Drugs in Dev. Genetic Disease
Phase II/ Phase III
Japan 
Lead Product(s) : Idursulfase
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Elaprase (Idursulfase) is a Enzyme drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Mucopolysaccharidosis II.
Product Name : Elaprase
Product Type : Enzyme
Upfront Cash : Inapplicable
September 11, 2023
Lead Product(s) : Idursulfase
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : TAK-881
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Takeda Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : TAK-881 is a drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Immunologic Deficiency Syndromes.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
March 06, 2023
Lead Product(s) : TAK-881
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Takeda Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : In this study, 50 patients with Hunter syndrome will be treated with JR-141 or the standard treatment for up to 105 weeks under a standard of care controlled, parallel-group trial design.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
February 10, 2021
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : JR-141 is a recombinant fusion protein of antibody against the human transferrin receptor and idursulfase, the enzyme missing or malfunctioning in subjects with MPS II.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
February 10, 2021
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : TAK-672
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of TAK-672 in Participants With Acquired Hemophilia A
Details : TAK-672 is a drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Factor 8 Deficiency, Acquired.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
October 08, 2020
Lead Product(s) : TAK-672
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Details : JR-141 is a Antibody drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Mucopolysaccharidosis II.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
April 16, 2020
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
JCR Announces Presentations on JR-141 and JR-162 at the 16th Annual WORLDSymposium 2020
Details : The presentations highlight two candidates from JCR’s development pipeline for lysosomal storage disorders; JR-141 and JR-162.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
February 13, 2020
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study of JR-141 in Patients With Mucopolysaccharidosis II
Details : JR-141 is a Antibody drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Mucopolysaccharidosis II.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
June 26, 2018
Lead Product(s) : JR-141
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)
Details : Resamirigene Bilparvovec is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of X-Linked Myotubular Myopathy.
Product Name : Undisclosed
Product Type : Cell & Gene Therapy
Upfront Cash : Inapplicable
June 27, 2017
Lead Product(s) : Resamirigene Bilparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II/ Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Reset All
