TDT

Unibet and Tour de Tietema launches heart health awareness campaign - 'Follow Your Heart'

Retrieved on: 
Wednesday, February 14, 2024

VALLETTA, Malta, Feb. 14, 2024 /PRNewswire/ -- Unibet in Belgium and the Netherlands launches a campaign with the Dutch cycling team Tour de Tietema to raise awareness and educate the public about heart health issues within the cycling community.

Key Points: 
  • VALLETTA, Malta, Feb. 14, 2024 /PRNewswire/ -- Unibet in Belgium and the Netherlands launches a campaign with the Dutch cycling team Tour de Tietema to raise awareness and educate the public about heart health issues within the cycling community.
  • The campaign is called 'Follow Your Heart'.
  • Kindred Group plc's (Kindred) flagship brand Unibet has, together with Tour de Tietema (TDT), developed an online learning tool in response to the growing concern over heart health issues within the cycling community.
  • In addition, the cycling team will wear a custom exclusive jersey to draw attention to the importance of heart health.

Unibet and Tour de Tietema launches heart health awareness campaign - 'Follow Your Heart'

Retrieved on: 
Wednesday, February 14, 2024

VALLETTA, Malta, Feb. 14, 2024 /PRNewswire/ -- Unibet in Belgium and the Netherlands launches a campaign with the Dutch cycling team Tour de Tietema to raise awareness and educate the public about heart health issues within the cycling community.

Key Points: 
  • VALLETTA, Malta, Feb. 14, 2024 /PRNewswire/ -- Unibet in Belgium and the Netherlands launches a campaign with the Dutch cycling team Tour de Tietema to raise awareness and educate the public about heart health issues within the cycling community.
  • The campaign is called 'Follow Your Heart'.
  • Kindred Group plc's (Kindred) flagship brand Unibet has, together with Tour de Tietema (TDT), developed an online learning tool in response to the growing concern over heart health issues within the cycling community.
  • In addition, the cycling team will wear a custom exclusive jersey to draw attention to the importance of heart health.

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Retrieved on: 
Tuesday, February 13, 2024

ZUG, Switzerland and BOSTON, Feb. 13, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

Key Points: 
  • “The approval by the European Commission is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for patients with severe sickle cell disease and transfusion-dependent beta thalassemia,” said Samarth Kulkarni, Chairman and Chief Executive Officer of CRISPR Therapeutics.
  • Vertex is working closely with national health authorities in the European Union (EU) to secure access for eligible patients as quickly as possible.
  • Through this work, they have secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • There are currently three activated ATCs in the EU with plans to activate a total of approximately 25 centers across Europe.

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, February 13, 2024

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.
  • CASGEVY is the only genetic therapy approved for SCD and TDT patients in the European Union (EU) and with this approval, there are now more than 8,000 patients potentially eligible for treatment.
  • Through this work, Vertex has secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • Vertex continues to engage with hospitals experienced in stem cell transplantation to establish a network of independently operated authorized treatment centers (ATCs) for the administration of CASGEVY.

Solvias to Perform Release Testing on World's First CRISPR-based Gene-Editing Therapy

Retrieved on: 
Tuesday, January 23, 2024

KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy. The company has signed a long-term agreement with Vertex Pharmaceuticals for CASGEVY™ (exagamglogene autotemcel or exa-cel) which received U.S. Food & Drug Administration (FDA) approval for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.

Key Points: 
  • KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy.
  • The companies' collaboration included establishing the testing methods that will be scaled for commercializing CASGEVY.
  • Solvias also has invested significantly in preparing one of its global facilities to support the commercial release work for this transformative therapy.
  • "Solvias is honored to be playing a critical role in delivering this breakthrough therapy to patients.

Solvias to Perform Release Testing on World's First CRISPR-based Gene-Editing Therapy

Retrieved on: 
Tuesday, January 23, 2024

KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy. The company has signed a long-term agreement with Vertex Pharmaceuticals for CASGEVY™ (exagamglogene autotemcel or exa-cel) which received U.S. Food & Drug Administration (FDA) approval for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.

Key Points: 
  • KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy.
  • The companies' collaboration included establishing the testing methods that will be scaled for commercializing CASGEVY.
  • Solvias also has invested significantly in preparing one of its global facilities to support the commercial release work for this transformative therapy.
  • "Solvias is honored to be playing a critical role in delivering this breakthrough therapy to patients.

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).

CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024

ZUG, Switzerland and BOSTON, Jan. 16, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

Key Points: 
  • “We are pleased with the approval of CASGEVY in TDT well ahead of the PDUFA date,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics.
  • “The approval is a reflection of the power and versatility of the CRISPR platform to bring a potentially curative treatment option to patients suffering from this devastating disease.”
    The administration of CASGEVY requires experience in stem cell transplantation; therefore, our partner Vertex Pharmaceuticals Incorporated is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com.

Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
  • “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com .