Rare Diseases and Disorders

Takeda Pharmaceutical

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

JAPAN Japan

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

Takeda Partners up with Carmine Therapeutics to Develop and Market Rare Disease Candidates

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.

Sanifit

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

United States

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

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

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.

Inovio Pharmaceuticals

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

United States

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

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

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.

Inovio Pharmaceuticals

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

United States

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

Inovio Publishes Patient Benefit Results From A Pilot Clinical Study

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.

Epygenix Therapeutics

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

United States

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

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 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.

Emendo Biotherapeutics

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

United States

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

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

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.

Ideaya Biosciences

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

United States

Post An Enquiry

Virtual Booth

CAMBODIA PHAR-MED 2020

Participation Not Confirmed

Update your events

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 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.

Find

Find Clinical Drug Pipelines for Rare Diseases and Disorders

Product Type

Development Status

Takeda Pharmaceutical

Takeda Partners up with Carmine Therapeutics to Develop and Market Rare Disease Candidates

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...