Hemoglobin A

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
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

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
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

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).

FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

Retrieved on: 
Friday, December 8, 2023
Malignancy, VOC, DNA, Sickle cell disease, Abdominal pain, Headache, Patient, Research, Hemoglobin A, Cas9, CRISPR, Oxygen, Volatile organic compound, Recurrence, Fever, Mutation, Priority review, DSM-IV codes, Vomiting, Bone marrow, Hematology, China Biologic Products, Safety, Food, Multimedia, Rejection, HBF, Ronas Voe, Health, Person, Nausea, Throat, Mouth, Hispanic and Latino Americans, Public, Adolescence, Stomatitis, Risk, Tissue, Febrile neutropenia, History, End organ damage, Fetal hemoglobin, Animal, Pain, SCD, B cell, Regenerative medicine, Heated tobacco product, Disease, FDA, African Americans, Mouth ulcer, Vaccine, Cosmetics, Blood, Human services, Dietary supplement, Freedom, Pharmaceutical industry

Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S.

Key Points: 
  • Sickle cell disease is a group of inherited blood disorders affecting approximately 100,000 people in the U.S.
  • The primary problem in sickle cell disease is a mutation in hemoglobin, a protein found in red blood cells that delivers oxygen to the body's tissues.
  • Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises.
  • In patients with sickle cell disease, increased levels of HbF prevent the sickling of red blood cells.

New gene therapy could provide cure for sickle cell disease, according to UAB study

Retrieved on: 
Friday, December 17, 2021
Hope, Cell, Korea Disease Control and Prevention Agency, Cancer, University of Alabama, The New England Journal of Medicine, Sickle cell disease, End organ damage, B cell, Vaccine, Gene editing, Gene silencing, Hydroxycarbamide, Patient, Center, Disease, Attention, Letter, Â, Kanter, Leukemia, GLOBE, University, HBS, Sickle cell trait, SCD, Hemoglobin A, Adult, Pharmaceutical industry, HBA

Kanter says there are several types of gene therapy (gene addition/transfer, gene editing, gene correction and gene silencing), but this particular therapy is gene addition or transfer.

Key Points: 
  • Kanter says there are several types of gene therapy (gene addition/transfer, gene editing, gene correction and gene silencing), but this particular therapy is gene addition or transfer.
  • In this therapy, we do not change or edit the gene that causes sickle cell disease, Kanter said.
  • Instead, we use a viral vector to deliver a new gene that will make a healthy hemoglobin a beta hemoglobin into the stem cell.
  • We need to make these treatments available, and we need all people with sickle cell disease to have a sickle cell doctor to make that happen, she said.

CHOP Researchers Develop Proof-of-Concept Treatment that Elevates Both Adult and Fetal Hemoglobin

Retrieved on: 
Tuesday, June 1, 2021
Hemoglobins, Medical specialties, Hematology, Branches of biology, Hemoglobin A, Fetal hemoglobin, Thalassemia, Beta thalassemia, Hemoglobin variants, Hemoglobinopathy

PHILADELPHIA, June 1, 2021 /PRNewswire/ --Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin.

Key Points: 
  • PHILADELPHIA, June 1, 2021 /PRNewswire/ --Researchers at Children's Hospital of Philadelphia (CHOP) have developed a proof-of-concept treatment for blood disorders like sickle cell disease and beta-thalassemia that could raise hemoglobin levels by activating production of both fetal and adult hemoglobin.
  • Using a viral vector engineered to reactivate fetal hemoglobin production, suppress mutant hemoglobin, and supply functional adult hemoglobin, the researchers developed an approach that could produce more hemoglobin through a single vector.
  • In utero, the gamma-globin gene produces fetal hemoglobin, but after birth, this gene is switched off and the beta-globin gene is turned on, producing adult hemoglobin.
  • By elevating both fetal and functional adult hemoglobin, the vector was able to induce more functional hemoglobin production than that of a vector expressing beta-globin alone.

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

Retrieved on: 
Monday, October 5, 2020
Hemoglobins, Branches of biology, Medical specialties, Medical genetics, Fetal hemoglobin, Hemoglobinopathy, Sickle cell disease, Thalassemia, HBB, Beta thalassemia, Hemoglobin A, Hemoglobin variants

FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.

Key Points: 
  • FTX-6058 is a small molecule designed to increase expression of fetal hemoglobin with the potential to treat hemoglobinopathies such as sickle cell disease and beta-thalassemia.
  • This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts.
  • Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene.
  • Fulcrum is also advancing FTX-6058, a small molecule designed to increase expression of fetal hemoglobin for the treatment of sickle cell disease and beta thalassemia into Phase 1 clinical development.

