Red blood cell

Cerus Corporation Announces Positive Topline Results for the Phase 3 Clinical Trial of the INTERCEPT Blood System for Red Blood Cells in Cardiovascular Surgery Patients

Retrieved on: 
Tuesday, March 19, 2024

Cerus Corporation (Nasdaq: CERS) today announced positive topline results for ReCePI, a pivotal Phase 3 clinical trial of pathogen reduced INTERCEPT Red Blood Cells (INTERCEPT RBCs) transfused to complex cardiac surgery patients.

Key Points: 
  • Cerus Corporation (Nasdaq: CERS) today announced positive topline results for ReCePI, a pivotal Phase 3 clinical trial of pathogen reduced INTERCEPT Red Blood Cells (INTERCEPT RBCs) transfused to complex cardiac surgery patients.
  • There was no clinical significance related to treatment-emergent RBC antibodies observed in five patients receiving INTERCEPT RBCs.
  • This safety endpoint is also being explored in the Company’s ongoing RedeS Phase 3 clinical trial of INTERCEPT RBCs.
  • “We are extremely pleased by the positive topline results of the ReCePI clinical trial,” stated Richard Benjamin, M.D., Ph.D., Cerus’ chief medical officer and principal investigator for the ReCePI clinical trial.

Cerus Corporation Announces Fourth Quarter and Full-Year 2023 Financial Results

Retrieved on: 
Tuesday, March 5, 2024

Product gross margin for the fourth quarter of 2023 was relatively stable year over year at 55.5% compared to 55.7% for the fourth quarter of 2022.

Key Points: 
  • Product gross margin for the fourth quarter of 2023 was relatively stable year over year at 55.5% compared to 55.7% for the fourth quarter of 2022.
  • R&D expenses for the fourth quarter of 2023 were $14.3 million, compared to $18.6 million for the fourth quarter of 2022.
  • Selling, general, and administrative (SG&A) expenses narrowed for the fourth quarter of 2023 and totaled $17.3 million, compared to $23.2 million for the fourth quarter of 2022.
  • Net loss attributable to Cerus Corporation for the fourth quarter of 2023 was $1.3 million, or $0.01 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $13.6 million, or $0.08 per basic and diluted share, for the fourth quarter of 2022.

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

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

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).

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.

Cerus Corporation Announces Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023

Cerus Corporation (Nasdaq: CERS) today announced financial results for the third quarter ended September 30, 2023.

Key Points: 
  • Cerus Corporation (Nasdaq: CERS) today announced financial results for the third quarter ended September 30, 2023.
  • Product gross margin for the third quarter of 2023 was 54.9% compared to 55.4% for the third quarter of 2022.
  • R&D expenses for the third quarter of 2023 were $16.8 million, compared to $16.2 million for the third quarter of 2022.
  • Net loss attributable to Cerus Corporation for the third quarter of 2023 was $7.3 million, or $0.04 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $8.5 million, or $0.05 per basic and diluted share, for the third quarter of 2022.

Cellectis to Present Pre-Clinical Data on HSPC Gene Therapy Program and Comprehensive Analysis of TALE-BE at the ESGCT 30th Annual Congress

Retrieved on: 
Tuesday, October 24, 2023

The data will be presented in three posters:

Key Points: 
  • The data will be presented in three posters:
    Presenter: Eduardo Seclen, Senior Scientist & Team Leader, Gene Editing
    Intronic editing enables lineage specific expression of therapeutics relevant for HSPC gene therapy.
  • TALEN®-mediated intron editing of the CD11b locus results in the lineage-specific expression of a reporter transgene in myeloid cells, with negligible expression in HSPC or other cellular subsets in vitro and in vivo.
  • We believe this intron editing approach could be disruptive in HSPC gene therapy and brain delivery of multiple therapeutics.
  • Using a combination of scRNA sequencing and multiple genomic read out methodologies, we demonstrate that the mutant HBB gene can be efficiently corrected in HSPCs by TALEN®-mediated gene editing coupled to non-viral gene delivery (ssODN) with a low risk of generating β-thalassemic RBCs.

Cerus Corporation Announces Second Quarter 2023 Financial Results

Retrieved on: 
Wednesday, August 2, 2023

Cerus Corporation (Nasdaq: CERS) today announced financial results for the second quarter ended June 30, 2023.

Key Points: 
  • Cerus Corporation (Nasdaq: CERS) today announced financial results for the second quarter ended June 30, 2023.
  • Selling, general, and administrative (SG&A) expenses for the second quarter of 2023 totaled $20.5 million, compared to $19.5 million for the second quarter of 2022.
  • R&D expenses for the second quarter of 2023 were $19.2 million, compared to $15.2 million for the second quarter of 2022.
  • Net loss attributable to Cerus Corporation for the second quarter of 2023 was $13.3 million, or $0.07 per basic and diluted share, compared to a net loss attributable to Cerus Corporation of $8.4 million, or $0.05 per basic and diluted share, for the second quarter of 2022.

Geron Announces U.S. Expanded Access Protocol for Imetelstat in Lower Risk MDS

Retrieved on: 
Thursday, June 29, 2023

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that an Expanded Access Protocol (EAP) is available for imetelstat, the Company’s first-in-class telomerase inhibitor.

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that an Expanded Access Protocol (EAP) is available for imetelstat, the Company’s first-in-class telomerase inhibitor.
  • In that trial, the primary endpoint of 8-week transfusion independence (TI) was significantly higher with imetelstat vs. placebo (P
  • Mean hemoglobin levels in imetelstat-treated patients increased significantly (P
  • Further, statistically significant and clinically meaningful efficacy results were achieved across key MDS subgroups: ring sideroblast (RS) status, baseline transfusion burden and IPSS risk category.

Keros Therapeutics Presents Clinical Trial and Preclinical Study Results from its KER-050 Program and Preclinical Data from its ALK2 Inhibitor Program at the 28th Annual Congress of the European Hematology Association

Retrieved on: 
Friday, June 9, 2023

In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.

Key Points: 
  • In addition, Keros announced preclinical data evaluating activin receptor-like kinase-2 (“ALK2”) inhibition, as well as its combination with RKER-050, as potential treatment options for anemia of inflammation.
  • Of these patients, 37 had completed at least 24 weeks of treatment or discontinued as of the data cut-off date (the “evaluated RP2D patients”).
  • Data for hematological response and markers of hematopoiesis were presented from exploratory analyses of these evaluated RP2D patients.
  • By targeting ALK2 inhibition to suppress hepcidin, RKER-216 increased iron availability for erythropoiesis and partially rescued anemia in CKD mice.