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Find Drugs for Rare Diseases and Disorders in Preclinical Development

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            Lead Product(s): SAN711

            Therapeutic Area: Rare Diseases and Disorders Product Name: SAN711

            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 May 12, 2021

            Details:

            SAN711 was designed using Saniona’s ion channel drug discovery engine to bind with high selectivity to the α3 subunit of the GABAA receptor, which in preclinical models has resulted in efficacy with fewer side effects.

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            Lead Product(s): AAV-based gene therapies

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: EcoR1 Capital

            Deal Size: $110.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing May 04, 2021

            Details:

            Proceeds to support continued development of proprietary platform for rationally designed adeno-associated virus vectors (AAV) and advance programs into the clinic.

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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.