The US Food and Drug Administration has granted Breakthrough Therapy Designation to biotech Spark Therapeutics and pharmacology giant Pfizer for the hemophilia B treatment SPK-9001. The designation allows expedited development and FDA review of the gene therapy currently undergoing a Phase 1/2 trial as a potential one-time therapy. The trial is recruiting patients.
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My eldest son, Julian, is 22. He is studying music at college. It’s his passion, and as I have a music education degree and my husband has a music degree, his course choice did not come as a surprise. With our recent move, Julian changed schools, and his…
CRISPR/Cas9 is a gene-editing technology that can be used to alter the genetic sequence of a specific gene inside a cell. Several preclinical studies have indicated that the molecular tools in CRISPR/Cas9 might be used to treat or even cure hemophilia.
This video, shared by Colleen, is all about a mobile app from Pfizer that helps hemophilia patients track bleeds, infusions and factor supply. It also allows those living with the disease to track their daily physical activities, steps and heart rate to help…
In the fall of 2023, I was at a work conference in Indianapolis and headed down to the hotel lobby to meet a colleague for lunch. I was excited, as we were going to eat my favorite food: Ethiopian! Suddenly, I felt a sharp, shooting pain in my hip. It…
Pfizer has partnered with Spark Therapeutics to launch a Phase 3 program to evaluate the safety and effectiveness of its factor IX replacement gene therapy for treating hemophilia B. Replacement of factor IX (FIX), the clotting protein lacking in hemophilia B, will be done via the fidanacogene elaparvovec gene…
A new collaboration between St. Jude Children’s Research Hospital and the World Federation of Hemophilia (WFH) is aimed at helping blood disorder patients in countries where they may not receive regular treatment. The partnership’s centerpiece will be a program to conduct a gene therapy clinical trial in…
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AGC Biologics to Produce Hemophilia B Clotting Factor That Catalyst Is Testing in Clinical Trials
AGC Biologics has agreed to produce the hemophilia B clotting therapy that Catalyst Biosciences is testing in clinical trials, the companies announced. The next step in testing CB 2679d will be a Phase 2b trial in the third quarter of 2018, according to Catalyst, which is based in South San Francisco.
South Korea has approved an Investigational New Drug (IND) application for Catalyst Biosciences’ experimental therapy CB 2679d/ISU304, a highly potent next-generation coagulation Factor IX variant for the treatment of hemophilia B. Catalyst focuses on developing medications for hematology conditions (blood disorders), which include coagulation factors that can prevent spontaneous…
Moving is challenging. Moving means packing, having to decide what to keep and what not to keep, learning a new place, having to find a new grocery store, a new hair and nail salon, a new healthcare provider. Doing all that this time offered an opportunity to remember and…