Agios First-in Class PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent α- and β-Thalassemia in Phase 2 Study

Retrieved on: 
Friday, June 12, 2020
Hemoglobins, Medical specialties, Branches of biology, Hematology, Thalassemia, Hemolysis, Hemoglobin A

Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes.

Key Points: 
  • Mitapivat is an investigational, first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase-R (PKR) enzymes.
  • Findings from the study indicate that activation of wild-type PKR by mitapivat, an oral treatment option, improved hemoglobin and associated markers of hemolysis and erythropoiesis in patients with - and -thalassemia.
  • Our focus now is to advance the development of mitapivat for these patients as quickly and efficiently as possible.
  • The ongoing, open-label Phase 2 study is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of mitapivat treatment in adults with non-transfusion-dependent - and -thalassemia who have a baseline hemoglobin (Hb) concentration of 10 g/dL.

bluebird bio to Present Data from Its Gene and Cell Therapy Programs During the Virtual Edition of the 25th European Hematology Association Annual Congress

Retrieved on: 
Thursday, May 14, 2020
Biotechnology, Health, Genetics, Clinical trials, Oncology, Branches of biology, Medical specialties, Medicine, RTT, Hemoglobins, Thalassemia, Sickle cell disease, Gene therapy, Multiple myeloma, Globin, Hemoglobin A, Sickle cell-beta thalassemia

(Nasdaq: BLUE) announced today that data from its gene therapy programs for sickle cell disease (SCD), transfusion-dependent -thalassemia (TDT) and its cell therapy program for relapsed and refractory multiple myeloma (RRMM) will be presented during the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.

Key Points: 
  • (Nasdaq: BLUE) announced today that data from its gene therapy programs for sickle cell disease (SCD), transfusion-dependent -thalassemia (TDT) and its cell therapy program for relapsed and refractory multiple myeloma (RRMM) will be presented during the Virtual Edition of the 25th European Hematology Association (EHA25) Annual Congress.
  • TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb).
  • Betibeglogene autotemcel adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs).
  • LentiGlobin for sickle cell disease is an investigational gene therapy being studied as a potential treatment for SCD.

bluebird bio Announces Launch in Germany of ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene) Gene Therapy for Patients 12 Years and Older with Transfusion-Dependent β-Thalassemia Who Do Not Have β0/β0 Genotype

Retrieved on: 
Monday, January 13, 2020
Oncology, Health, Stem cells, Genetics, General Health, Pharmaceutical, Hemoglobins, Branches of biology, Medical specialties, Medicine, Thalassemia, Zynteglo, Hemoglobin A, Beta thalassemia, Alpha-thalassemia, LentiGlobin for β-Thalassemia (autologous CD34+ cells encoding βA-T87Q-globin gene), bluebird bio, Inc.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in significantly reduced or absent adult hemoglobin (HbA).

Key Points: 
  • TDT is a severe genetic disease caused by mutations in the -globin gene that result in significantly reduced or absent adult hemoglobin (HbA).
  • bluebird bio has established a collaboration with University Hospital of Heidelberg as the first qualified treatment center in Germany.
  • We are thrilled to announce that ZYNTEGLO will now be available for patients in the EU living with this severe disease, says Alison Finger, chief commercial officer, bluebird bio.
  • LentiGlobin for -thalassemia adds functional copies of a modified form of the -globin gene (A-T87Q-globin gene) into a patients own hematopoietic (blood) stem cells (HSCs).

bluebird bio Presents New Data Demonstrating Long-Term Transfusion Independence and Safety for LentiGlobin™ Gene Therapy for β-thalassemia (betibeglogene autotemcel) at 61st ASH Annual Meeting and Exposition

Retrieved on: 
Monday, December 9, 2019
Biotechnology, Pharmaceutical, Genetics, Health, Branches of biology, Medical specialties, Hemoglobins, Medicine, Blood transfusion, Globin, Hemoglobin Lepore syndrome, Hemoglobin A, LentiGlobin for β-Thalassemia (betibeglogene autotemcel), bluebird bio, Inc.

TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb).

Key Points: 
  • TDT is a severe genetic disease caused by mutations in the -globin gene that result in reduced or significantly reduced hemoglobin (Hb).
  • TI is defined as weighted average Hb 9 g/dL without red blood cell (RBC) transfusions for more than 12 months.
  • These patients ranged in age from 8 to 34 years, including six pediatric (
  • bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for -thalassemia.