior therapeutic candidate, intrathecal HPBCD (VTS-270 by Vtesse, Inc.), is ongoing. Phase I trial showed that HPBCD for 6-12 months slowed the rate of disease progression and improved domains of cognition and speech. HPBCD was well tolerated other than worsening of eye movement and hearing in some patients.

Intrathecal HPGCD administration to NPC model mice indicated longer-term prolonged survival  than intravenous HPGCD. We believe that Intrathecal HPGCD improved brain abnormalities in NPC patients without side effects such as worsening of eye movement and hearing.

Data and Publication

  We can disclose the experimental data of intrathecal HPGCD administration to NPC model mice under a confidential disclosure agreement


WO2015083736 (JP, US, EP)


Dr. Era et al. (Kumamoto University, Japan)


We are preparing pre-clinical study of Intrathecal HPGCD. We can provide HPGCD based on GMP standards soon. We are looking for a pharmaceutical company focusing on rare disease therapy to develop this technology further.

Product No: TP0351



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