Details:
RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo, has been shown to have potent effects in limiting excessive interactions between fibroblast growth factors, known to cause achondroplasia.
Lead Product(s): RBM-007
Therapeutic Area: Genetic Disease Product Name: RBM-007
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 19, 2022
Details:
Praxis will utilize the Ciitizen platform to provide deeper insights across disease populations through the analysis of real-world data, initially to inform the development of PRAX-222 and PRAX-562 in SCN2A developmental and epileptic encephalopathy (SCN2A-DEE).
Lead Product(s): PRAX-562
Therapeutic Area: Genetic Disease Product Name: PRAX-562
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Praxis Precision Medicines
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership February 16, 2021
Details:
Preclinical data and results from Astellas’ Phase I healthy volunteer study collectively suggest that ASP0367 could improve exercise intolerance and fatigue in PMM patients by increasing the number and enhancing the function of mitochondria in patient’s cells.
Lead Product(s): ASP0367
Therapeutic Area: Genetic Disease Product Name: MA-0211
Highest Development Status: Phase I Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 20, 2020
Details:
RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo.
Lead Product(s): RBM-007
Therapeutic Area: Genetic Disease Product Name: RBM-007
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 15, 2020