Hemolytic anemia

AB Science: The clinical development of masitinib in sickle cell disease is among the 19 winning projects under the sixth call for “Hospital-Inuversity Research in health (RHU)”

Retrieved on: 
Monday, November 27, 2023

THE CLINICAL DEVELOPMENT OF MASITINIB IN SICKLE CELL DISEASE, A HIGHLY PREVALENT GENETIC CONDITION, IS AMONG THE 19 WINNING PROJECTS UNDER THE SIXTH CALL FOR "HOSPITAL-UNIVERSITY RESEARCH IN HEALTH (RHU)".

Key Points: 
  • THE CLINICAL DEVELOPMENT OF MASITINIB IN SICKLE CELL DISEASE, A HIGHLY PREVALENT GENETIC CONDITION, IS AMONG THE 19 WINNING PROJECTS UNDER THE SIXTH CALL FOR "HOSPITAL-UNIVERSITY RESEARCH IN HEALTH (RHU)".
  • A new patent has been filed, which, if granted, will extend the international protection of masitinib in sickle cell disease until 2040.
  • Current treatment options such as hydroxycarbamide, chronic transfusion or anti-P-selectin antibodies, do not fully prevent life-threatening acute and chronic complications of sickle cell disease.
  • There is a significant medical need to prevent the acute and chronic complications of sickle cell disease.

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events

Retrieved on: 
Saturday, December 9, 2023

The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.

Key Points: 
  • The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.
  • Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with the most patients treated and longest follow-up in the field.
  • As of February 13, 2023, 59 patients were treated across the entire clinical development program with follow-up beyond 8 years in the earliest treated patients.
  • In sickle cell disease, hemolysis happens too quickly due to the fragility of sickled red blood cells, resulting in hemolytic anemia.

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

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.

ImmuneOncia Announces Phase 1 Results of CD47 Antibody at ESMO 2023

Retrieved on: 
Monday, October 23, 2023

ImmuneOncia (CEO Heung Tae Kim) announced the results of its Phase 1a solid tumour clinical trial (IMC-002-K102 Study) of IMC-002, a CD47 monoclonal antibody, at the European Society for Medical Oncology (ESMO 2023) held in Madrid, Spain on October 23rd, 2023.

Key Points: 
  • ImmuneOncia (CEO Heung Tae Kim) announced the results of its Phase 1a solid tumour clinical trial (IMC-002-K102 Study) of IMC-002, a CD47 monoclonal antibody, at the European Society for Medical Oncology (ESMO 2023) held in Madrid, Spain on October 23rd, 2023.
  • The dose-escalation study aimed to assess the safety and tolerability, and to establish the recommended Phase 2 dose (RP2D) of IMC-002.
  • 92% of treatment-related adverse events (TRAE) were of Grade 1-2, with 94% occurring during the first treatment cycle.
  • In addition to the CD47 antibody IMC-002, ImmuneOncia also has a wide range of pipeline that includes the Phase 2 clinical-stage PD-L1 antibody IMC-001, and the preclinical-stage bispecific antibody IMC-201.

SIKLOS® (hydroxyurea) is now covered by State Medicaid programs in 19 states

Retrieved on: 
Thursday, September 21, 2023

“Since launching Siklos® five years ago, Medunik USA has been focusing its efforts on easing access to this valuable treatment option which is now covered by State Medicaid programs in 19 states,” said Tanya Carro, Executive Vice-President of Medunik USA.

Key Points: 
  • “Since launching Siklos® five years ago, Medunik USA has been focusing its efforts on easing access to this valuable treatment option which is now covered by State Medicaid programs in 19 states,” said Tanya Carro, Executive Vice-President of Medunik USA.
  • Most of those affected are of African ancestry; a minority are of Hispanic or southern European, Middle Eastern or Asian Indian descent3.
  • Your healthcare provider will check your blood cell counts before and every 2 weeks during treatment with SIKLOS®.
  • Your healthcare provider may do blood tests if you have persistent or worsening anemia not related to sickle cell anemia.

