HSPC

Cellectis Presents Novel TALEN® Editing Processes Enabling Highly Efficient Gene Correction and Gene Insertion in HSPCs

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月曜日, 4月 22, 2024
Brain, Euronext Growth, Pericyte, IDUA, Traffic barrier, Integrin alpha M, HSPC, Mouse, CD4, Myelocyte, CD20, Cell, Intron, DNA, Therapy, Patient, Gene, Society, BBB, DSM-IV codes, Mucopolysaccharidosis, Vaccine

NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ:  CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, will present first data exploring novel TALEN® editing processes in hematopoietic stem and progenitor cells (HSPCs) at the American Society of Gene and Cell Therapy (ASGCT) being held on May 7-11, 2024.

Key Points: 
  • “These two posters showcase the potential and versatility of the TALEN® technology to promote efficient gene insertion in HSPCs.
  • Cellectis has developed a TALEN® mediated promoter-less intron editing technology that enables the expression of a therapeutic transgene exclusively by monocyte derived from edited HSPCs.
  • This novel editing approach is an important addition to the HSPC gene editing toolbox that might unlock new strategies for the treatment of metabolic and neurological diseases.
  • Cellectis has developed and optimized a novel gene editing process, leveraging the TALEN® technology and circular single strand DNA template delivery, enabling highly efficient gene insertion in HSPCs.

Cellectis Announces Two Poster Presentations on Novel TALEN® Editing Process for Gene Correction and Gene Insertion in HSPCs at the ASGCT Annual Meeting

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月曜日, 4月 8, 2024
Euronext Growth, Gene, DNA, HSPC, Society, Therapy, Cell, Gene editing, Vaccine

Cellectis presents the development of a novel gene editing process, leveraging the TALEN® technology and non-viral DNA template delivery, enabling highly efficient gene correction and gene insertion in hematopoietic stem and progenitor cells (HSPCs).

Key Points: 
  • Cellectis presents the development of a novel gene editing process, leveraging the TALEN® technology and non-viral DNA template delivery, enabling highly efficient gene correction and gene insertion in hematopoietic stem and progenitor cells (HSPCs).
  • Gene therapy using hematopoietic and progenitor stem cells (HSPC) has the potential to provide a lifelong supply of genetically encoded therapeutics.
  • Gene editing strategies enabling supra-endogenous expression of therapeutics often rely on constitutive promoters resulting in transgene overexpression irrespective of cellular differentiation, which could be detrimental for HSPC function.
  • Full abstracts and presentations will be available on Cellectis’ website following the event:

Coeptis Therapeutics to Pursue Phase 2 Program Using Proprietary First-in-Class Allogeneic NK Cell Therapy for the Treatment of Viral Infections

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水曜日, 2月 7, 2024
CD34, Infrared spectroscopy correlation table, COVID-19, Cell, Immune system, World Health Organization, NK, Patient, CRS, Safety, AML, Virus, Hospital, COVID, FDA, Death, CDC, MDS, Cytokine release syndrome, HSPC, Therapy, Vaccination, Master of Science, RSV, Infection, Viral, Physician, Cancer, Epidemic, Vaccine

WEXFORD, Pa., Feb. 7, 2024 /PRNewswire/ -- Coeptis Therapeutics Holdings, Inc. (Nasdaq: COEP) (the "Company" or "Coeptis"), a biopharmaceutical company developing innovative cell therapy platforms for cancer and infectious diseases, today announced plans to pursue DVX201, its proprietary allogeneic stem cell expansion and directed differentiation platform of unmodified natural killer (NK) cell therapy, as an antiviral treatment addressing various respiratory viral infections that lead to hospitalization, including the "tripledemic" of influenza, COVID-19 and respiratory syncytial virus (RSV).

Key Points: 
  • DVX201 is produced by pooling CD34+ cells isolated from multiple cord blood donations to increase lot size and immune system diversity.
  • Importantly, doses undergo stringent testing including a potency assay before treatment, to assess their ability to kill virally infected cells.
  • Additionally, DVX201 does not contain T-cells, which significantly reduces the possibility for major rejection by patients, which is a major concern for some cell therapy products.
  • DVX201, the first-ever allogeneic, cord-blood derived NK cell therapy generated from pooled donor CD34+ HSPC cells, has the potential to offer significant support to patients and to our strained healthcare system."

Coeptis Therapeutics President and CEO Issues Letter to Shareholders Highlighting 2023 Accomplishments and Outlook for 2024

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木曜日, 1月 4, 2024
Physician, CD34, Risk, Infection, Mesa, Letter, Cell, Cytokine release syndrome, Master of Science, Karolinska Institute, Research, MDS, Therapy, Cancer, Infrared spectroscopy correlation table, Ligand, University, GEAR, COVID-19, HSPC, Observation, Toxicity, Interim, GVHD, CRS, Growth, Patient, Safety, AML, NK, Pharmaceutical industry

WEXFORD, Pa., Jan. 4, 2024 /PRNewswire/ -- Coeptis Therapeutics Holdings, Inc. (NASDAQ: COEP) (the "Company" or "Coeptis"), a biopharmaceutical company developing innovative cell therapy platforms for cancer, today announced that Dave Mehalick, President and CEO of Coeptis Therapeutics Holdings, has issued a Letter to Shareholders providing a review of its 2023 achievements and anticipated milestones for 2024.

