Promoted Content
Promoted Content

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

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 Full Screen

            Companies Full Screen

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

            Contact the Supplier

            Lead Product(s): REN001

            Therapeutic Area: Genetic Disease Product Name: REN001

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

            Partner/Sponsor/Collaborator: Novo Ventures

            Deal Size: $95.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing December 09, 2020

            Details:

            Funds will support developments of REN001 which is an oral, once-daily investigational drug known to control several genes involved in mitochondrial activity.

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

            Contact the Supplier

            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

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

            Partner/Sponsor/Collaborator: Jefferies

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering December 08, 2020

            Details:

            Funds to be used in the development of its lead program FT-4202 in sickle cell disease and completion of Phase 1 clinical trial, advancement of FT-7051 in metastatic castration-resistant prostate cancer, pre-approval activities for FT-2102 in AML.

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

            Contact the Supplier

            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 07, 2020

            Details:

            FT-4202 is a novel, investigational, selective red blood cell pyruvate kinase-R activator in development as a potential disease-modifying therapy for SCD. 6 of 7 (86%) patients on 300 mg of FT-4202 for 14 days achieved a hemoglobin increase > 1 g/dL from baseline.

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

            Contact the Supplier

            Lead Product(s): ARO-AAT

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

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

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $1,040.0 million Upfront Cash: $300.0 million

            Deal Type: Licensing Agreement November 24, 2020

            Details:

            Under the terms of the agreement, Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the United States under a 50/50 profit-sharing structure.

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

            Contact the Supplier

            Lead Product(s): RTA 402

            Therapeutic Area: Genetic Disease Product Name: RTA 402

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 09, 2020

            Details:

            Bardoxolone achieved the year 2 primary and key secondary endpoints with statistically significant improvements in EGFR as compared to placebo at week 100 and week 104 .

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

            Contact the Supplier

            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: FT-4202

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 04, 2020

            Details:

            The oral presentation will feature clinical data from the multiple ascending dose cohort of a randomized, multi-center, placebo-controlled Phase 1 trial of FT-4202 in people living with sickle cell disease (SCD).

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

            Contact the Supplier

            Lead Product(s): Elamipretide

            Therapeutic Area: Genetic Disease Product Name: MTP-131

            Highest Development Status: Phase II/ Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 19, 2020

            Details:

            The poster titled "TAZPOWER Analysis: Elamipretide Significantly Improves Disease Symptomatology versus Natural History Controls in Barth Syndrome," shows the importance of longitudinal natural history data in understanding and validating clinical trial outcomes for Barth.

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

            Contact the Supplier

            Lead Product(s): Cannabidiol

            Therapeutic Area: Genetic Disease Product Name: Zygel

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 15, 2020

            Details:

            New data demonstrate that in patients diagnosed with FXS with a fully methylated FMR1 gene significantly more patients who received Zygel achieved a clinically meaningful improvement in their behavioral symptoms compared to patients who received placebo.

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

            Contact the Supplier

            Lead Product(s): ARO-AAT

            Therapeutic Area: Genetic Disease Product Name: ARO-AAT

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

            Partner/Sponsor/Collaborator: Takeda Pharmaceutical

            Deal Size: $1,040.0 million Upfront Cash: $300.0 million

            Deal Type: Collaboration October 08, 2020

            Details:

            Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the U.S under a 50/50 profit-sharing structure. Outside the U.S., Takeda will lead the global marketing strategy and receive an exclusive license to commercialize ARO-AAT.

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

            Contact the Supplier

            Lead Product(s): Risdiplam

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Royalty Pharma

            Deal Size: $650.0 million Upfront Cash: $650.0 million

            Deal Type: Acquisition July 20, 2020

            Details:

            Risdiplam, to be marketed by Roche, is an investigational, orally administered survival motor neuron-2 (SMN2) splicing modifier for the treatment of spinal muscular atrophy (SMA).