Table 1. TRND partnerships. List of NIH Therapeutics for Rare and Neglected Diseases (TRND) partnerships as of Dec. 12, 2011.

Source: NIH Center for Translational Therapeutics





Pilot projects



Small molecule that binds sickle hemoglobin

Sickle cell disease

New Zealand Pharmaceuticals Ltd./National Human Genome Research Institute

N-Acetyl-d-mannosamine (ManNAc)

Correct sialic acid deficiency

Hereditary inclusion body myopathy (hIBM)

Leukemia & Lymphoma Society/The University of Kansas Medical Center/National Heart, Lung, and Blood Institute


Selectively kills chronic lymphocytic leukemia (CLL) cells


Niemann-Pick Type C disease foundations/Washington University in St. Louis/Albert Einstein College of Medicine of Yeshiva University

2-Hydroxypropyl-b-cyclodextrin (HPBCD)

Reduction of cholesterol and sphingolipid storage

Niemann-Pick disease type C1 (NPC1)

First round of solicited projects

Afraxis Inc.

Not applicable

p21 protein (Cdc42 Rac)-activated kinase (PAK) inhibitor

Fragile X syndrome

ReveraGen Biopharma Inc.


Modified glucocorticoid with fewer side effects

Duchenne muscular dystrophy (DMD)

Viamet Pharmaceuticals Inc.


Cytochrome P450 C-14 α-demethylase (CYP51) inhibitor

Cryptococcal meningitis

National Human Genome Research Institute

Not applicable

Inhibitor of core binding factor (CBF) protein interactions

CBF-driven acute myelogenous leukemia (AML) and acute lymphoblastic leukemia (ALL)

Second round of solicited projects

Harvard Medical School

Dorsomorphin derivative (small molecule)

Bone morphogenetic protein type I receptor inhibitor

Fibrodysplasia ossificans progressiva

Lumos Pharma Inc.

CincY, an undisclosed repurposed molecule with an inactive IND

Blood brain barrier-penetrant creatine mimetic

X-linked creatine transporter defect (CTD)

The University of Alabama at Birmingham

CMX001, a lipophilic derivative of cidofovir

CNS-penetrant double-stranded DNA viral synthesis inhibitor

Neonatal herpes simplex virus infection

Concert Pharmaceuticals Inc.

Deuterium-modified praziquantel derivative

Small molecule antischistosome


AVI BioPharma Inc.

AVI-5038, a phosphorodiamidate morpholino oligomer targeting dystrophin exon 50

Corrects dystrophin exon 50 splicing defect

Duchenne muscular dystrophy (DMD)

Cincinnati Children's Hospital Medical Center

Inhaled formulation of granulocyte macrophage colony-stimulating factor (GM-CSF; CSF2)

Restore GM-CSF function

Pulmonary alveolar proteinosis