Find Clinical Drug Pipelines for Rare Diseases and Disorders

Lead Product(s): Clemizole hydrochloride

Therapeutic Area: Rare Diseases and Disorders

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.

Lead Product(s): OMNI nuclease

Therapeutic Area: Rare Diseases and Disorders

Highest Development Status: Undisclosed Product Type: Large molecule

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.

Lead Product(s): IDE196

Therapeutic Area: Rare Diseases and Disorders

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.