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    Find Drugs for Rare Diseases and Disorders in Preclinical Development

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              Cerecor

              • Development Update

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              UNITED STATES United States

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              CFRT 2020

              Participation Not Confirmed

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              Cerecor Receives Orphan Drug Designation for CERC-006 in Lymphatic Malformations

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              Lead Product(s): CERC-006

              Therapeutic Area: Rare Diseases and Disorders Product Name: CERC-006

              Highest Development Status: Preclinical Product Type: Small molecule

              Partner/Sponsor/Collaborator: Calidi Biotherapeutics

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable August 03, 2020

              Details:

              U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to CERC-006, a dual inhibitor of mTOR complexes 1 and 2 for the treatment of lymphatic malformations (LM).

              Ideaya Biosciences

              • Development Update

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              UNITED STATES United States

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              CFRT 2020

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              IDEAYA Biosciences and Boston Children's Hospital Collaborate on Preclinical Evaluation of IDE196 for Sturge Weber Syndrome - a Rare Disease...

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              Lead Product(s): IDE196

              Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

              Highest Development Status: Preclinical Product Type: Small molecule

              Partner/Sponsor/Collaborator: Not Applicable

              Deal Size: Not Applicable Upfront Cash: Not Applicable

              Deal Type: Not Applicable January 10, 2020

              Details:

              Preclinical studies will test IDE196, in vitro, to determine whether endothelial cells with GNAQ mutations can recover normal cell function as well as in vivo pharmacological inhibition of PKC.

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