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Ribomic

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RIBOMIC Announces Completion of IND Submission for an Observational Study for Continuous Phase 2 Trial of RBM-007 for Treatment of Achondroplasia

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

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Ciitizen

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Praxis Precision Medicines Partners With Ciitizen to Improve Patient-Guided Drug Development

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

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Astellas Pharma

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U.S. FDA Grants Fast Track Designation to Astellas Pharma's ASP0367/MA-0211

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

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Ribomic

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RIBOMIC Announces Dosing of First Subject in RBM-007 Phase 1 Clinical Trial for Achondroplasia

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

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