Leading the Way in Life Science Technologies

GEN Exclusives

More »

GEN News Highlights

More »
March 14, 2017

Ranedis and AMO Partner to Develop HDAC Inhibitor for Rare Genetic Diseases

  • AMO Pharma and Ranedis Pharmaceuticals inked a collaboration agreement to develop the latter’s histone deacetylase (HDAC) inhibitor RND-001 for the potential treatment of specific lysosomal storage disorders and other rare neurodegenerative and genetic diseases. As part of the agreement, AMO Pharma retains an exclusive option to acquire global rights to RND-001.

    RND-001 is a novel HDAC inhibitor formulation that Ranedis claims demonstrates enhanced brain exposure and has shown potential efficacy in a mouse model of Neimann-Pick type C (NPC) disease. "The preclinical data has provided strong validation of the potential for RND-001 to uniquely address central nervous system symptoms in NPC and other rare diseases,” said William Claypool, CEO at Ranedis Pharmaceuticals. "We are pleased that the development program will advance under this agreement with AMO Pharma."

    "RND-001 is a promising therapy supported by compelling early-stage research that gives Ranedis Pharmaceuticals and AMO Pharma an excellent opportunity to leverage known mechanisms in previously inaccessible rare diseases,” added Michael Snape, Ph.D., AMO Pharma’s CEO. “This collaboration represents an important next step in the expansion of our pipeline at AMO Pharma.”

    Ranedis was established in 2014 to develop technology, licensed from the University of Notre Dame, for treating rare and neglected diseases. The firm says that in addition to demonstrating promising results in the NPC mouse model, research indicates that RND-001 can also impact gene expression implicated in other rare metabolic diseases, including Gaucher’s disease, GM-1 gangliosidosis, and Krabbe’s disease. 

    Founded in February 2015, AMO Pharma is acquiring a pipeline of therapeutic assets for treating rare and orphan diseases. The firm’s clinical candidates include the RAS-ERK inhibitor AMO-01, which is in Phase I development for the potential treatment of fragile X syndrome. A U.K. Phase II study with the glycogen synthase kinase-3 beta (GSK3b) inhibitor tideglusib (AMO-02 ) was initiated in August 2016 in adult patients with congenital and juvenile onset myotonic dystrophy.

Related content