Haematopoietic system

New Chair and Executive Committee Members Appointed of Lymphoma Research Foundation Mantle Cell Lymphoma Consortium

Retrieved on: 
Thursday, February 15, 2024

NEW YORK, Feb. 15, 2024 /PRNewswire-PRWeb/ -- The Lymphoma Research Foundation (LRF) – the nation's largest non-profit organization devoted exclusively to funding innovative lymphoma research and serving the lymphoma community through a comprehensive series of education programs, outreach initiatives, and patient services – is pleased to announce Tycel Phillips, MD of City of Hope and Kami Maddocks, MD of Ohio State University to the Executive Committee members and Peter Martin, MD of Weill Cornell Medicine as Chair of its Mantle Cell Lymphoma Consortium (MCLC) Executive Committee.

Key Points: 
  • "LRF's Mantle Cell Lymphoma Consortium provides a unique forum for the world's leading MCL researchers to share scientific and clinical findings, exchange ideas, and plan new collaborations," said Meghan Gutierrez, CEO of the Lymphoma Research Foundation
    The Mantle Cell Lymphoma Consortium Executive Committee helps to guide the MCLC by identifying gaps in research and patient care for this historically understudied patient population.
  • Since its inception the Mantle Cell Lymphoma Consortium brings together leading international experts to discuss the latest research findings, foster collaboration within the mantel cell lymphoma (MCL) research community, create new directions for research, and ultimately improve diagnosis and treatment for this disease.
  • "The Lymphoma Research Foundation's Mantle Cell Lymphoma Consortium provides a unique forum for the world's leading MCL researchers to share scientific and clinical findings, exchange ideas, and plan new collaborations," said Meghan Gutierrez, Chief Executive Officer of the Lymphoma Research Foundation.
  • "I'm honored to lead this vital consortium dedicated to improving outcomes for all those impacted by mantle cell lymphoma," said Dr. Martin.

City of Hope Research Featuring the Successful Treatment of the Oldest Patient to Achieve Remission for Leukemia and HIV Published in The New England Journal of Medicine (NEJM)

Retrieved on: 
Wednesday, February 14, 2024

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20240214747351/en/
    "City of Hope Patient" Paul Edmonds with City of Hope doctors, Jana K. Dickter, M.D., and Monzr Al Malki, M.D.
  • Edmonds is also the person who had HIV the longest — for over 31 years — among these five patients.
  • Known as the “City of Hope patient” among these five patients, Edmonds received a transplant at City of Hope on Feb. 6, 2019, and is now considered to be cured of leukemia.
  • City of Hope has exceptional transplant outcomes year after year, according to the Center for International Blood & Marrow Transplant Research.

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.

Midwest Transplant Network Surpasses Expectations to Save and Enhance Lives in 2023

Retrieved on: 
Monday, February 5, 2024

“What we achieved at Midwest Transplant Network in 2023 reflects the commitment of the staff who do the work every day to support organ donation and transplantation.

Key Points: 
  • “What we achieved at Midwest Transplant Network in 2023 reflects the commitment of the staff who do the work every day to support organ donation and transplantation.
  • The work is hard, and happens in countless ways, but we are all dedicated to the mission of enhancing and saving lives through organ, eye and tissue donation” said Jan Finn, RN, MSN, President & CEO, Midwest Transplant Network.
  • The United Network for Organ Sharing reported that across the country, clinical workers performed more than 46,000 lifesaving transplants in 2023.
  • Incorporated in 1973, Midwest Transplant Network facilitates organ, eye and tissue donation in partnership with hospitals and other professional partners to give hope and share life.

Singular Genomics Unveils G4X™ Spatial Sequencer, Transforming the Landscape of In Situ Multiomic Analysis

Retrieved on: 
Monday, February 5, 2024

The G4X is designed to offer high-throughput in situ direct sequencing of RNA (Direct-Seq™), targeted transcriptomics, and proteomics profiling from formalin-fixed, paraffin-embedded (FFPE) tissues.

Key Points: 
  • The G4X is designed to offer high-throughput in situ direct sequencing of RNA (Direct-Seq™), targeted transcriptomics, and proteomics profiling from formalin-fixed, paraffin-embedded (FFPE) tissues.
  • The G4X Spatial Sequencer is designed to offer novel capabilities, stackable data streams, and unprecedented throughput for spatial profiling of tissue.
  • Its novel capabilities will include Direct-Seq, which is the sequencing of RNA molecules in situ and has the potential to open new areas of scientific discovery.
  • Singular also plans to provide G4X Spatial Sequencing Technology Access Services starting in the second quarter of 2024.

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 .

City of Hope Research Reveals an Immune Cell That Can Attack Cancer

Retrieved on: 
Wednesday, January 10, 2024

According to preclinical research published online today in Cell , one of the world’s premier scientific journals, researchers with City of Hope®, one of the largest cancer treatment and research organizations in the United States, have discovered that a type of immune cell in the human body known to be important for allergy and other immune responses can also attack cancer.

Key Points: 
  • According to preclinical research published online today in Cell , one of the world’s premier scientific journals, researchers with City of Hope®, one of the largest cancer treatment and research organizations in the United States, have discovered that a type of immune cell in the human body known to be important for allergy and other immune responses can also attack cancer.
  • View the full release here: https://www.businesswire.com/news/home/20240109475331/en/
    City of Hope researchers discovered that ILC2s, pictured here, can attack cancer cells.
  • However in the highly translational labs at City of Hope, researchers prioritized the examination of human cells and found that human ILC2s do not work the same as mouse ILC2s.
  • In fact, Yu is founding director of the Natural Killer Cell Biology Research Program at City of Hope, a national leader in the field.

City of Hope Children’s Cancer Center, Children’s Oncology Group conduct largest clinical trial seeking to prevent heart failure among childhood cancer survivors

Retrieved on: 
Tuesday, January 9, 2024

Physicians at City of Hope, one of the largest cancer research and treatment organizations in the United States, in cooperation with the Children’s Oncology Group (COG) , have conducted the largest clinical trial to date seeking to reduce the risk of people who have survived childhood cancer from developing heart failure.

Key Points: 
  • Physicians at City of Hope, one of the largest cancer research and treatment organizations in the United States, in cooperation with the Children’s Oncology Group (COG) , have conducted the largest clinical trial to date seeking to reduce the risk of people who have survived childhood cancer from developing heart failure.
  • View the full release here: https://www.businesswire.com/news/home/20240109428042/en/
    Physicians at City of Hope Children's Cancer Center, in cooperation with the Children’s Oncology Group (COG), have conducted the largest clinical trial to date seeking to reduce the risk of people who have survived childhood cancer from developing heart failure.
  • Unfortunately, after the onset of heart function decline, the downward cascade is irreversible, highlighting an urgent need for early prevention strategies.
  • “The growing number of childhood cancer survivors makes the development of early interventions imperative.