Pediatric Transplantation

Vor Bio Presents Positive Data Update on Trem-cel at HSCT² Conference

Retrieved on: 
Thursday, November 9, 2023
Aplastic anemia, Webcast, Baptist Health South Florida, Transplant, Toxicity, ASH, Acute myeloid leukemia, Leukemia, Patient, Conference, CD33, Pediatric Transplantation, AML, Doctor of Philosophy, Cytokine release syndrome, Pharmaceutical industry, Gemtuzumab ozogamicin, MD

Additionally, platelet recovery occurred at a median of 15.5 days, excluding one patient with previously documented anti-platelet antibodies (immune thrombocytopenia).

Key Points: 
  • Additionally, platelet recovery occurred at a median of 15.5 days, excluding one patient with previously documented anti-platelet antibodies (immune thrombocytopenia).
  • This data further validates Vor Bio’s approach of using transplant donor cells as CAR-T starting material that are healthy, stem-like, and exactly matched to the patient’s immune system.
  • Vor Bio management will host a live webcast today at 4:30 PM ET.
  • A replay of the webcast will be available via the investor section of the Company’s website at www.vorbio.com approximately two hours after the call’s conclusion.

Vor Bio Reports Third Quarter 2023 Financial Results and Provides Company Update

Retrieved on: 
Tuesday, November 7, 2023
Gene editing, Acute myeloid leukemia, Assistant, Doctor of Philosophy, Flexner Report, Data, Gene, HCT, Hospital, Research and development, Engineering, University, Hospital of the University of Pennsylvania, Patient, Administration, Transplant, DNA, Pediatric Transplantation, Congress, MD, G&A, Research, ASH, Clinical trial, Cytokine release syndrome, General Administration of Sport of China, Editas Medicine, CRS, Principal, Adolescence, IND, Manuscript, Harvard Medical School, Bone marrow, CRISPR, Phenotype, R, CD33, HERE, Toxicity, Associate, AML, Pharmaceutical industry, Dentistry, Vaccine, Nursing, Pediatrics, Gemtuzumab ozogamicin

CAMBRIDGE, Mass., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period ended September 30, 2023, and provided a business update.

Key Points: 
  • CAMBRIDGE, Mass., Nov. 07, 2023 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period ended September 30, 2023, and provided a business update.
  • Research & Development (R&D) Expenses: R&D expenses for the third quarter of 2023 were $27.6 million, compared to $16.9 million for the third quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses for the third quarter of 2023 were $7.7 million, compared to $7.2 million for the third quarter of 2022.
  • Net Loss: Net loss for the third quarter of 2023 was $33.2 million, compared to $23.8 million for the third quarter of 2022.

Multiple Clinical and Preclinical Presentations at ASH 2023 Highlight Vor Bio’s Novel eHSC and CAR-T Platform

Retrieved on: 
Thursday, November 2, 2023
Acute myeloid leukemia, Doctor of Philosophy, Cell, HIV disease progression rates, Division, Abstract (summary), Abstract, Head, HCT, Exposition, Hospital, CAR, Janssen, Bone marrow, CHOP, Haematopoiesis, University, Toxicity, Safety, Therapy, Transplant, MSCE, Pediatric Transplantation, Lymphoma, ASH, Clinical trial, Prognosis, Cytokine release syndrome, Biomarker, CRS, Investigator's brochure, The Relapse, University of Colorado School of Medicine, CRISPR, Phenotype, Society, National Cancer Institute, CD33, Associate, AML, Nursing, Rare-earth element, Dentistry, Vaccine, Mobile phone, Pharmaceutical industry, Child, Pediatrics, Diagnosis, Patient, MD, Leukemia

An abstract providing a clinical update from the VBP101 clinical trial (NCT048499910), a Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (VOR33) in patients with AML, was accepted by ASH for oral presentation.

Key Points: 
  • An abstract providing a clinical update from the VBP101 clinical trial (NCT048499910), a Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (VOR33) in patients with AML, was accepted by ASH for oral presentation.
  • This data supports robust neutrophil engraftment of trem-cel and provides evidence of hematologic protection from MylotargTM, a CD33-targeted antibody drug conjugate.
  • An updated data release from VBP101 is expected by the Relapse After Transplant and Cellular Therapy (HSCT²) conference taking place November 10-11, 2023.
  • Dr. Tasian commented: “The interim results from our CD33 CAR T cell immunotherapy clinical trial are very encouraging.

OmniLife Health Launches First and Only Clinical Workflow Automation Tool For Pediatric Organ Intake Management

Retrieved on: 
Tuesday, April 25, 2023
Software, Surgery, Health, Children, Data Management, Hospitals, Technology, Consumer, Estadio Akron, Communication, Kid, Pediatrics, The Action Network, National Institutes of Health, Bangladesh Technical Education Board, Pediatric Transplantation, Hospital, MD, Patient safety, Transplant, Organ transplantation, Microsoft Excel, UPMC, Pancreatic Cancer Action Network, Dentistry

To help transplant centers improve organ intake and save more children, OmniLife Health , a leader in clinical workflow automation, has expanded its award-winning FlowHawk platform to pediatric care.

