Accelerating the development of novel Niemann-Pick Type C therapies.

The NPC Community is experiencing unprecedented investment and progress in developing treatments for the disease. Today, at number of companies have NPC programs underway, two of which are in later stages of regulatory review with the FDA. Additionally, there are a number of preclinical programs being funded and supported by NPC organizations, government grants, or investors. There are also a number of collaborative research programs established to tackle key aspects of the disease that are being managed by various organizations and research groups.

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Our Goal

The goal of the Accelerator is to:

  • Provide easily accessible information from all companies willing to share their NPC therapy pipeline;
  • Inform companies on how to engage with the committee, and the benefits of working with the NTAI as a resource to help them connect with NPC researchers, clinicians, tools, data, mentors, and even potential investors;
  • Compile all major NPC publications that physicians and scientists use to advance their research program and launch a clinical trial;
  • Provide shared space for all NPC initiatives and data, and ultimately bring this information together in a shared data platform;
  • Provide relevant updates for research funding opportunities; and
  • Additional NPC patient and caregiver preference data development.
A collaborative group of researchers with tools to advance NPC research including models, techniques, equipment, and expertise.
Learn more about exciting potential therapies that are in the preclinical stage of research.
Conferences are a wonderful way to share ideas and meet the NPC community.
Learn more about NPC research funding opportunities.
Learn how the Accelerator team is working with federal and healthcare organizations to provide better access for your family.
This collaboration is working with Federal Agencies to bring awareness and expedite NPC research and therapy approval.

Contact the Accelerator Team

The NPC Therapy Accelerator team is eager to assist you navigate the NPC community — from finding a collaborator to learning the latest news in NPC drug development. Please send us a message if you have any questions. We will be glad to follow up and discuss the Accelerator with you.