FDA-&-EMA-New-Drug Approvals(Mid-2020 Recap)-insert
X

Find Drugs for Genetic Disease in Phase II Clinical Development in UNITED STATES

Loading...

All Data

Filters Filter refresh
×
FILTER DATA :
News Type filter
    Company filter
      Product Type filter
        Deal Size filter
          Upfront Payment filter
            refresh

            Product Type Full Screen

            Companies Full Screen

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Infigratinib

            Therapeutic Area: Genetic Disease Product Name: BBP-831

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 15, 2020

            Details:

            First child with achondroplasia has been dosed with the investigational medicine infigratinib, an orally available small molecule, that targets the overactivity of fibroblast growth factor receptor 3 (FGFR3) in the PROPEL 2 Phase 2 clinical trial.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Marzeptacog alfa

            Therapeutic Area: Genetic Disease Product Name: MarzAA

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 13, 2020

            Details:

            Subcutaneous marzeptacog alfa (activated) (MarzAA) rapidly achieves and maintains therapeutic levels. Data confirm Phase 3 study design to treat acute bleeding events in hemophilia.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Allogeneic human retinal progenitor cells

            Therapeutic Area: Genetic Disease Product Name: jCell

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 07, 2020

            Details:

            Results from the Ph 2b trial of jCell in retinitis pigmentosa will be presented at the American Society of Retina Specialists. jCell is an investigational treatment for retinitis pigmentosa which has received FDA Regenerative Medicine Advanced Therapy designation.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Isotretinoin

            Therapeutic Area: Genetic Disease Product Name: TMB-001

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 01, 2020

            Details:

            U.S. and Australia participating in the Phase 2b CONTROL study evaluating TMB-001 (topical isotretinoin) are now initiated and patients with moderate to severe congenital ichthyosis (CI) are actively enrolling.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): KVD900

            Therapeutic Area: Genetic Disease Product Name: KVD900

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 01, 2020

            Details:

            The company has opened an Investigational New Drug (IND) Application for KVD900 with the U.S. Food and Drug Administration (FDA) to enable clinical development in the United States.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Arimoclomol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2020

            Details:

            Arimoclomol demonstrated a marked and clinically meaningful dose-dependent reduction in liver and spleen size. Data supports Orphazyme's intention to proceed with pivotal stage clinical development in Gaucher disease .

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Marzeptacog alfa

            Therapeutic Area: Genetic Disease Product Name: MarzAA

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2020

            Details:

            Data from Phase 1 study to evaluate the pharmacokinetics, pharmacodynamics, and safety of ascending doses of subcutaneous marzeptacog alfa (activated) in adult subjects with hemophilia, will be highlited.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): IMR-687

            Therapeutic Area: Genetic Disease Product Name: IMR-687

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 24, 2020

            Details:

            The U.S. FDA has granted Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassemia. The FDA previously granted Orphan Drug Designation for IMR-687 for the treatment of patients with sickle cell disease.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): Dalcinonacog alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 15, 2020

            Details:

            Data of trial showed that 28 days of daily SQ dosing of DalcA achieved protective target FIX levels of >12%, with FIX levels of up to 27% and a half-life of 2.5 to 5.1 days with no bleeds, demonstrating effective prophylaxis and the potential for lower or less frequent dosing.

            Virtual Booth Upgrade this company profile, update & customize all content// Ask PharmaCompass, click HERE

            Contact the Supplier

            Lead Product(s): SRP-9001

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 15, 2020

            Details:

            In study 101, four ambulatory participants between the ages of 4 and 7 were treated with an infusion of SRP-9001 at a dose of 2x1014 vg/kg. The therapy was safe and tolerable in all participants.

            PharmaCompass