Lead Product(s) : Elocalcitol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Adge Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Partnership
Details : Adge has secured a global license to RO269228, a clinical stage oral small molecule originally developed by Roche for osteoporosis. RO269228, also known as elocalcitol, is a vitamin D analog which has demonstrated efficacy and safety in multiple Phase 2 ...
Product Name : Undisclosed
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
April 25, 2022
Lead Product(s) : Elocalcitol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Adge Pharmaceuticals
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : Ribitol
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
BridgeBio Pharma Announces Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)
Details : 43% increase in ratio of glycosylated alpha-dystroglycan to total αDG from baseline were measured for BBP-418, across all three dosing cohorts, signifying the oral therapy has potential to address both the root cause of LGMD2i and drive functional impro...
Product Name : Undisclosed
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
March 14, 2022
Lead Product(s) : Ribitol
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : HM15136
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Undisclosed
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
February 01, 2021
Lead Product(s) : HM15136
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Immunoglobulin G
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Safety and Efficacy of IGIV 10% in Patients With Autoimmune Encephalitis:
Details : Undisclosed
Product Name : Undisclosed
Product Type : Antibody
Upfront Cash : Inapplicable
November 25, 2019
Lead Product(s) : Immunoglobulin G
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Hepastem
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients
Details : Undisclosed
Product Name : Undisclosed
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
March 21, 2019
Lead Product(s) : Hepastem
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Thalidomide
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : CW Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Thalidomide/Dexamethasone Treatment And PET Evaluation In Organ Involvemenet of Cardiac Amyloidosis
Details : Undisclosed
Product Name : Undisclosed
Product Type : Other Small Molecule
Upfront Cash : Inapplicable
November 17, 2016
Lead Product(s) : Thalidomide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : CW Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : HL2351
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Undisclosed
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
August 02, 2016
Lead Product(s) : HL2351
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Idursulfase Beta
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
Details : Undisclosed
Product Name : Undisclosed
Product Type : Enzyme
Upfront Cash : Inapplicable
January 26, 2016
Lead Product(s) : Idursulfase Beta
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable