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Lead Product(s): Engenious vectors,HSC gene therapy
Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed
Highest Development Status: Preclinical Product Type: Large molecule
Partner/Sponsor/Collaborator: 5AM Ventures
Deal Size: $70.0 million Upfront Cash: Undisclosed
Deal Type: Series A Financing February 11, 2021
Details:
Company’s Engenious™ vectors Delivered without Stem Cell Collection or Myeloablative Conditioning in Outpatient and Low-Resource Settings, Enabling Access Worldwide.
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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).
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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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