XTalks

JANUARY 28, 2022

Up to 50 percent of patients will develop metastatic disease, mainly spreading to the liver, making treatment difficult and limited. Kimmtrak has a price tag of $18,760 per vial, which amounts to a weekly dose. Kimmtrak has also become the first bispecific T cell engager to be FDA-approved for the treatment of a solid tumor.

FDA Approval 98

FDA Approval Development Licensing Licensing 98

pharmaphorum

FEBRUARY 22, 2021

The case comes just weeks after UniQure halted a trial of its haemophilia B gene therapy AMT-061 after an unexpected case of liver cancer. News of the pause came as UK cost-effectiveness watchdog NICE rejected Zynteglo for regular NHS use in first draft guidance, despite a confidential discount to its price tag of around €1.57

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

Delveinsight

FEBRUARY 25, 2021

The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs). DNA hypomethylating agents are epigenetic drugs that eliminate specific chemical tags on DNA, hence turning specific genes on and off and changing the cells’ function.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

NOVEMBER 24, 2022

That can now be replaced with one injection of Hemgenix, which will enable patients to produce it continuously on their own in the liver. Conventional treatment for hemophilia B involves prophylactic infusions of factor IX replacement therapy.

Gene Therapy 52

Gene Therapy Gene FDA Approval Genetics 52

XTalks

DECEMBER 21, 2022

The multi-million-dollar price tags are largely attributable to the high R&D and manufacturing costs associated with making a gene therapy, as the raw materials for producing the therapy can be expensive. Currently, CSL Behring’s one-dose Hemophilia B gene therapy Hemgenix holds the title at $3.5 million for a single infusion.

Life Science 52

Life Science Gene Therapy Gene Clinical Trials 52

Pharmaceutical Technology

NOVEMBER 30, 2022

High rates of obesity place a major burden on healthcare systems as the condition is associated with many major health complications like type 2 diabetes, hypertension, non-alcoholic fatty liver disease, and other bodily dysfunctions. In the US, obesity care is estimated at to cost $147 billion in healthcare spending annually.

Drugs 264

Drugs Manufacturing Hormones Contract Manufacturing 264

Pharmaceutical Technology

NOVEMBER 30, 2022

High rates of obesity place a major burden on healthcare systems as the condition is associated with many major health complications like type 2 diabetes, hypertension, non-alcoholic fatty liver disease, and other bodily dysfunctions. In the US, obesity care is estimated at to cost $147 billion in healthcare spending annually.

Drugs 246

Drugs Manufacturing Hormones Contract Manufacturing 246