Wavelength-Pharmaceuticals-Insert
X

Find Clinical Drug Pipelines for Rare Diseases and Disorders

Loading...

All Data

Filters Filter refresh
×
FILTER DATA :
Study Phase filter
    Country filter
      News Type filter
        Company filter
          Product Type filter
            refresh

            Product Type Full Screen

            Development Status Full Screen

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

            Contact the Supplier

            Lead Product(s): RBCEV-based gene therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Carmine Therapeutics

            Deal Size: $900.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration June 30, 2020

            Details:

            The collaboration will leverage Carmine’s REGENT technology, which is based on red blood cell extracellular vesicles, allowing for repeat dosing, enhanced bio-distribution and larger transgene payload capacity compared to adeno-associated virus-based gene therapy.

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

            Contact the Supplier

            Lead Product(s): SNF472

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 13, 2020

            Details:

            CALCIPHYX, is designed to assess the effect of SNF472 when added to background care for the treatment of calciphylaxis patients with end stage kidney disease being treated with hemodialysis.

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

            Contact the Supplier

            Lead Product(s): INO-3107

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Vaccine

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2020

            Details:

            The open-label, multicenter Phase 1/2 trial will enroll approximately 63 subjects in the U.S. and will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107.

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

            Contact the Supplier

            Lead Product(s): INO-3106

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Vaccine

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 03, 2020

            Details:

            Study results demonstrated that INO-3106 generated immunogenicity and engagement and expansion of an HPV 6-specific cellular response, including cytotoxic T cells.

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

            Contact the Supplier

            Lead Product(s): Clemizole hydrochloride

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 27, 2020

            Details:

            This Phase 1 study successfully confirmed that EPX-100 is safe, well-tolerated, and does not show any clinically significant abnormalities among equal numbers of male and female subjects.

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

            Contact the Supplier

            Lead Product(s): OMNI nuclease

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Anges

            Deal Size: $61.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing January 15, 2020

            Details:

            The funding will accelerate novel OMNI gene editing platform into a broad therapeutic product pipeline.

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

            Contact the Supplier

            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.