Date: 2017-03-14
Type of information: Development agreement
Compound: RND-001
Company: AMO Pharma (UK) Ranedis Pharmaceuticals (USA - PA)
Therapeutic area: Rare diseases - Genetic diseases - CNS diseases -Neurodegenerative diseases
Type agreement: collaboration development
Action mechanism: histone deacetylase inhibitor. 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.
Disease:
Details: * On March 14, 2017, AMO Pharma announced it has entered a collaboration agreement with Ranedis Pharmaceuticals 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. 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.
Financial terms:
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