Ronas Voe

bluebird bio Announces FDA Approval of LYFGENIA™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
Friday, December 8, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Red blood cell, Sickle cell disease, Patient, Anemia, Quality of life, Bangladesh Technical Education Board, Qualification, Webcast, Priority review, Lung, Vasculitis, Food, Incidence, Pain, Priapism, Acute myeloid leukemia, Bluebird, PIN, Genetic disorder, Regenerative medicine, Division, Ronas Voe, DSM-IV codes, Hematology, Vaso-occlusive crisis, Courage, Adolescence, Hemolytic anemia, Stomatitis, Cellular, Febrile neutropenia, History, Leukopenia, Thrombocytopenia, Neutropenia, University, QTC, Life, NHLBI, Bone marrow, Research, HBA, Bluebird bio, Telephone, Disease, Acute chest syndrome, Caregiver, NIH, MD, Blood transfusion, Pharmaceutical industry, Vaccine, Online shopping

LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Key Points: 
  • LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
  • “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events.
  • The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
  • A replay of the webcast will be available on the bluebird website for 90 days following the event.

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.

bluebird bio to Present New and Updated Data from Gene Therapy Programs in Sickle Cell Disease and Beta-Thalassemia at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition

Retrieved on: 
Thursday, November 2, 2023
Health, Genetics, Other Health, General Health, Pharmaceutical, Biotechnology, Hemoglobinopathy, FDA, MSCE, PDUFA, Busulfan, Food, LVV, SCD, History, Sickle cell disease, Ronas Voe, Prescription Drug User Fee Act, Data, Children's Hospital of Philadelphia, Thalassemia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, TDT, Max Healthcare, University of Pennsylvania, Pediatrics, Society, ASH, Priority review, Quality of life, San Diego Convention Center, Division, HRQOL, Perelman School of Medicine at the University of Pennsylvania, University, Exposition, Month, Hospital, BLA, Betibeglogene autotemcel, Pharmaceutical industry, Vaccine, Patient, Quality of life (healthcare), Safety, Hematology

The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.

Key Points: 
  • The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.
  • lovo-cel treatment regimen largely reflects known side effects of hematopoietic stem cell collection and busulfan conditioning regimen and underlying sickle cell disease.
  • Updated iron management outcomes demonstrating sustained improvements in iron burden, with the majority of patients able to stop iron chelation therapy, will also be presented.
  • Safety of beti-cel treatment largely reflects the known side effects of hematopoietic stem cell collection and busulfan conditioning regimen.

bluebird bio Announces FDA Priority Review of the Biologics License Application for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events

Retrieved on: 
Wednesday, June 21, 2023
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Biologics license application, Priority review, File, SCD, Ronas Voe, History, Regenerative medicine, Prescription Drug User Fee Act, Patient, PDUFA, Sickle cell disease, Diagnosis, Caregiver, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, DSM-IV codes, Safety, Disease, Pharmaceutical industry

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review.

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review.
  • Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
  • The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.
  • The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.

bluebird bio Submits Biologics License Application (BLA) to FDA for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events

Retrieved on: 
Monday, April 24, 2023
Science, Stem Cells, Biotechnology, Research, Pharmaceutical, General Health, Health, FDA, Priority review, File, SCD, Ronas Voe, History, DSM-IV codes, Regenerative medicine, Patient, Sickle cell disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, Caregiver, Safety, Pharmaceutical industry

bluebird bio, Inc. (Nasdaq: BLUE) today announced the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) today announced the submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
  • “The severity of sickle cell disease, and its impact on patients and caregivers, has been underappreciated and overlooked for far too long.
  • Transformative therapies for this community are long overdue,” said Andrew Obenshain, chief executive officer, bluebird bio.
  • The BLA submission also includes safety data from 50 patients treated across the entire lovo-cel program, including six patients with six or more years of follow-up.