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Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Lead Product(s): Vosoritide
Therapeutic Area: Genetic Disease Product Name: Voxzogo
Highest Development Status: ApprovedProduct Type: Peptide
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 20, 2023
Details:
Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Lead Product(s): Vosoritide
Therapeutic Area: Genetic Disease Product Name: Voxzogo
Highest Development Status: Phase IIIProduct Type: Peptide
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 15, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec-rvox
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Recipient: Orsini Specialty Pharmacy
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 11, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 29, 2023
Details:
Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Lead Product(s): Vosoritide
Therapeutic Area: Genetic Disease Product Name: Voxzogo
Highest Development Status: ApprovedProduct Type: Peptide
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 07, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 06, 2023
Details:
Roctavian (valoctocogene roxaparvovec) is a gene therapy that uses an adeno-associated virus 5, codes for human Factor VIII, together with a human liver-specific promoter. It is currently being investigated for adults with severe hemophilia A.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 09, 2023
Details:
RMAT is an expedited program intended to facilitate development and review of regenerative medicine therapies, such as valoctocogene roxaparvovec, that are expected to address an unmet medical need in patients with serious conditions.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 23, 2022
Details:
Roctavian (valoctocogene roxaparvovec) also received orphan drug designation from the EMA and FDA for severe hemophilia A. Phase 1/2 study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with severe hemophilia A with active or prior Factor VIII inhibitors.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 09, 2022
Details:
The GENEr8-1 Phase 3 study for valoctocogene roxaparvovec demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate.
Lead Product(s): Valoctocogene Roxaparvovec
Therapeutic Area: Genetic Disease Product Name: Roctavian
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 12, 2022
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