APMRF is underwriting 12 projects for the 2026-27 Grant Funding Program. These support a collaborative group of leading Niemann-Pick Type C scientists from around the world.

Grants funded back a variety of high-impact investigations that include understanding the NPC pathway, the structure of the NPC1 protein, and how NPC proteins traffic cholesterol in and out of the cell.

Additionally, the APMRF is strengthening the expansion of novel tools to assist researchers studying NPC disease, the development of novel therapeutics for the treatment of the disease, and the identification of novel biomarkers that will be used to accelerate the therapeutic development process. We are also enthusiastic about our partnership with the International Niemann-Pick Disease Registry, and we have a current multiyear commitment to support a newborn screen to identify children with NPC disease at birth.

The 2026 funded grantees

• Monther Abu-Remaileh (The Board of Trustees of the Leland Stanford Junior University)
  BMP Homeostasis as a Therapeutic Axis in Niemann-Pick Type C Disease
• Brian Blagg (University of Notre Dame - Warren Center)
  Chemical synthesis core
• Shaun Bolton (International Niemann-Pick Disease Registry)
  Growing the patient-led International Niemann-Pick Disease Registry in the United States & Canada
• Tatiana Bremova-Ertyl (Inselspital Bern)
  To complement existing assessments by establishing objective, high-resolution digital measures of gait and balance using wearable sensor-based shoes (NUSHU) and evaluating their responsiveness to vibrational biofeedback.
• Eamonn Dickson (University of California, Davis)
  JPH3 Regulation of Neuronal Cholesterol Homeostasis in Niemann-Pick Type C Disease
• Xiaochun Li (UT Southwestern Medical Center)
  establish whether p75NTR targeting can deliver true disease modification in NPCD. Given the clinical readiness of LM11A-31
• Andy Lieberman (University of Michigan)
  To provide unique insights into lysosomal dysfunction in the Niemann-Pick C brain and into the factors that contribute to selective neuronal vulnerability.
• Fred Maxfield (Weill Cornell Medicine)
  Examining two approaches to understanding and treating Niemann Pick C disease.
• Denny Porter (Eunice Kennedy Shriver National Institute of Child Health and Human Development)                                                                                  Preclinical Evaluation of Novel TfR1-Targeting AAV Vectors for Systemic Gene Therapy of Niemann-Pick Type C1 Disease
• Rajat Rohatgi (Stanford University School of Medicine)                                                                                                                                                                        Mapping druggable sites on NPC1 using base-editing screens
• Hijai Regina Shin (UT Southwestern Medical Center)
  Defining the cellular, metabolic, and molecular mechanisms by which INT-777 restores lysosomal function and to evaluate its therapeutic potential in vivo
• Roberto Zoncu (University of California Berkeley)
  To elucidate how mTORC1 signaling regulates neuronal function in Niemann-Pick type C (NPC) disease and evaluate the therapeutic potential mTORC1 inhibition.