Agios Launches “Red Cell Revolution” in Recognition of Rare Disease Day

Retrieved on: 
Tuesday, February 28, 2023

CAMBRIDGE, Mass., Feb. 28, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced the launch of a multi-stakeholder advocacy advisory council for hemolytic anemias including PK deficiency, thalassemia and sickle cell disease.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 28, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced the launch of a multi-stakeholder advocacy advisory council for hemolytic anemias including PK deficiency, thalassemia and sickle cell disease.
  • “The Red Cell Revolution advisory council is designed to build connections among PK deficiency, thalassemia and sickle cell disease advocates and thought leaders, and to generate insights to raise disease awareness, inform Agios’ mission and contribute to shaping the broader hemolytic anemia field through evidence-based patient advocacy,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios.
  • I’m grateful to Agios for spearheading this initiative and facilitating these connections which can fuel positive changes for people with sickle cell disease, thalassemia and PK deficiency.”
    To learn more about the Red Cell Revolution, watch this video or listen to this podcast episode , which debuted yesterday across three Agios-sponsored podcasts, including Just Listen: Voices of PK Deficiency , Thal Pals: The Alpha Beta Revolution and Cheat Codes: A Sickle Cell Podcast .
  • The Agios team is also supporting rare disease communities throughout the month of February as a sponsor of the following initiatives:

PYRUKYND® (mitapivat) Approved in the EU for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients

Retrieved on: 
Thursday, November 10, 2022

Agios is providing access to PYRUKYND for the treatment of PK deficiency in adults receiving care in the EU through a global managed access program.

Key Points: 
  • Agios is providing access to PYRUKYND for the treatment of PK deficiency in adults receiving care in the EU through a global managed access program.
  • PYRUKYND was previously granted orphan drug designation by the EMA, which is maintained at the time of EU marketing authorization.
  • PYRUKYND was approved by the U.S. Food and Drug Administration (FDA) in February 2022 for the treatment of hemolytic anemia in adults with PK deficiency.
  • PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States, and for the treatment of PK deficiency in adult patients in the European Union.

Baebies Receives FDA 510(k) Clearance for G6PD Test on FINDER Platform

Retrieved on: 
Wednesday, November 9, 2022

DURHAM, N.C., Nov. 9, 2022 /PRNewswire/ -- Baebies has received U.S. Food and Drug Administration (FDA) 510(k) clearance for its rapid, point-of-care test for glucose-6-phosphate dehydrogenase (G6PD) deficiency.

Key Points: 
  • DURHAM, N.C., Nov. 9, 2022 /PRNewswire/ -- Baebies has received U.S. Food and Drug Administration (FDA) 510(k) clearance for its rapid, point-of-care test for glucose-6-phosphate dehydrogenase (G6PD) deficiency.
  • The test is run on the FINDER platform, which features a toaster-sized instrument and a disposable cartridge.
  • Test results display G6PD enzyme activity in units per gram of hemoglobin and adjusted male median values.
  • With G6PD as the first FDA-cleared test on our FINDER platform, we look forward to adding many additional types of tests to the versatile and multifunctional platform."

Sickle Cell Disease Association of America & Partners Launch New PSA to Boost Awareness and Understanding of the Disease

Retrieved on: 
Friday, September 30, 2022

The Sickle Cell Disease Association of America, Inc. (SCDAA), in partnership with Forma Therapeutics, Inc. (Nasdaq: FMTX) today announced that they have joined forces to create a public service announcement (PSA) to drive awareness and advance meaningful understanding of sickle cell disease (SCD).

Key Points: 
  • The Sickle Cell Disease Association of America, Inc. (SCDAA), in partnership with Forma Therapeutics, Inc. (Nasdaq: FMTX) today announced that they have joined forces to create a public service announcement (PSA) to drive awareness and advance meaningful understanding of sickle cell disease (SCD).
  • campaign urges viewers to imagine they are experiencing the agonizing but unseen pain that is faced by people who live with SCD.
  • This incredibly powerful PSA brings the experience of living with SCD to life and makes it extraordinarily real for the viewers.
  • The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services.

Immunovant Announces IMVT-1402, a Next Generation Anti-FcRn  

Retrieved on: 
Wednesday, September 28, 2022

The combined franchise of batoclimab and IMVT-1402 may enable multiple paths to enhanced value creation for Immunovant.

Key Points: 
  • The combined franchise of batoclimab and IMVT-1402 may enable multiple paths to enhanced value creation for Immunovant.
  • All forward-looking statements are based on estimates and assumptions by Immunovants management that, although Immunovant believes to be reasonable, are inherently uncertain.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Immunovant expected.
  • Immunovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.