Rafael Holdings Sets Stage for Key Presentations on Rare Disease Treatment
Rafael Holdings Highlights Important Presentations at ICIEM
Rafael Holdings, Inc. announced that its subsidiary, Cyclo Therapeutics, has two research abstracts chosen for both oral and poster presentations at the International Congress of Inborn Errors of Metabolism (ICIEM). This prestigious event will be hosted in Kyoto, Japan.
Presentation Details
The selected presentations reflect the company's commitment to addressing rare diseases like Niemann-Pick Disease Type C1. Here are the details:
Oral Presentation
Title: Hydroxypropyl-beta-cyclodextrin for Long-Term Treatment of Niemann Pick Type C1: Efficacy and Safety Data from Clinical Studies
Presenter: Dr. Caroline Hastings, Pediatric Hematologist Oncologist, UCSF Benioff Children's Hospital
Date & Time: September 3, 2025, 14:00 - 16:00 JST
Location: Room 3, Kyoto International Conference Center
Poster Presentation
Title: Trappsol Cyclo Open Label Treatment in the TransportNPC Sub-Study in Patients Under Age 3 with Niemann Pick Disease Type C1
Presenter: Dr. Orna Staretz Chacham, Senior Neonatologist, Soroka Medical Center, Israel
Date & Time: September 3, 2025, 18:00 - 19:00 JST
Location: Poster 1, Annex Hall, Kyoto International Conference Center
Overview of Trappsol Cyclo
Trappsol Cyclo is an innovative formulation of hydroxypropyl-beta-cyclodextrin, administered intravenously. It's designed to facilitate the mobilization of cholesterol from cellular structures. The aim is to address the underlying causes of Niemann-Pick Disease Type C1 by removing excess cholesterol from late-stage endosomes and lysosomes.
Impact of Trappsol on Clinical Outcomes
This drug has demonstrated the ability to cross the blood-brain barrier, potentially allowing therapeutic levels of treatment to reach the central nervous system during administration. The ongoing Phase 3 TransportNPC study, where Trappsol Cyclo is assessed for safety and efficacy, enrolled 94 participants in various global locations.
Phase 3 TransportNPC Study
This comprehensive study evaluates the drug given at a dose of 2000 mg/kg bi-weekly for patients confirmed with Niemann-Pick Disease Type C1. Significant endpoints include evaluating clinical severity based on established clinical scales utilized in both the United States and Europe.
Niemann-Pick Disease Type C1 Explained
Niemann-Pick Disease Type C1 is a rare genetic disorder affecting approximately 1 in 100,000 live births worldwide. This condition interrupts cholesterol processing in cells, leading to organ damage in critical areas such as the brain and liver. Patients often experience various symptoms, including cognitive decline and loss of motor skills.
About Rafael Holdings, Inc.
Rafael Holdings, Inc. is known for its investment in biotechnology ventures that focus on rare diseases. Alongside Cyclo Therapeutics, Rafael also collaborates with multiple pharmaceutical and medical device companies, aiming to create impactful solutions in healthcare.
The Future of Rare Disease Treatment
Through Cyclo Therapeutics and its lead clinical candidate Trappsol Cyclo, Rafael Holdings hopes to change the treatment landscape for rare diseases. The ongoing trials promise potential breakthroughs for patients and families affected by genetic disorders.
Frequently Asked Questions
1. What is Trappsol Cyclo?
Trappsol Cyclo is a proprietary drug formulation aimed at treating Niemann-Pick Disease Type C1 by helping to mobilize cholesterol from cells.
2. Where will the ICIEM be held?
The International Congress of Inborn Errors of Metabolism will take place in Kyoto, Japan.
3. Who are the presenters at the upcoming congress?
Presenters include Dr. Caroline Hastings and Dr. Orna Staretz Chacham, both experts in their respective fields.
4. What is Niemann-Pick Disease Type C1?
Niemann-Pick Disease Type C1 is a rare genetic disorder that leads to the inability to process cholesterol, causing various health complications.
5. What role does Rafael Holdings play in biotechnology?
Rafael Holdings invests in companies like Cyclo Therapeutics, focusing on developing treatments for rare diseases, enhancing patient care.
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