pipeline-prospector-insert-v1
X

Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development in UNITED STATES

A collaboration with SCORR Marketing

Loading...

All Data

Filters Filter refresh
×
FILTER DATA :
News Type filter
    Company filter
      Product Type filter
        Deal Size filter
          Upfront Payment filter
            refresh

            Product Type

            Companies

            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): 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): Dirocaftor,Posenacaftor,PTI-428

            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): Resamirigene bilparvovec

            Therapeutic Area: Genetic Disease

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

            Partner/Sponsor/Collaborator: Astellas Pharma

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Acquisition January 15, 2020

            Details:

            Combined company establishes to become a global leader in AAV-based medicines, with Audentes to operate as a wholly-owned subsidiary and Center of Excellence for Astellas' newly created Genetic Regulation.

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

            Contact the Supplier

            Lead Product(s): AVR-RD-02

            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 14, 2020

            Details:

            First patient consented in GAU-201, a global Phase 1/2 trial of AVR-RD-02 in Gaucher disease type 1.

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

            Contact the Supplier

            Lead Product(s): SHP656

            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 14, 2020

            Details:

            The results from this single-dose study indicate that polysialylation of rFVIII confers a half-life extension similar to that of approved extended half-life products that use PEG or Fc fusion.

            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

            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 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.