Hematopoietic stem cell transplantation

Viracta Therapeutics to Host R&D Day Highlighting Nana-val in Epstein-Barr Virus (EBV)-Associated Cancers

Retrieved on: 
Wednesday, October 4, 2023
Lymphoma, PTCL, Peripheral T-cell lymphoma, Hematopoietic stem cell transplantation, Initiation, Creatinine, DLBCL, Thomas Jefferson University, Valganciclovir, Oncology, EDT, B-cell lymphoma, Stage 2, Nasopharyngeal carcinoma, Bone marrow, CRR, 2009 Tour de France, Stage 1 to Stage 11, SDD, Dor, Patient, Division, CR, Toxic leukoencephalopathy, NPC, R, FDA, Therapy, Stomach, Engagement, ORR, EBV, Pharmaceutical industry, Fine chemical, Medical device, Medical imaging, Leiomyosarcoma, Lymphoepithelioma

SAN DIEGO, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Viracta Therapeutics, Inc. (Nasdaq: VIRX), a clinical-stage precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, today announced that it plans to highlight new preliminary clinical and preclinical data from studies of nanatinostat and valganciclovir (Nana-val), its all-oral investigational therapy targeting Epstein-Barr virus (EBV)-associated cancers, during an R&D Day today, Wednesday, October 4, 2023, at 8:00 a.m. EDT.

Key Points: 
  • “The clinical responses and favorable safety profile observed in multiple relapsed or refractory EBV-positive lymphoma patient populations continue to be encouraging.
  • In addition, the R&D Day will feature presentations by expert key opinion leaders who will discuss the high unmet medical needs of EBV-associated lymphomas.
  • Enrollment completed through the fifth dose level of the Phase 1b dose escalation portion of the trial without any dose-limiting toxicities reported.
  • A live video webcast of the presentation will be available here and on the Investors section of the Viracta website under " Events and Webcasts ".

Nexcella Announces Complete Response in 9th Relapsed/Refractory AL Amyloidosis Patient in NXC-201 Clinical Trial at IMS 20th Annual Meeting

Retrieved on: 
Tuesday, October 3, 2023
Travel, Hematopoietic stem cell transplantation, AUC, AL amyloidosis, MRD, Hadassah Medical Center, AL, Patient, IMS, Amyloidosis, LOS, Daratumumab, BCMA, Pharmaceutical industry, Immunotherapy

LOS ANGELES, Oct. 03, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced presentation of additional AL Amyloidosis clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, at an oral presentation at the 20th International Myeloma Society Annual (IMS) Meeting being held in Athens, Greece on September 27-30 2023. One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update. All patients were DARZALEX® (daratumumab) combination therapy relapsed/refractory and experienced a median of 6 earlier treatments that failed to stop worsening of disease (lines of therapy) prior to receiving NXC-201.

Key Points: 
  • One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update.
  • “NXC-201’s 100% response rate in relapsed/refractory AL amyloidosis patients, including t(11;14) and cardiac involved, indicates a potential broad mechanism of action.
  • Median follow-up was 7.3 months (range: 2.5 – 16.5 months) as of the September 20, 2023 data cutoff.
  • Data highlights:
    Best responder had a duration of response of 19.2 months as of the data cutoff of September 20, 2023, with response ongoing
    For the 7 relapsed/refractory AL Amyloidosis patients with cardiac involvement:
    4 patients with t(11;14) relapsed/refractory AL Amyloidosis:
    The 20th IMS AL Amyloidosis presentation can be accessed on the Nexcella corporate website at this link: https://nexcella.com/publications/
    20th International Myeloma Society Annual Meeting, Athens, Greece

Immix Biopharma Announces Complete Response in 9th Relapsed/Refractory AL Amyloidosis Patient in NXC-201 Clinical Trial at IMS 20th Annual Meeting

Retrieved on: 
Tuesday, October 3, 2023
Travel, Hematopoietic stem cell transplantation, AUC, AL amyloidosis, Thirty Days, MRD, Hadassah Medical Center, AL, Patient, IMS, Amyloidosis, LOS, Daratumumab, BCMA, Pharmaceutical industry, Hospital, Immunotherapy

One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update.

