Blood disorders Archives

News and Trends 9 Jan 2025

Von Willebrand disease: Star Therapeutics, Chugai and Band Therapeutics are breaking new ground

Explore advances in von Willebrand disease treatment, including Star Therapeutics’ VGA039, and other key players in the space.

January 9, 2025 - 7 minutesmins - By Jules Adam

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.

April 4, 2024 - 11 minutesmins - By Clara Rodríguez Fernández

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 […]

July 25, 2023 - 5 minutesmins - By Jim Cornall

blood bleeding disorders von Willebrand disease

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 […]

June 2, 2023 - 2 minutesmins - By Jim Cornall

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 […]

May 8, 2023 - 5 minutesmins - By Jim Cornall

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 […]

April 18, 2023 - 10 minutesmins - By Roohi Mariam Peter

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 […]

February 21, 2023 - 2 minutesmins - By Jim Cornall

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 […]

February 14, 2023 - 2 minutesmins - By Jim Cornall

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 […]

January 26, 2023 - 4 minutesmins - By Jim Cornall

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 […]

January 26, 2023 - 3 minutesmins - By Jim Cornall

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 […]

December 20, 2022 - 2 minutesmins - By Jim Cornall

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. […]

December 16, 2022 - 2 minutesmins - By Jim Cornall

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Blood disorders

Von Willebrand disease: Star Therapeutics, Chugai and Band Therapeutics are breaking new ground

Seven diseases CRISPR technology could cure

Roche and Alnylam to work on RNAi therapeutic to treat hypertension

von Willebrand disease treatment gets FDA orphan drug designation

How thalassemia gene therapy approval is making a difference

Gene therapy: a powerful tool for hemophilia B?

Hemab Therapeutics receives $135M to address bleeding and thrombotic disorders

Disc Medicine raises $62.5M for hematology programs

Scopio on the future of telehematology

Milestone for gene-edited therapy for sickle cell disease

Watch: Sernova’s Cell Pouch System tackles diabetes

CSL closer to gene therapy treatments for hemophilia B patients in Europe

Subscribe to our newsletter to get the latest biotech news!

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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