Rare Diseases and Disorders

Sanifit

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First patient dosed in pivotal phase 3 trial of SNF472 in calciphylaxis

Lead Product(s): SNF472

Therapeutic Area: Rare Diseases and Disorders

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.

Inovio Pharmaceuticals

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Inovio Receives Authorization from FDA To Begin Trial for INO-3107

Lead Product(s): INO-3107

Therapeutic Area: Rare Diseases and Disorders

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.

Inovio Pharmaceuticals

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Inovio Publishes Patient Benefit Results From A Pilot Clinical Study

Lead Product(s): INO-3106

Therapeutic Area: Rare Diseases and Disorders

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.

Epygenix Therapeutics

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Epygenix Therapeutics Successfully Completes EPX-100 Phase I, Placebo-Controlled, Double-Blind, 2-Period Study and Ready to Phase 2 Studies

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.

Emendo Biotherapeutics

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Emendo Biotherapeutics Raises $61 Million to Advance Next Generation Genome Editing Therapeutics

Lead Product(s): OMNI nuclease

Therapeutic Area: Rare Diseases and Disorders

Highest Development Status: Undisclosed Product Type: 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.

Ideaya Biosciences

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

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.

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Development Status

Sanifit

First patient dosed in pivotal phase 3 trial of SNF472 in calciphylaxis

Inovio Pharmaceuticals

Inovio Receives Authorization from FDA To Begin Trial for INO-3107

Inovio Pharmaceuticals

Inovio Publishes Patient Benefit Results From A Pilot Clinical Study

Epygenix Therapeutics

Epygenix Therapeutics Successfully Completes EPX-100 Phase I, Placebo-Controlled, Double-Blind, 2-Period Study and Ready to Phase 2 Studies

Emendo Biotherapeutics

Emendo Biotherapeutics Raises $61 Million to Advance Next Generation Genome Editing Therapeutics

Ideaya Biosciences

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