gmp
gmp

Find Clinical Drug Pipeline Developments & Deals by Ultragenyx Pharmaceutical

Loading...

All Data

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

            Companies By Therapeutic Area 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): BAY2599023

            Therapeutic Area: Genetic Disease Product Name: DTX201

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Recipient: Bayer HealthCare

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 24, 2020

            Details:

            Two of the six patients treated to date in this clinical study, both given the therapy at lowest dose, are still showing FVIII activity at more than one year post-treatment, its investigators report.

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

            Contact the Supplier

            Lead Product(s): Triheptanoin

            Therapeutic Area: Genetic Disease Product Name: Dojolvi

            Highest Development Status: Approved Product Type: Small molecule

            Recipient: Orsini Pharmaceutical

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership July 10, 2020

            Details:

            Orsini Pharmaceutical Services has been selected by Ultragenyx Pharmaceutical as a limited distribution specialty pharmacy partner for Dojolvi (triheptanoin). Dojolvi™ is indicated as a source of calories and fatty acids for the treatment of molecularly confirmed LC-FAOD.

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

            Contact the Supplier

            Lead Product(s): DTX301

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 13, 2020

            Details:

            Six of nine patients in the study have responded to the gene therapy, DTX301, including all three patients in Cohort 3 who are now confirmed responders.

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

            Contact the Supplier

            Lead Product(s): AAV-based gene therapy

            Therapeutic Area: Oncology Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Daiichi Sankyo Co Ltd

            Deal Size: $225.0 million Upfront Cash: $125.0 million

            Deal Type: Licensing Agreement April 01, 2020

            Details:

            Ultragenyx granted Daiichi Sankyo license to intellectual property, including know-how and patent applications, with respect to its HeLa manufacturing platform for AAV-based gene therapy products.

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

            Contact the Supplier

            Lead Product(s): Gene Therapy-AAV8

            Therapeutic Area: Endocrinology Product Name: Undisclosed

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

            Recipient: Regenxbio

            Deal Size: Undisclosed Upfront Cash: $7.0 million

            Deal Type: Licensing Agreement March 31, 2020

            Details:

            Ultragenyx has been granted an exclusive, worldwide license, with rights to sublicense REGENXBIO's NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments.

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

            Contact the Supplier

            Lead Product(s): Burosumab

            Therapeutic Area: Musculoskeletal Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 14, 2020

            Details:

            The sBLA package includes data from two single-arm Phase 2 studies including 14 adult patients conducted by Ultragenyx in the U.S. and 13 adult patients conducted by Kyowa Kirin in Japan.

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

            Contact the Supplier

            Lead Product(s): DTX301

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 09, 2020

            Details:

            The company reported more uniform response in Cohort 3 with 2 confirmed responders and 1 potential responder. In cohort 2, 1 female patient has newly demonstrated a response starting at Week 52.