Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target
Lead Product(s) : Human Alpha1-Proteinase Inhibitor
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
CSL Behring Expands Commitment to Alpha-1 Community with ZEMAIRA® 4- and 5-Gram Vials
Details : Zemaira is a human alpha1-proteinase inhibitor approved for augmentation and maintenance therapy in adults with Alpha-1 Antitrypsin Deficiency.
Brand Name : Zemaira
Molecule Type : Large molecule
Upfront Cash : Not Applicable
January 02, 2024
Lead Product(s) : Human Alpha1-Proteinase Inhibitor
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Etranacogene Dezaparvovec-drlb
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 Hemophil...
Brand Name : Hemgenix
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
June 20, 2023
Lead Product(s) : Etranacogene Dezaparvovec-drlb
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Etranacogene Dezaparvovec-drlb
Therapeutic Area : Genetic Disease
Study Phase : Approved
Recipient : Uniqure
Deal Size : $2,050.0 million
Deal Type : Licensing Agreement
uniQure Announces Achievement of $100 Million Milestone Related to Hemophilia B Gene Therapy
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...
Brand Name : Hemgenix
Molecule Type : Cell and Gene therapy
Upfront Cash : $450.0 million
June 20, 2023
Lead Product(s) : Etranacogene Dezaparvovec-drlb
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Recipient : Uniqure
Deal Size : $2,050.0 million
Deal Type : Licensing Agreement
Lead Product(s) : Immune Globulin G
Therapeutic Area : Immunology
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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 a...
Brand Name : Hizentra
Molecule Type : Large molecule
Upfront Cash : Not Applicable
April 18, 2023
Lead Product(s) : Immune Globulin G
Therapeutic Area : Immunology
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Etranacogene Dezaparvovec-drlb
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Orsini Specialty Pharmacy
Deal Size : Undisclosed
Deal Type : Partnership
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 hemor...
Brand Name : Hemgenix
Molecule Type : Cell and Gene therapy
Upfront Cash : Undisclosed
December 20, 2022
Lead Product(s) : Etranacogene Dezaparvovec-drlb
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Orsini Specialty Pharmacy
Deal Size : Undisclosed
Deal Type : Partnership
Lead Product(s) : Lyophilized Human C1-esterase
Therapeutic Area : Genetic Disease
Study Phase : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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).
Brand Name : Berinert
Molecule Type : Large molecule
Upfront Cash : Not Applicable
September 26, 2022
Lead Product(s) : Lyophilized Human C1-esterase
Therapeutic Area : Genetic Disease
Highest Development Status : Approved
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
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.
Brand Name : AMT-061
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
February 04, 2022
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Not Applicable
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Recipient : Uniqure
Deal Size : Not Applicable
Deal Type : Not Applicable
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).
Brand Name : AMT-061
Molecule Type : Cell and Gene therapy
Upfront Cash : Not Applicable
December 09, 2021
Lead Product(s) : Etranacogene Dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Recipient : Uniqure
Deal Size : Not Applicable
Deal Type : Not Applicable
Lead Product(s) : Human Plasma-derived Polyvalent Immunoglobulin G
Therapeutic Area : Pulmonary/Respiratory Diseases
Study Phase : Phase I
Sponsor : HCmed Innovations
Deal Size : Undisclosed
Deal Type : Collaboration
HCmed Reaches Milestone in Immunoglobin Combination Product Development with CSL Behring
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.
Brand Name : CSL787
Molecule Type : Large molecule
Upfront Cash : Undisclosed
October 24, 2021
Lead Product(s) : Human Plasma-derived Polyvalent Immunoglobulin G
Therapeutic Area : Pulmonary/Respiratory Diseases
Highest Development Status : Phase I
Sponsor : HCmed Innovations
Deal Size : Undisclosed
Deal Type : Collaboration
Lead Product(s) : Etranacogene dezaparvovec
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Recipient : Uniqure
Deal Size : $2,050.0 million
Deal Type : Licensing Agreement
UniQure Announces Closing of Commercialization and License Agreement With CSL Behring
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...
Brand Name : AMT-061
Molecule Type : Cell and Gene therapy
Upfront Cash : $450.0 million
May 06, 2021
Lead Product(s) : Etranacogene dezaparvovec
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Recipient : Uniqure
Deal Size : $2,050.0 million
Deal Type : Licensing Agreement
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