Key Points: 
  • One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update.
  • “NXC-201’s 100% response rate in relapsed/refractory AL amyloidosis patients, including t(11;14) and cardiac involved, indicates a potential broad mechanism of action.
  • Median follow-up was 7.3 months (range: 2.5 – 16.5 months) as of the September 20, 2023 data cutoff.
  • Data highlights:
    Best responder had a duration of response of 19.2 months as of the data cutoff of September 20, 2023, with response ongoing
    For the 7 relapsed/refractory AL Amyloidosis patients with cardiac involvement:
    4 patients with t(11;14) relapsed/refractory AL Amyloidosis:
    The 20th IMS AL Amyloidosis presentation can be accessed on the ImmixBio corporate website at this link: https://immixbio.com/pipeline/#publications
    20th International Myeloma Society Annual Meeting, Athens, Greece

Edward Owusu Kwarteng, Ph.D., BSc, MSc, Honored With The 2023 ASH-CIBMTR-ASTCT Career Development Award

Retrieved on: 
Monday, October 2, 2023
University of Texas Southwestern Medical Center, National Marrow Donor Program, University, MCW, ASH, Senior, Virology, Medical college, Exposition, Transplant, Cell, Stem cell, Bachelor of Science, Health, University of Cape Coast, Glycolysis, Metabolism, MD, Patient, NMDP, Master of Science, Diversity, Medical College of Wisconsin, Inclusion, INRS, B cell, Society, Synthetic biology, Hematopoietic stem cell transplantation, Nursing, Hematology, Research

This award is exclusively for clinical and translational researchers who plan to practice hematopoietic stem cell transplantation and cellular therapy clinical patient care.

Key Points: 
  • This award is exclusively for clinical and translational researchers who plan to practice hematopoietic stem cell transplantation and cellular therapy clinical patient care.
  • The CIBMTR is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW).
  • The award is part of a program to increase racial and ethnic diversity in the next generation of medical professionals.
  • Dr. Kwarteng receives registration for the 2023 Tandem Meetings: Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR.

Nexcella Announces 72-Patient NXC-201 Clinical Data at the IMS 20th Annual Meeting, 95% Overall Response Rate in Multiple Myeloma

Retrieved on: 
Monday, October 2, 2023
Hematopoietic stem cell transplantation, Hospital, Multiple myeloma, AL amyloidosis, Cell, Clinical data repository, Hadassah Medical Center, Patient, Data, IMS, Amyloidosis, LOS, IND, BCMA, Safety, Pharmaceutical industry, Medical device, Immunotherapy

95% overall response rate observed in relapsed/refractory multiple myeloma patients not previously treated with BCMA-targeted therapy.

Key Points: 
  • 95% overall response rate observed in relapsed/refractory multiple myeloma patients not previously treated with BCMA-targeted therapy.
  • “We feel these overall response rate data are compelling as 72% was the overall response rate reported for ABECMA from its pivotal 100-patient KarMMa trial in relapsed/refractory multiple myeloma.
  • “The waiting lists at major academic medical centers in the United States for multiple myeloma CAR-Ts reflect unmet medical need.
  • This could meaningfully reduce hospital costs and enable NXC-201 CAR-T therapy to be widely adopted.”
    The 20th IMS multiple myeloma poster can be accessed on the Nexcella corporate website at this link: https://nexcella.com/publications/
    Nexcella Announces 72-Patient NXC-201 Clinical Data at the IMS 20th Annual Meeting, 95% Overall Response Rate in Multiple Myeloma
    20th International Myeloma Society Annual Meeting, Athens, Greece

Immix Biopharma Announces 72-Patient NXC-201 Clinical Data at the IMS 20th Annual Meeting, 95% Overall Response Rate in Multiple Myeloma

Retrieved on: 
Monday, October 2, 2023
Hematopoietic stem cell transplantation, Hospital, Multiple myeloma, AL amyloidosis, Cell, Clinical data repository, Hadassah Medical Center, Patient, Data, IMS, Amyloidosis, LOS, IND, BCMA, Safety, Pharmaceutical industry, Medical device, Immunotherapy

LOS ANGELES, Oct. 02, 2023 (GLOBE NEWSWIRE) -- Immix Biopharma Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us”) today announced the presentation of updated data from the ongoing Phase 1b/2 NEXICART-1 (NCT04720313) study of its novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201. The updated dataset consists of 63 multiple myeloma patients (including data on 13 new patients and continued follow-up data on 50 previously enrolled patients), at a poster presentation at the 20th International Myeloma Society Annual Meeting in Athens, Greece on September 27-30, 2023 (72 patient count includes 9 AL Amyloidosis patients which will be announced separately).

Key Points: 
  • 95% overall response rate observed in relapsed/refractory multiple myeloma patients not previously treated with BCMA-targeted therapy.
  • “We feel these overall response rate data are compelling as 72% was the overall response rate reported for ABECMA from its pivotal 100-patient KarMMa trial in relapsed/refractory multiple myeloma.
  • “The waiting lists at major academic medical centers in the United States for multiple myeloma CAR-Ts reflect unmet medical need.
  • This could meaningfully reduce hospital costs and enable NXC-201 CAR-T therapy to be widely adopted.”
    The 20th IMS multiple myeloma poster can be accessed on the ImmixBio corporate website at this link: https://immixbio.com/pipeline/#publications
    Immix Biopharma Announces 72-Patient NXC-201 Clinical Data at the IMS 20th Annual Meeting, 95% Overall Response Rate in Multiple Myeloma
    20th International Myeloma Society Annual Meeting, Athens, Greece

