U.S. Food and Drug Administration approves CSL’s HEMGENIX® (etranacogene dezaparvovec-drlb), the first gene therapy for hemophilia B

  • This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion
  • With the approval of HEMGENIX, CSL now offers an even more comprehensive portfolio of treatments for people living with hemophilia B, ushering in a new era of treatment options 


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Ultrasound activated microbubbles enhance the activity of standard of care therapeutics in pancreatic cancer

PHOENIX, Arizona and OSLO, Norway — November 21, 2022 — Scientists at the Phoenix-based Translational Genomics Research Institute (TGen), part of City of Hope, and EXACT Therapeutics AS (“EXACT-Tx”, Euronext Growth: EXTX), a Norwegian clinical stage precision health company, today report that the ultrasound based Acoustic Cluster Therapy (ACT®) significantly enhanced the antitumor efficacy of standard care therapies in a preclinical model for pancreatic cancer.Continue reading

Researchers develop drug to treat Alzheimer’s disease in Down syndrome patients

When a baby is born, each cell in his or her body contains 23 pairs of chromosomes or 46 chromosomes in total, with half coming from each parent. At least, this is the case most of the time.

Yet, the biological machinery responsible for assuring proper chromosome distribution sometimes goes awry. This can result in a range of serious genetic diseases, the most common of which is Down syndrome. In this case, an extra copy of chromosome 21 is inherited from one parent. The condition is also known as Trisomy 21.Continue reading