X
Upload your Marketing & Sales content on your company Virtual Booth, click HERE.
Details:
Zemaira (human alpha1-proteinase inhibitor) is a highly purified form of AAT (human) currently approved for chronic augmentation and maintenance therapy in adults with Alpha 1 Antitrypsin Deficiency and clinical evidence of emphysema.
Lead Product(s): Human Alpha1-Proteinase Inhibitor
Therapeutic Area: Genetic Disease Product Name: Zemaira
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 02, 2024
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: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 20, 2023
Details:
The licensing agreement provided CSL Behring with exclusive global rights to Hemgenix (etranacogene dezaparvovec), uniQure’s first approved gene therapy for hemophilia B in the United States, EU and European Economic Area, and the UK, for patients with hemophilia B.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Recipient: Uniqure
Deal Size: $2,050.0 million Upfront Cash: $450.0 million
Deal Type: Licensing Agreement June 20, 2023
Details:
Hizentra (immune globulin subcutaneous [human] 20% liquid) supplies a broad spectrum of opsonizing and neutralizing IgG antibodies against a wide variety of bacterial and viral agents. It is indicated primary immunodeficiency and maintenance therapy in adults with CIDP.
Lead Product(s): Immune Globulin G
Therapeutic Area: Immunology Product Name: Hizentra
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable April 18, 2023
Details:
Hemgenix (etranacogene dezaparvovec-drlb) is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B who Currently use Factor IX prophylaxis therapy, or Have current or historical life-threatening hemorrhage.
Lead Product(s): Etranacogene Dezaparvovec-drlb
Therapeutic Area: Genetic Disease Product Name: Hemgenix
Highest Development Status: ApprovedProduct Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Orsini Specialty Pharmacy
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Partnership December 20, 2022
Details:
In the COMPACT study, acute HAE attacks were significantly lower in the "Berinert"(Lyophilized Human C1-esterase) 60 IU/kg group at 0.52 per month compared to 4.03 per month in the placebo group (p<0.001, mixed model).
Lead Product(s): Lyophilized Human C1-esterase
Therapeutic Area: Genetic Disease Product Name: Berinert
Highest Development Status: ApprovedProduct Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 26, 2022
Details:
Data from the largest gene therapy study in Hemophilia B to date shows that etranacogene dezaparvovec is statistically superior in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy.
Lead Product(s): Etranacogene Dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
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 February 04, 2022
Details:
Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable FIX expression, assessed at 18 months following a single dose of AMT-061(etranacogene dezaparvovec).
Lead Product(s): Etranacogene Dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Recipient: Uniqure
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 09, 2021
Details:
CSL Behring partnered with HCmed to develop a combination product consisting of a plasma-derived immunoglobulin formulation (CSL787) administered via a customized version of the AdheResp nebulizer, HCmed's breath-actuated mesh nebulizer.
Lead Product(s): Human Plasma-derived Polyvalent Immunoglobulin G
Therapeutic Area: Pulmonary/Respiratory Diseases Product Name: CSL787
Highest Development Status: Phase IProduct Type: Large molecule
Partner/Sponsor/Collaborator: HCmed Innovations
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Collaboration October 24, 2021
Details:
uniQure announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec expired on May 5, 2021, and the agreement became fully effective on May 6, 2021.
Lead Product(s): Etranacogene dezaparvovec
Therapeutic Area: Genetic Disease Product Name: AMT-061
Highest Development Status: Phase IIIProduct Type: Cell and Gene therapy
Recipient: Uniqure
Deal Size: $2,050.0 million Upfront Cash: $450.0 million
Deal Type: Licensing Agreement May 06, 2021
FULL SCREEN VIEW Click here to open all results in a new tab [this preview display 10 results] 
Market Place
