Beta thalassemia

Study of First Affordable and Rapid Test for Beta Thalassemia Performs at 99% Accuracy in Diagnosing the Blood Disorder

Retrieved on: 
Lunedì, Febbraio 5, 2024

This study concluded that Gazelle provides, for the first time, an affordable and rapid solution for identifying beta-thal at point of care.

Key Points: 
  • This study concluded that Gazelle provides, for the first time, an affordable and rapid solution for identifying beta-thal at point of care.
  • The ability to test accurately and rapidly (results are available in 8 minutes) as demonstrated in this study suggests that Gazelle is suitable for large-scale testing of beta-thal at point of care.
  • Beta thalassemia is an inherited blood disorder.
  • A study published in the European Journal of Hematology has shown, however, that incidence rates decrease in areas with screening and education programs.

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: 
Mercoledì, Gennaio 17, 2024

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: 
Mercoledì, Gennaio 17, 2024

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).

Vertex Receives CHMP Positive Opinion for the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Venerdì, Dicembre 15, 2023

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
  • An approval decision by the European Commission is expected in February 2024.
  • “This positive opinion is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for eligible patients with sickle cell and transfusion-dependent beta thalassemia,” said Nia Tatsis, Ph.D., Executive Vice President and Chief Regulatory and Quality Officer at Vertex.
  • “As an investigator, I have witnessed first-hand the transformative impact exa-cel can have on patients’ lives and I eagerly await the approval in the European Union.”

Editas Medicine Announces Third Quarter 2023 Results and Business Updates

Retrieved on: 
Venerdì, Novembre 3, 2023

CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the third quarter 2023 and provided business updates.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today reported financial results for the third quarter 2023 and provided business updates.
  • Cash, cash equivalents, and marketable securities as of September 30, 2023, were $446.4 million compared to $480.0 million as of June 30, 2023.
  • The increase is primarily related to an upfront payment for the non-exclusive Cas9 license to Vor Bio in the third quarter of 2023.
  • Editas Medicine plans to participate in the following scientific and medical conference:
    Editas Medicine plans to participate in the following investor events:

California State Treasurer, Fiona Ma, and US-India cross-border association, AllianceIndus, congratulate Hemex Health for India partnerships that led to a breakthrough test for sickle cell disease

Retrieved on: 
Mercoledì, Ottobre 18, 2023

The Hemex Diagnostic Platform, Gazelle™, supports a powerful test for hemoglobinopathies including SCD and beta thalassemia and was launched into the Indian market in early 2021.

Key Points: 
  • The Hemex Diagnostic Platform, Gazelle™, supports a powerful test for hemoglobinopathies including SCD and beta thalassemia and was launched into the Indian market in early 2021.
  • It is manufactured in India and currently ships from India to over 30 countries.
  • “Hemex utilized invention from Case Western Reserve University (CWRU), Hemex’s experience in low-cost design, and India’s competitive manufacturing.
  • This winning combination will help us realize our vision of improving access to accurate testing.”
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Prime Medicine and Cimeio Therapeutics Announce Research Collaboration to Develop Prime Edited Shielded-Cell & Immunotherapy Pairs™ for Genetic Diseases, Acute Myeloid Leukemia and Myelodysplastic Syndrome

Retrieved on: 
Giovedì, Giugno 22, 2023

CAMBRIDGE, Mass. and BASEL, Switzerland, June 22, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, and Cimeio Therapeutics, a biotechnology company that is reinventing cell therapy through its leadership in the emerging field of epitope shielding, today announced a research collaboration to combine their respective technologies, including Prime Medicine’s Prime Editing platform and Cimeio’s Shielded Cell and Immunotherapy Pairs (SCIP) platform.

Key Points: 
  • Cimeio’s SCIP platform combined with Prime Editing may significantly improve the accessibility, eligibility and outcomes for patients who could benefit from HSC transplant.
  • These combined technologies may also enable selection of in vivo edited HSCs, which could allow for the treatment of genetic diseases without a transplant.
  • If the research collaboration is successful, the companies will grant exclusive license options to each other for their technology.
  • We look forward to partnering with the extremely talented team at Prime Medicine to advance our novel CD117 program.”

Editas Medicine Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Sickle Cell Disease

Retrieved on: 
Giovedì, Aprile 27, 2023

CAMBRIDGE, Mass., April 27, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of sickle cell disease.

Key Points: 
  • CAMBRIDGE, Mass., April 27, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of sickle cell disease.
  • The FDA previously granted Orphan Drug Designation to EDIT-301 for the treatment of beta thalassemia and Rare Pediatric Disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease.
  • “Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and early death.
  • Receiving Orphan Drug Designation for EDIT-301 for sickle cell disease highlights the urgent need for new treatment options for patients and supports our belief that EDIT-301 can be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients,” Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine.

Silence Therapeutics Announces Publication in Blood Demonstrating Role for Iron Regulation in Polycythemia Vera

Retrieved on: 
Martedì, Aprile 4, 2023

The paper is authored by senior medical and scientific experts from Silence and leading population health and hematology researchers from WEHI (Walter and Eliza Hall Institute) in Melbourne, Australia as well as Cambride UK.

Key Points: 
  • The paper is authored by senior medical and scientific experts from Silence and leading population health and hematology researchers from WEHI (Walter and Eliza Hall Institute) in Melbourne, Australia as well as Cambride UK.
  • The study further demonstrated in a mouse model of PV that hepcidin, a master regulator of iron availability whose expression is influenced by HFE, governs the red blood cell (erythroid) phenotype in PV.
  • “The results we reported in Blood provide a genetic and biological rationale for treating PV with SLN124 by raising hepcidin to control systemic iron levels, thus reducing red blood cell count.
  • SLN124 works by silencing TMPRSS6, a gene that negatively regulates hepcidin expression, to increase production of hepcidin in the liver.

Hemex Health Awarded $3M NIH Grant to Bring Gazelle Hb Variant Test for Sickle Cell Disease to US Market

Retrieved on: 
Lunedì, Marzo 27, 2023

NHLBI, part of the National Institutes of Health, awarded a SBIR Phase IIB Bridge Award to Hemex to advance the commercialization of the company’s Gazelle® Hb Variant Test for the United States market.

Key Points: 
  • NHLBI, part of the National Institutes of Health, awarded a SBIR Phase IIB Bridge Award to Hemex to advance the commercialization of the company’s Gazelle® Hb Variant Test for the United States market.
  • Hemex intends to use the grant to complete activities required for FDA 510(k) clearance.
  • The Gazelle Hb Variant Test can detect Sickle Cell Disease (SCD) and beta thalassemia – genetic blood disorders that can result in debilitating pain, disability, severe complications or death.
  • As an affordable, portable, point-of-care test, Gazelle’s results are equivalent to standard lab tests but do not require complex logistical support.