Key Points: 
  • WEXFORD, Pa., Jan. 4, 2024 /PRNewswire/ -- Coeptis Therapeutics Holdings, Inc. (NASDAQ: COEP) (the "Company" or "Coeptis"), a biopharmaceutical company developing innovative cell therapy platforms for cancer, today announced that Dave Mehalick, President and CEO of Coeptis Therapeutics Holdings, has issued a Letter to Shareholders providing a review of its 2023 achievements and anticipated milestones for 2024.
  • Our strategy is to leverage these innovative and complementary platforms to develop powerful, personalized cell-based treatments to improve outcomes for patients with cancer.
  • As we look to 2024, we see enormous potential to advance our pipeline and achieve meaningful growth milestones.
  • On behalf of the entire Coeptis team, I wish you all a happy and healthy 2024.

Cimeio Therapeutics Announces Poster Presentations at ASH 2023

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金曜日, 11月 3, 2023
Biotechnology, Health, Stem Cells, Pharmaceutical, Oncology, Bone marrow, HSPC, American Society of Hematology, CD45, Stem cell, Society, ADC, Abstract, Immunotherapy, CD33, Therapy, Patient, AML, Targeted therapy, Pharmaceutical industry, Vaccine

Cimeio Therapeutics, a biotechnology company that is the leader of the field of epitope editing, today announced the acceptance of two abstracts to be presented at the American Society of Hematology meeting in December 2023 in San Diego.

Key Points: 
  • Cimeio Therapeutics, a biotechnology company that is the leader of the field of epitope editing, today announced the acceptance of two abstracts to be presented at the American Society of Hematology meeting in December 2023 in San Diego.
  • The CD45 ADC therapy was enabled by epitope-edited blood stem cells (HSPCs), which maintain their function but resist depletion by the ADC.
  • CD33 represents the fourth target Cimeio has demonstrated it can effectively shield from immunotherapy depletion.
  • “These data represent important steps in our quest to develop a novel therapy with the potential to cure patients with AML,” said Cimeio CEO Thomas Fuchs.

ReNAgade Therapeutics to Present Preclinical Data Demonstrating Potent Extra-Hepatic Delivery of mRNA Using Proprietary Lipid Nanoparticles at 11th International mRNA Health Conference

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月曜日, 10月 30, 2023
Health, Clinical Trials, Research, Pharmaceutical, Science, Biotechnology, Stem cell, RNA, Renegade, LNP, Liver, Conference, Cell, CD34, NK, Messenger, MPP, Therapy, NHP, HSPC, Vaccine

ReNAgade Therapeutics, a company unlocking the limitless potential for RNA medicines, today announced it will present preclinical data demonstrating novel and proprietary lipid nanoparticle (LNP)-based delivery of mRNA to extra-hepatic tissue in a plenary session at the 11th International mRNA Health Conference, being held October 31 to November 2, 2023 in Berlin, Germany.

Key Points: 
  • ReNAgade Therapeutics, a company unlocking the limitless potential for RNA medicines, today announced it will present preclinical data demonstrating novel and proprietary lipid nanoparticle (LNP)-based delivery of mRNA to extra-hepatic tissue in a plenary session at the 11th International mRNA Health Conference, being held October 31 to November 2, 2023 in Berlin, Germany.
  • “Delivery to extra-hepatic tissue is foundational to our approach, and these data help validate how our diverse LNP library can complement our multi-modal RNA platform to create a robust therapeutic pipeline.
  • Proprietary LNP delivery to natural killer (NK) cells was also identified via a high-throughput, functional barcoding screening in NHP models.
  • Taken together, potent delivery of ReNAgade’s proprietary LNPs was observed in natural killer cells and several subsets of HSCs over multiple animal models (including NHP and humanized murine models), demonstrating robust LNP delivery of mRNA cargo to extra-hepatic tissue.

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

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火曜日, 10月 24, 2023
Knowledge, Gene editing, ISCB Senior Scientist Award, TC, AAV, Therapy, HBB, SCD, Y RNA, Architecture, Euronext Growth, Genome, Cell, Risk, Gene, Integrin alpha M, Congress, ESGCT, European Society of Gene and Cell Therapy, Red blood cell, Myelocyte, DNA, Vaccine, HSPC

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.

