Lead Product(s) : JR-446
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
JR-446 gets US FDA Orphan Drug Status for Mucopolysaccharidosis IIIB
Details : JR-446 is an investigational drug being developed for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B).
Product Name : JR-446
Product Type : Protein
Upfront Cash : Inapplicable
May 07, 2025
Lead Product(s) : JR-446
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-446
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Medipal Holdings Corporation
Deal Size : Inapplicable
Deal Type : Inapplicable
JCR Initiates Phase 1/2 Trial Of JR-446 for MPS Type IIIB in Japan
Details : JR-446, is a blood-brain barrier-penetrating α-N-acetylglucosaminidase, developed using JCR’s proprietary J-Brain Cargo technology, is under investigation for mucopolysaccharidosis type IIIB.
Product Name : JR-446
Product Type : Other Large Molecule
Upfront Cash : Inapplicable
December 05, 2024
Lead Product(s) : JR-446
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Medipal Holdings Corporation
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
JCR Pharmaceuticals Initiates Phase I Trial of JR-441 for Mucopolysaccharidosis Type IIIA
Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.
Product Name : JR-441
Product Type : Other Large Molecule
Upfront Cash : Inapplicable
October 31, 2024
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
JCR Completes Clinical Trial Notification for JR-441 in Mucopolysaccharidosis Type IIIA
Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.
Product Name : JR-441
Product Type : Other Large Molecule
Upfront Cash : Inapplicable
March 09, 2024
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with Mucopolysaccharidosis Type IIIA (MPS IIIA).
Product Name : JR-441
Product Type : Other Large Molecule
Upfront Cash : Inapplicable
December 15, 2023
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.
Product Name : JR-441
Product Type : Other Large Molecule
Upfront Cash : Inapplicable
October 11, 2023
Lead Product(s) : JR-441
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Lepunafusp Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : JR-171 (lepunafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in subjects with MPS I.
Product Name : JR-171
Product Type : Enzyme
Upfront Cash : Inapplicable
September 29, 2023
Lead Product(s) : Lepunafusp Alpha
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : AT845
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, delivers a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).
Product Name : AT845
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
February 22, 2023
Lead Product(s) : AT845
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : AT845
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, delivers a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).
Product Name : AT845
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
January 20, 2023
Lead Product(s) : AT845
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : AT845
Therapeutic Area : Genetic Disease
Study Phase : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, to deliver a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).
Product Name : AT845
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
July 02, 2022
Lead Product(s) : AT845
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I/ Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable