AMO Pharma Enters into Collaboration Agreement with Ranedis Pharmaceuticals for Development of RND-001

LONDON, UK and PHILADELPHIA, PA, USA I March 14, 2017 I AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment options, today announced it has entered a collaboration agreement with Ranedis Pharmaceuticals, a privately held U.S. biopharmaceutical company, to progress the development of  RND-001 for rare genetic diseases, including certain lysosomal storage disorders and other diseases affecting the central nervous system. In addition, Ranedis has granted AMO an exclusive option to acquire the worldwide rights to RND-001 during the collaboration period. Terms of the deal were not disclosed.  

RND-001 is a unique formulation of a histone deacetylase inhibitor (HDACi) whose properties allow for delivery of the molecule to the brain.  In some genetic disorders, HDACis can result in increased or decreased transcriptional expression of mutated genes and confer other benefits associated with modulations of key transcriptional proteins. In pre-clinical research, RND-001 has been demonstrated to both stimulate gene transcription and protein expression in the brain and has shown potential efficacy in a mouse model of Neimann-Pick Type C disease (NPC). On this basis, AMO and Ranedis will research a range of applications in the treatment of rare diseases including certain neurodegenerative diseases for which there are currently no or limited treatments.

"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.  This collaboration represents an important next step in the expansion of our pipeline at AMO Pharma. RND-001 reflects many of the specific qualities we look for in asset acquisition, such as strong scientific rationale and opportunity to address unmet medical needs in rare diseases.  We will continue to target new opportunities that fit our strategic focus," said Michael Snape, chief executive officer of AMO Pharma.

"The preclinical data have provided strong validation of the potential for RND-001 to uniquely address CNS symptoms in NPC and other rare diseases.  We are pleased that the development program will advance under this agreement with AMO Pharma," said William Claypool, chief executive officer of Ranedis Pharmaceuticals. "

About AMO Pharma
AMO Pharma is a biopharmaceutical company incorporated in February of 2015. The co-founder, Dr. Michael Snape, has extensive experience in senior scientific and operational roles in both large pharma and biotech companies spanning more than twenty five years, and has brought together a targeted and experienced senior management team with a proven track record of success in all phases of product development and acquisition. The company is working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare genetic diseases. AMO Pharma is currently advancing two investigational therapies for treatment of fragile X syndrome and myotonic dystrophy, and is in the process of licensing additional development-stage products in diverse areas including autism and other CNS disorders. For more information, please visit the AMO Pharma website at

About Ranedis Pharmaceuticals
Ranedis Pharmaceuticals, a private biopharmaceutical company established in 2014, is developing novel therapies for the treatment and management of rare and neglected diseases. The Company's initial efforts are focused on select monogenetic disorders, including lysosomal storage diseases. Ranedis' co-founder, Dr. William Claypool, has been in the pharmaceutical industry for over twenty-five years and has assembled a senior management team with extensive pharmaceutical and biotech industry experience. For more information, please visit the Ranedis Pharmaceuticals website at


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