Betibeglogene autotemcel

AffyImmune Therapeutics Appoints Matt Britz as Chief Executive Officer and Pete Gelinas as SVP of CMC

Retrieved on: 
Tuesday, March 19, 2024
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AffyImmune Therapeutics, Inc ., a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, affinity-tuned CAR T cell therapies, announced today the appointment of Matt Britz to Chief Executive Officer and Pete Gelinas as Senior Vice President of Chemistry, Manufacturing, and Controls.

Key Points: 
  • AffyImmune Therapeutics, Inc ., a clinical-stage biopharmaceutical company committed to developing novel, first-in-class, affinity-tuned CAR T cell therapies, announced today the appointment of Matt Britz to Chief Executive Officer and Pete Gelinas as Senior Vice President of Chemistry, Manufacturing, and Controls.
  • Matt is well-suited to lead AffyImmune as CEO as the Company accelerates clinical development of AIC100 in thyroid cancer, and in other indications with high unmet medical need.
  • Matt Britz joined AffyImmune in June 2021 as Senior Vice President (SVP), Business Development and was promoted to Chief Operating Officer in January 2022.
  • Pete Gelinas brings over 15 years of extensive experience in Manufacturing and CMC Product Development to AffyImmune.

Long-Term Data Presented at ASH Support beti-cel as a Potentially Curative Gene Therapy for β-Thalassemia Patients Who Require Regular Transfusions Through Achievement of Durable Transfusion Independence and Normal or Near-Normal Adult Hb Levels

Retrieved on: 
Sunday, December 10, 2023
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As of the data cutoff date, TI was maintained through last follow-up (up to 6 years) across ages and genotypes.

Key Points: 
  • As of the data cutoff date, TI was maintained through last follow-up (up to 6 years) across ages and genotypes.
  • Among patients in the Phase 1/2 studies, 68.2% (15/22) achieved TI, with 14 patients sustaining TI through the last follow-up (up to 9 years).
  • The patient continued to benefit from beti-cell therapy and was not receiving chronic red blood cells transfusions as of the last follow-up.
  • The most common beti-cel-related AEs (occurring in ≥3 patients) were abdominal pain (7.9% of patients) and thrombocytopenia (4.8% of patients).

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
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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.

FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions

Retrieved on: 
Wednesday, August 17, 2022
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SILVER SPRING, Md., Aug. 17, 2022 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

Key Points: 
  • SILVER SPRING, Md., Aug. 17, 2022 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.
  • The reduced levels of red blood cells can lead to a number of health issues including dizziness, weakness, fatigue, bone abnormalities and more serious complications.
  • Transfusion-dependent beta-thalassemia, the most severe form of the condition, generally requires life-long red blood cell transfusions as the standard course of treatment.
  • The safety and effectiveness of Zynteglo were established in two multicenter clinical studies that included adult and pediatric patients with beta-thalassemia requiring regular transfusions.

bluebird bio Reports Second Quarter 2022 Financial Results and Highlights Operational Progress

Retrieved on: 
Thursday, August 4, 2022
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bluebird bio, Inc. (NASDAQ: BLUE) (bluebird bio or the Company) today reported financial results and business highlights for the second quarter ended June 30, 2022, and shared recent operational progress.

Key Points: 
  • bluebird bio, Inc. (NASDAQ: BLUE) (bluebird bio or the Company) today reported financial results and business highlights for the second quarter ended June 30, 2022, and shared recent operational progress.
  • The second quarter marked significant progress for bluebird bio and a precedent-setting moment for the field of gene therapy, said Andrew Obenshain, chief executive officer, bluebird bio.
  • CONTINUED PROGRESS TOWARD BLA SUBMISSION bluebird bio remains on track to submit a biologics licensing application (BLA) to the FDA for lovotibeglogene autotemcel (lovo-cel) for sickle cell disease in the first quarter of 2023.
  • This quarter, the Company completed manufacturing of commercial drug product validation lots, marking significant progress on CMC requirements and final steps to BLA submission.

FDA Advisory Committee Unanimously Supports beti-cel Gene Therapy for People with beta-thalassemia Who Require Regular Red Blood Cell Transfusions

Retrieved on: 
Friday, June 10, 2022
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Patients with the most severe form experience severe anemia and lifelong dependence on red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks.

Key Points: 
  • Patients with the most severe form experience severe anemia and lifelong dependence on red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks.
  • This can result in severe anemia and lifelong dependence on red blood cell (RBC) transfusions.
  • Patients who require regular RBC transfusions to maintain adequate Hb levels typically undergo the 4-7-hour process every 2-5 weeks.
  • betibeglogene autotemcel (beti-cel) (pronounced BEH tee cell) is a one-time gene therapy custom-designed to treat the underlying cause of beta-thalassemia in patients who require regular red blood cell (RBC) transfusions.

bluebird bio stock trading halted today June 9th and tomorrow June 10th

Retrieved on: 
Thursday, June 9, 2022
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bluebird bio, Inc. (NASDAQ: BLUE) (bluebird bio or the Company) today announced that Nasdaq has halted trading of the companys common stock on Thursday, June 9 and Friday, June 10, 2022.

Key Points: 
  • bluebird bio, Inc. (NASDAQ: BLUE) (bluebird bio or the Company) today announced that Nasdaq has halted trading of the companys common stock on Thursday, June 9 and Friday, June 10, 2022.
  • bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
  • Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the worldsetting the standard for the industry.
  • bluebird bio is a trademark of bluebird bio, Inc.

bluebird bio Announces Posting of Briefing Documents for Upcoming FDA Advisory Committee Meeting

Retrieved on: 
Tuesday, June 7, 2022
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The advisory committee meeting will take place June 9-10, 2022.

Key Points: 
  • The advisory committee meeting will take place June 9-10, 2022.
  • The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.
  • bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
  • bluebird bio is a trademark of bluebird bio, Inc.

bluebird bio Initiates Restructuring to Reduce Operating Expenses and Advance Near-term Opportunities to Bring Potentially Curative Gene Therapies to Patients in the US

Retrieved on: 
Tuesday, April 5, 2022
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As part of the changes, bluebird plans to reduce its workforce by approximately 30%.

Key Points: 
  • As part of the changes, bluebird plans to reduce its workforce by approximately 30%.
  • The restructuring is expected to extend the Companys cash runway into the first half of 2023.
  • Today, we are taking decisive action to extend our cash runway, and put bluebird in a stronger position to execute on our strategic priorities and ultimately bring potentially curative gene therapies to patients and their families.
  • bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.

2seventy bio Announces Key Management Appointments

Retrieved on: 
Monday, February 14, 2022
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Also announced today, Susan Abu-Absi, Ph.D. has been appointed chief technology & manufacturing officer at 2seventy bio.

Key Points: 
  • Also announced today, Susan Abu-Absi, Ph.D. has been appointed chief technology & manufacturing officer at 2seventy bio.
  • She most recently served as our Senior Vice President and Head of Technical Development & Operations at 2seventy bio.
  • 2seventy bio is a trademark of 2seventy bio, Inc.
  • All information in this press release is as of the date of the release, and 2seventy bio undertakes no duty to update this information unless required by law.