In Depth 4 Apr 2024
Seven diseases CRISPR technology could cure
Here are some of the diseases that scientists aim to tackle using CRISPR technology, testing its possibilities and limits as a medical tool.
Blood disorders
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News and Trends 25 Jul 2023
Roche and Alnylam to work on RNAi therapeutic to treat hypertension
Roche has announced a partnership with Alnylam to develop and commercialize zilebesiran, an investigational RNAi therapeutic currently in phase 2 for the treatment of hypertension. Hypertension affects more than 1.2 billion adults worldwide and is the main, independent, preventable risk factor for death and disability, with approximately 80% of the adults with hypertension worldwide not […]
News and Trends 2 Jun 2023
von Willebrand disease treatment gets FDA orphan drug designation
The U.S. Food and Drug Administration (FDA) has granted Vega Therapeutics, Inc. orphan drug designation for its antibody therapy, VGA039, for the treatment of the rare bleeding disorder, von Willebrand disease (VWD). Vega Therapeutics is a clinical stage biotechnology company developing novel therapies for rare blood disorders. VGA039 is a first-in-class antibody therapy with a […]
News and Trends 8 May 2023
How thalassemia gene therapy approval is making a difference
A newly-approved gene therapy is transforming the lives of patients with beta thalassemia – an inherited blood disorder – by helping to stop or significantly reduce their reliance on blood transfusions, says an expert from a leading global health system, Cleveland Clinic, on International Thalassemia Day on May 8. A gene therapy known as betibeglogene […]
In Depth 18 Apr 2023
Gene therapy: a powerful tool for hemophilia B?
The approval of HEMGENIX, the first-ever gene therapy for hemophilia B, by the U.S. Food and Drug Administration (FDA) followed by the European Commission, has been momentous in rare disease therapeutic research, paving the way for various gene therapies that are currently being studied. As we observed World Hemophilia Day on April 17, let us […]
News and Trends 21 Feb 2023
Hemab Therapeutics receives $135M to address bleeding and thrombotic disorders
Hemab Therapeutics, a clinical-stage biotechnology company developing the first prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, has announced the closing of an oversubscribed $135 million Series B financing. Access Biotechnology led the round, with participation from new investors Deep Track Capital, Avoro Ventures, Invus, Rock Springs Capital, and Maj Invest Equity, as well […]
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News and Trends 14 Feb 2023
Disc Medicine raises $62.5M for hematology programs
Disc Medicine, Inc., a U.S. clinical-stage biopharma company focused on the discovery, development and commercialization of treatments for patients suffering from serious hematologic diseases, has entered into a securities purchase agreement with investors resulting in an aggregate $62.5 million in gross proceeds through a registered direct offering. The financing was led by Bain Capital Life […]
Interview 26 Jan 2023
Scopio on the future of telehematology
Through full-field digital cell morphology, Scopio Labs’ technology can automate the analysis of tens of thousands of cells at a time. This could mean bringing earlier detection and diagnosis of cancers, infections, and other diseases, expediting patients’ access to better care and life-saving treatments. Erez Na’aman, co-founder and CTO of Scopio Labs, tells us about […]
News and Trends 26 Jan 2023
Milestone for gene-edited therapy for sickle cell disease
Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics have announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application of exa-cel for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The submission is supported by two global phase 3 studies investigating exa-cel as a potential one-time therapy for people with […]
News and Trends 20 Dec 2022
Watch: Sernova’s Cell Pouch System tackles diabetes
Sernova Corp. is a Canadian-based clinical-stage biotechnology company that is developing its Cell Pouch System. This is a novel implantable and scalable medical device with immune protected therapeutic cells, for the treatment of chronic diseases such as insulin-dependent diabetes, thyroid disease, and blood disorders that include hemophilia A. On implantation, the Cell Pouch forms a […]
News and Trends 16 Dec 2022
CSL closer to gene therapy treatments for hemophilia B patients in Europe
CSL says the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec. Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds with a single infusion. […]
News and Trends 13 Dec 2022
Blood clotting research holds hope for sepsis
Researchers from the University of Birmingham in the U.K., who identified a novel mechanism for platelet activation in pathogenic blood clotting (thrombosis), are now turning their attention to sepsis. Identified by associate professor Julie Rayes and Martina Colicchia from the Birmingham Platelet Group, and described in a recent paper in Blood, this previously unknown axis […]
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