Details:
Hemgenix (etranacogene dezaparvovec-drlb) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua. Its single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 26, 2023
Details:
Hemgenix (etranacogene dezaparvovec) is an AAV5 based gene therapy designed to deliver a copy of a gene encoding hFIX-Padua. Its single IV infusion results in cell transduction and increase in circulating Factor IX activity in patients with Hemophilia B.
Lead Product(s): Etranacogene Dezaparvovec
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 20, 2023
Details:
Hemgenix (etranacogene dezaparvovec, also known as CSL222, previously known as AMT-061), is an adeno-associated virus five (AAV5)-based gene therapy given as a one-time treatment for moderately severe to severe hemophilia B patients.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 16, 2022
Details:
HEMGENIX (Etranacogene Dezaparvovec-drlb), is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 10, 2022
Details:
Hemgenix (etranacogene dezaparvovec-drlb) is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient associated virus (AAV).
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: Approved Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 22, 2022
Details:
As per agreement, Aptar Pharma will evaluate the commercial applications for the Orbital device & further develop the prototype device to meet unmet market needs. Pharmaxis retains the rights to devices containing Orbital intellectual property used to deliver inhaled mannitol.
Lead Product(s): Mannitol API
Therapeutic Area: Genetic Disease Product Name: Bronchitol
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Aptar Pharma
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Agreement August 17, 2021
Details:
CYSTADROPS demonstrated a significant reduction in cystine crystal deposits in the cornea of the eye and is the first and only FDA-approved cysteamine drop formulation with four times a day dosing.
Lead Product(s): Cysteamine Hydrochloride
Therapeutic Area: Genetic Disease Product Name: Cystadrops
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 25, 2020
Details:
Cystadrops (cysteamine) ophthalmic solution, acts as a cystine-depleting agent by converting cystine to cysteine and is indicated for the treatment of corneal cystine crystal deposits in adults and children with cystinosis.
Lead Product(s): Cysteamine Bitartrate
Therapeutic Area: Genetic Disease Product Name: Cystadrops
Highest Development Status: Approved Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable August 25, 2020