Drugs in Dev. Genetic Disease
Phase I
Sweden 
Lead Product(s) : Efanesoctocog Alfa
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : PSI CRO
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Protein
Upfront Cash : Inapplicable
August 30, 2024
Lead Product(s) : Efanesoctocog Alfa
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : PSI CRO
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : KL1333
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Abliva receives positive FDA feedback on its KL1333 development plant
Details : KL1333 is a potent modulator of the cellular levels of NAD, a central co-enzyme in the cell’s energy metabolism. The clinical KL1333 development plan will target patients with genetically confirmed MIDD-MELAS or KSS-CPEO spectrum disorders with multi-o...
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
July 10, 2020
Lead Product(s) : KL1333
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Melphalan Flufenamide
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : PRA Health Sciences
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Melflufen (Melphalan Flufenamide) is a Peptide drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Immunoglobulin Light-chain Amyloidosis.
Product Name : Melflufen
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
October 04, 2019
Lead Product(s) : Melphalan Flufenamide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : PRA Health Sciences
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Nitisinone
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Taste and Palatability of Orfadin Suspension
Details : Nitisinone is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Tyrosinemias.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
November 28, 2012
Lead Product(s) : Nitisinone
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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