AMO Pharma entered into a collaboration agreement with Ranedis Pharmaceuticals to advance the development of RND-001 for rare genetic diseases, including certain lysosomal storage disorders and certain diseases affecting the central nervous system. RND-001 is a 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 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). This provides a strong scientific basis supporting the potential use of RND-0001 in a range of applications in the treatment of rare diseases including certain neurodegenerative diseases for which there are currently no or limited treatments.
For more information on the RND-0001 program, please visit www.ranedis.com.