CHOP Researchers Improve Fitness of Cells Used in Cell Transplants

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水曜日, 9月 27, 2023
Hematopoietic stem cell transplantation, University, Blood, CHOP, HLA, Culture, Genomics, Gene editing, Proteomics, Research, Cell, T32, Child, Doctor of Philosophy, HSCT, EVS, Transplant, Seminal vesicles, Flow cytometry, MD, Patient, Risk, DOI, Sickle cell disease, Mouse, Hospital, NIH, Hematopathology, Safety, Children's Hospital of Philadelphia, Vaccine, EV, HSPC

PHILADELPHIA, Sept. 27, 2023 /PRNewswire/ -- A readily available, inexpensive small molecule drug can improve the fitness of hematopoietic stem and progenitor cells (HSPCs) that are modified outside of the body, potentially improving the success of procedures like ex vivo gene therapy, according to a new study by researchers at Children's Hospital of Philadelphia (CHOP). The study, published in the journal Blood, showed that targeting components in the cells called extracellular vesicles (EVs) relieves the stress on cells when outside of the body, improving their performance when they are transplanted back inside.

Key Points: 
  • Hematopoietic stem cell transplantation (HSCT) involves the transfer of HSPCs from a donor to patients with both benign and malignant diseases.
  • However, maintaining the fitness of these cells outside of the body poses a challenge, and any loss of fitness introduces the risk that the cells will not properly restore the blood and immune cell system in patients.
  • One process that we now learn contributes to cell equilibrium is the secretion of EVs, which are important for delivering messages from cell to cell and maintaining proper cell function.
  • "Neutral sphingomyelinase blockade enhances hematopoietic stem cell fitness through an integrated stress response," Blood, online Sept. 12, 2023, DOI: 10.1182/blood.2023022147

CoImmune Announces Publication of Preclinical Data Demonstrating Efficacy and Specificity of CAR-CIK Cells in Acute Myeloid Leukemia

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水曜日, 7月 5, 2023
HSPC, University, Pediatrics, Toxicity, Acute myeloid leukemia, CAR, AML, Science, Cytokine release syndrome, Patient, CD33, CD123, American Society of Hematology, Risk, Growth, Toxic shock syndrome toxin-1, Leukemia, Society, Endothelium, University of Milano-Bicocca, Vaccine

DURHAM, N.C., July 5, 2023 /PRNewswire/ -- CoImmune, Inc., a clinical stage immuno-oncology company working to redefine cancer treatment using best-in-class cellular immunotherapies, today announced the publication of preclinical data demonstrating that a strategy based on the company's proprietary Chimeric Antigen Receptor-Cytokine Induced Killer (CAR-CIK) cell platform provides advantages over single-targeting CARs including improved efficacy and high specificity in a model of acute myeloid leukemia (AML). The preclinical data are published in Blood Advances, a peer-reviewed journal of the American Society of Hematology.

Key Points: 
  • The preclinical data are published in Blood Advances, a peer-reviewed journal of the American Society of Hematology.
  • The dual strategy demonstrated powerful antitumor efficacy against AML targets without any relevant toxicity on hematopoietic stem and progenitor cells (HSPCs) and endothelial cells.
  • Specifically, the researchers found:
    CD123 and/or CD33 knockout impairs leukemia growth by modulating multiple cancer pathways in a model of NPM1-mutated AML.
  • Low affinity CAR-CIK cells preserve antileukemic efficacy in vitro and improve antitumor control in vivo.

Sana Biotechnology Highlights Preclinical Data from Hypoimmune and Fusogen Platforms at the International Society for Stem Cell Research (ISSCR) 2023 Annual Meeting

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金曜日, 6月 16, 2023
Insulin, Human, Hyperglycemia, Biotechnology, HSPC, Tukey's test of additivity, Cell, IPSC, Diabetes, ISSCR, Survival, Bone marrow, Medicine, Autoimmunity, Transplant, Research and development, CAR, HIP, Immunosuppression, Patient, C-peptide, Engineering, Immune system, Annual general meeting, Mouse, Poster, SANA, International Society for Stem Cell Research, International Society, Vaccine, Research

SEATTLE, June 16, 2023 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, today announced preclinical data from six presentations, including two oral presentations, at the International Society for Stem Cell Research (ISSCR) 2023 Annual Meeting.

Key Points: 
  • SEATTLE, June 16, 2023 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, today announced preclinical data from six presentations, including two oral presentations, at the International Society for Stem Cell Research (ISSCR) 2023 Annual Meeting.
  • “Our leading presence at ISSCR showcased key preclinical data generated from our programs using our hypoimmune and fusogen platforms,” said Doug Williams, Ph.D., Sana’s President of Research and Development.
  • We also shared data demonstrating in vivo delivery of various genetic payloads to human hematopoietic stem/progenitor cells, highlighting an important capability with the fusogen platform.
  • The studies showed that these fully differentiated SC-islets can be delivered intramuscularly with robust function and without the need for bioscaffolding.