Drugs in Dev. Genetic Disease
Phase I
Japan 
Lead Product(s) : ASP2016
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
Details : ASP2016 is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Friedreich Ataxia.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
July 03, 2024
Lead Product(s) : ASP2016
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Umedaptanib Pegol
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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 ...
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
April 19, 2022
Lead Product(s) : Umedaptanib Pegol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : ASP0367
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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 patientâ...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
October 20, 2020
Lead Product(s) : ASP0367
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Umedaptanib Pegol
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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.
Product Name : Undisclosed
Product Type : Oligonucleotide
Upfront Cash : Inapplicable
July 15, 2020
Lead Product(s) : Umedaptanib Pegol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Bocidelpar
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Bocidelpar is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Muscular Dystrophy, Duchenne.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 04, 2019
Lead Product(s) : Bocidelpar
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : TAS-205
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study of TAS-205 for Duchenne Muscular Dystrophy
Details : TAS-205 is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Muscular Dystrophy, Duchenne.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
September 22, 2014
Lead Product(s) : TAS-205
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Ixazomib Citrate
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of Oral Ixazomib in Adult Participants With Relapsed or Refractory Light Chain Amyloidosis
Details : Ixazomib is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
March 21, 2011
Lead Product(s) : Ixazomib Citrate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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