Spotlight on Australian Research: DL33, a Potential New Therapy for Niemann-Pick Type C
- ANPDF
- Oct 14
- 2 min read
Niemann-Pick Disease Type C (NPC) is a devastating and ultra-rare condition, often described as a form of childhood dementia. With no cure currently available, families face the heartbreaking reality of watching their loved ones lose skills and independence over time.
Here in Australia, world-class researchers are working to change that story. One promising initiative is DL33, a potential small molecule therapy being advanced by Meizon Innovations.
Where DL33 Stands Today
At the 2025 Australian Niemann-Pick Conference, Associate Professor Wah Chin Boon shared exciting progress on DL33. Since then, the program has achieved two major milestones:
✅ Successful GMP-grade large-scale manufacturing with >99% purity — a critical step before moving into safety studies.
✅ Funding secured to initiate animal toxicology studies.
Toxicology testing is set to begin in Q3 2025, with the goal of raising USD $4M to launch a Phase 1 clinical trial in early 2026. Each of these steps brings DL33 closer to the first trials in patients.
What this research means
For families affected by NPC, even incremental progress offers profound hope.
“Bringing the fight to Niemann-Pick, we are changing their tomorrow!” – Associate Professor Wah Chin Boon
Experts agree that combination therapies will be essential to treating NPC. DL33 represents one of several innovative approaches being developed to slow or alter the course of this devastating disease.
These milestones bring the project closer to the first trials in patients.
Learn More & Get Involved
You can find out more about DL33 and Meizon’s work here:
A Shared Effort for Awareness Month
This Niemann-Pick Awareness Month, ANPDF is proud to share updates on Australian-based research. These efforts reflect Australia’s role in advancing global NPC science — and in bringing treatments closer to families right here at home.
About Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C (NPC) is a rare, progressive, and life-limiting genetic disorder. Often referred to as “childhood dementia,” NPC affects the body’s ability to transport and store cholesterol and other lipids, leading to severe neurological decline. Symptoms can include difficulties with movement, learning, memory, swallowing, and speech. NPC affects children and adults alike, with no approved cure currently available in Australia. Learn more at Information and Support.
About DL33
DL33 is a novel small molecule therapy in development by Meizon Innovations. It is designed to target the underlying biological mechanisms of NPC and represents a step toward potential disease-modifying treatment.
About Meizon Innovations
Meizon Innovations is an Australian biotechnology company dedicated to developing new therapies for rare diseases. Its lead program, DL33, is focused on Niemann-Pick Disease Type C. Learn more at https://meizon.health/
About the Australian NPC Disease Foundation (ANPDF)
The ANPDF is the national organisation supporting families impacted by Niemann-Pick disease in Australia. We work to raise awareness, drive research, advocate for access to treatments, and build community support. Learn more at About Us.
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