Akari Therapeutics Reports First Half 2023 Financial Results and Highlights

Retrieved on: 
Friday, September 29, 2023
Consensus, Manchester University NHS Foundation Trust, EBMT, Male, PRV, Ministry of Health (Poland), Medical device, IMPD, Civil service commission, Leukotriene B4, Blood, Priority review, Marketing, Health care, Clinical trial, Pediatrics, Royal Manchester Children's Hospital, Geographic atrophy, Committee on Herbal Medicinal Products, CTA, Medication, GA, Type, HSCT, Hot game, Hematopoietic stem cell transplantation, European Commission, Patient, NHS foundation trust, Akari, CFO, Diagnosis, Poster, Hospital, Regulatory affairs, European Society for Medical Oncology, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, LTB4, Food, Autoimmunity, Safety, C5, Inflammation, Pharmaceutical industry, Nursing

Adult study design will be an important topic of discussion during a Type C meeting with the U.S. Food and Drug Administration (FDA) scheduled for November 15, 2023.

Key Points: 
  • Adult study design will be an important topic of discussion during a Type C meeting with the U.S. Food and Drug Administration (FDA) scheduled for November 15, 2023.
  • Akari was granted orphan drug designation from the European Commission for treatment in hematopoietic stem cell transplantation.
  • Akari also selected Wacker Biotech GmbH as the manufacturing partner to support production of PAS-nomacopan for use in clinical trials.
  • Akari appointed experienced life sciences executive Wendy DiCicco as interim Chief Financial Officer (CFO).

CHOP Researchers Improve Fitness of Cells Used in Cell Transplants

Retrieved on: 
Wednesday, September 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

Akari Therapeutics to Host Key Opinion Leader Webinar on Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy (HSCT-TMA) and the Potential of Nomacopan to Address Significant Unmet Needs

Retrieved on: 
Tuesday, September 19, 2023
Manchester University NHS Foundation Trust, Hematopoietic stem cell transplantation, Pediatrics, University, Leukotriene B4, Royal Manchester Children's Hospital, Hot game, MD, Division, NHS foundation trust, University of Manchester, Akari, Diagnosis, Hospital, Health, Therapy, LTB4, C5, Pharmaceutical industry

The potential of Akari’s investigational nomacopan, a novel bispecific inhibitor of both complement C5 and leukotriene B4 (LTB4), as a potential treatment for HSCT-TMA also will be discussed.

Key Points: 
  • The potential of Akari’s investigational nomacopan, a novel bispecific inhibitor of both complement C5 and leukotriene B4 (LTB4), as a potential treatment for HSCT-TMA also will be discussed.
  • The Akari management team will present an overview of nomacopan and an update on the two registrational Phase 3 clinical trials investigating nomacopan as a potential treatment option for pediatric and adult HSCT-TMA.
  • A live question and answer session will follow the formal presentations.
  • A replay will be available after the event.

Abu Dhabi Stem Cells Center Successfully Manufactures UAE's First CAR-T Cell to Treat an 11-Year Old Leukemia Patient

Retrieved on: 
Monday, September 18, 2023
SKMC, Workforce, GMP, Heart, Center of excellence, Hematopoietic stem cell transplantation, Murad, Neoplasm, Physician, Bone marrow, ABU, Ferid Murad, Research, Cell, CAR, Department of Health (Abu Dhabi), Laboratory, Patient, Good manufacturing practice, Child, Engineering, Immune system, Excellency, Leukemia, Health, Safety, Family, Sheikh Khalifa Medical City, Vaccine, Nursing, Pharmaceutical industry

ABU DHABI, UAE, Sept. 18, 2023 /PRNewswire/ -- Abu Dhabi Stem Cells Center (ADSCC), a PureHealth subsidiary and the largest healthcare platform in the Middle East, has successfully manufactured the UAE's first CAR-T cells to treat an 11-year-old boy with leukemia.

Key Points: 
  • ABU DHABI, UAE, Sept. 18, 2023 /PRNewswire/ -- Abu Dhabi Stem Cells Center (ADSCC), a PureHealth subsidiary and the largest healthcare platform in the Middle East, has successfully manufactured the UAE's first CAR-T cells to treat an 11-year-old boy with leukemia.
  • The child, Murad, was diagnosed with leukemia more than five years ago and went into remission, but unfortunately, the leukemia returned approximately six months ago.
  • The treatment took five weeks, during which Murad was admitted at ADSCC, the only accredited Centre of Excellence in Hematopoietic Stem Cell Transplantation in Abu Dhabi.
  • Physicians withdrew the cells from the patient and genetically modified his immune cells in the laboratory to fight the cancer cells after intravenously injecting them into the patient.