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Science + Therapies Pipeline

A short summary of the research and clinical trials being conducted that impact Niemann-Pick disease.

Therapy Pipeline.png

The Australian NPC Disease Foundation continues to advocate for all research and development programs that present potential treatment options for those living with Niemann-Pick disease (NP-C and ASMD) in Australia.

If you would like to submit your program or have updated information, please contact the Australian NPC Disease Foundation at info@npcd.org.au.

Last updated

October 2024

Program Information and Links:

mRNA gene therapy

The Florey Research Institute of Neuroscience and Mental Health ↪️

Preclinical Stage

MIPLYFFA (Arimoclomol)

Zevra Therapeutics ↪️

Commercial Product

RAINBOW

Azafaros ↪️

Phase 2 Study

DL33

Meizon Innovations ↪️

Preclinical Stage

Xenpozyme (olipudase alfa)

Sanofi ↪️

Commercial Product

AQNEURSA™ (levacetylleucine)

IntraBio Inc ↪️

Commercial Product

Trappsol®️Cyclo™️

Cyclo Therapeutics ↪️

Pivotal Phase 3 Study

Substrate reduction using ASOs

University of Tasmania ↪️

Preclinical Stage

Adrabetadex

Mandos Health ↪️

Expanded Access only

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