Pipeline-Prospector-Insert
X

Find drug development pipeline and deal Updates information

A collaboration with SCORR Marketing

Loading...

All Data

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

            Product Type

            Development Status

            Company Locations

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

            Contact the Supplier

            Lead Product(s): Olipudase alfa

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II/ Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

            Details:

            Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

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

            Contact the Supplier

            Lead Product(s): RTX-134

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 30, 2020

            Details:

            The trial is designed to evaluate the safety and pharmacokinetics of RTX-134 in adult patients with PKU.

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

            Contact the Supplier

            Lead Product(s): RP-L201

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Lonza

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Partnership January 30, 2020

            Details:

            The agreement covers development and manufacturing of RP-L201, a phase 1/2 candidate for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).

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

            Contact the Supplier

            Lead Product(s): OTL-102

            Therapeutic Area: Genetic Disease

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 29, 2020

            Details:

            Early clinical data support ex vivo hematopoietic stem cell gene therapy as a potentially promising treatment option for X-CGD.

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

            Contact the Supplier

            Lead Product(s): Isotretinoin

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Timber Pharmaceuticals

            Deal Size: $20.0 million Upfront Cash: Undisclosed

            Deal Type: Merger January 28, 2020

            Details:

            Under the terms of the merger agreement, a wholly-owned subsidiary of BioPharmX will be merged with and into Timber, with Timber surviving the merger as a wholly-owned subsidiary of BioPharmX.

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

            Contact the Supplier

            Lead Product(s): Sonlicromanol

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II 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:

            The study’s primary objective is to evaluate the dose-effect of Sonlicromanol on the attention domain score of cognitive functioning, Results are expected in the second half of 2020.

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

            Contact the Supplier

            Lead Product(s): Adeno-associated virus-based gene therapy

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 23, 2020

            Details:

            Encouraging preliminary signs of biologic activity and a favorable safety profile are outcomes that are clinically relevant to patients.

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

            Contact the Supplier

            Lead Product(s): ARU-1801

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 22, 2020

            Details:

            Aruvant's ARU-1801, received FDA Orphan Drug designation for its efficacy to deliver a highly potent gene payload with enhanced anti-sickling properties, for a Reduced Intensity Conditioning regimen.

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

            Contact the Supplier

            Lead Product(s): Dirocaftor, Nesolicaftor

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I/ Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 22, 2020

            Details:

            The organoid system is a European Commission-funded global project that has attracted a selected number of drug developers and leading CF experts to create a blueprint for customized CF therapy.

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

            Contact the Supplier

            Lead Product(s): Trehalose

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 22, 2020

            Details:

            Seelos seeks scientific advice and protocol assistance for a European clinical study of SLS-005 in patients with Sanfillipo syndrome type A and B from EMA.

            PharmaCompass