Drugs in Dev. Genetic Disease
Phase I
Denmark 
Lead Product(s) : Cagrilintide
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Cagrilintide is a Peptide, Unconjugated drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Congenital Hyperinsulinism.
Product Name : Undisclosed
Product Type : Peptide, Unconjugated
Upfront Cash : Inapplicable
September 19, 2025
Lead Product(s) : Cagrilintide
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : INV-347
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Safety Follow-up Study of Participants Dosed With INV-347 in Study INV-CL-108
Details : INV-347 is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Congenital Hyperinsulinism.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
September 03, 2025
Lead Product(s) : INV-347
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Digoxin
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants
Details : Digoxin is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Anemia, Sickle Cell.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
February 07, 2025
Lead Product(s) : Digoxin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : NNC0442-0344
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy Men
Details : NNC0442-0344 is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
October 21, 2024
Lead Product(s) : NNC0442-0344
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etavopivat
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants
Details : Etavopivat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Anemia, Sickle Cell.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
September 03, 2024
Lead Product(s) : Etavopivat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : NMD670
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
NMD Pharma Receives IND Clearance from FDA to Start Phase 2 Trial of NMD670 for CMT
Details : NMD670 is a first-in-class small molecule inhibitor of ClC-1. It is being developed by NMD Pharma for the treatment of patients with charcot-marie-tooth disease.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
June 18, 2024
Lead Product(s) : NMD670
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Etavopivat
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Research Study of the Effect of Food on Etavopivat in Healthy Participants
Details : Etavopivat is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Anemia, Sickle Cell.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
May 30, 2024
Lead Product(s) : Etavopivat
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : NNC0365-3769
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : NNC0365-3769 is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Antibody, Unconjugated
Upfront Cash : Inapplicable
January 12, 2023
Lead Product(s) : NNC0365-3769
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Mim8 B
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
A Research Study of How a New Medicine NNC0365-3769 (Mim8) Works in the Body of Healthy People
Details : Mim8 B is a drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemophilia A.
Product Name : Undisclosed
Product Type : Undisclosed
Upfront Cash : Inapplicable
November 19, 2021
Lead Product(s) : Mim8 B
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : Tesofensine,Metoprolol Tartrate
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Saniona Completes Submission of Manufacturing Data for Tesomet Capsules To U.S. FDA
Details : Tesomet capsules contain two active ingredients as microspheres of fixed-dose combination therapy of tesofensine (triple monoamine reuptake inhibitor) and metoprolol (a beta-1 selective blocker) used for treatment for hypothalamic obesity and Prader-Will...
Product Name : Tesomet
Product Type : Miscellaneous
Upfront Cash : Inapplicable
October 18, 2021
Lead Product(s) : Tesofensine,Metoprolol Tartrate
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
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