Key Points: 
  • To help transplant centers improve organ intake and save more children, OmniLife Health , a leader in clinical workflow automation, has expanded its award-winning FlowHawk platform to pediatric care.
  • Called FlowHawk for Kids, this end-to-end solution automates and digitizes the transplant journey, from patient referral and waitlist management to organ transplantation and post-transplant care.
  • “There’s no shortage of organs available for children, yet many kids die on the waitlist,” said Dalton Shaull, Co-founder and CEO, OmniLife Health.
  • OmniLife Health worked with the nation’s top pediatric transplant hospitals to develop FlowHawk for Kids’ best practice workflows for every stage of the organ transplant journey.

Mesoblast Operational and Financial Highlights for Quarter Ended September 30, 2022

Retrieved on: 
Sunday, October 30, 2022
Transplant, EAP, MESO, Inflammation, GLOBE, Director, Mesoblast, Chief, MACE, MSB, Private Securities Litigation Reform Act, GVHD, Company, BLA, FDA, Executive, Patient, Fast track (FDA), IND, Industry, Australian Securities Exchange, Death, Mostafa Rashidi Suja, Heart, ASX, Survival, SEC, Noriyuki Abe, Appendix, Policy, Investigational New Drug, LVEF, Pediatric Transplantation, Forward-looking statement, Pain, McMillan, Marketing, LVAD, Regenerative medicine, Hematopoietic stem cell transplantation, MOA, Risk, Immune system, Technology, Pharmaceutical industry, Birth control, Dietary supplement, Medical imaging, Management, Cryptocurrency

NEW YORK, Oct. 30, 2022 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the fourth quarter ended September 30, 2022.

Key Points: 
  • NEW YORK, Oct. 30, 2022 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an activity report for the fourth quarter ended September 30, 2022.
  • The Company plans to have clearance from the FDA by year-end 2022 for the pivotal trial with primary endpoint 12-month reduction in pain.
  • Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets.
  • Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms.

Mesoblast Submits New Information to FDA IND File in Response to Items in the CRL to the Remestemcel-L BLA for SR-aGVHD

Retrieved on: 
Monday, October 3, 2022
MSB, IND, Private Securities Litigation Reform Act, CRL, Fast track (FDA), SWOT, Marketing, Pediatric Transplantation, Endpoint security, Investigational New Drug, Lymphatic system, McMillan, Risk, Immune system, BMT, Death, BLA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Mostafa Rashidi Suja, Australian Securities Exchange, Patient, Survival, Inflammation, Hematopoietic stem cell transplantation, SEC, Biologics license application, Food, MESO, Twitter, FDA, Forward-looking statement, GLOBE, Company, Heart, Chief, Mortality, Technology, LinkedIn, Transplant, Industry, GVHD, Blood, Pharmaceutical industry, Dentistry, Dietary supplement, Mesoblast

Remestemcel-L has been granted Fast Track Designation and BLA Priority Review from the FDA.

Key Points: 
  • Remestemcel-L has been granted Fast Track Designation and BLA Priority Review from the FDA.
  • The submission summarizes controlled data providing further evidence of remestemcel-Ls ability to save lives, said Dr. Silviu Itescu, Chief Executive of Mesoblast.
  • Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions.
  • Mesoblast has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets.

Digital Adherence Program Leads to Over 90% Medication Adherence and Improves Outcomes in Pediatric Transplant Patients

Retrieved on: 
Wednesday, May 11, 2022
University of Florida College of Medicine-Jacksonville, Florida State University, University of Florida Health, Hepatitis C, University, Student, Health, UF Health Shands Hospital, Tuberculosis, Smartphone, DOT, Pediatric Transplantation, MSW, Graduate school, Adolescence, COPD, Patient, Opioid use disorder, Asthma, Florida State University College of Social Work, Survival, Program, Medicine, Diabetes, Transplant, Hospital, U.S. News & World Report, History, Adult, Learning, Social work, Achievement, News, Assistant, Graduate, Infection, Technology, Research, Dentistry, Medical device, Doctor of Philosophy, Central Florida

BALTIMORE, May 11, 2022 /PRNewswire/ -- Video-enabled directly observed therapy (DOT) can help adolescent heart transplant recipients better stay on their medications, avoid hospitalizations, and reduce the potential of rejection, a new pilot study published in the journal Pediatric Transplantation suggests.

Key Points: 
  • Video DOT scales a model of care for medication adherence support called "directly observed therapy" with the use of mobile devices.
  • Non-adherence with immunosuppressant medications is a huge challenge for adolescent transplant patients," said pediatric transplant cardiologist and co-author Dr. Dipankar Gupta of the University of Florida's Congenital Heart Center.
  • Those who completed the program had a 90.1% adherence rate, submitting 1,211 videos out of 1,344 expected.
  • Providers or emocha's clinician-led Adherence Solutions team use a secure web portal to assess adherence and engage with patients.

CareDx Partners with the North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) Organization in Launching Prospective, Multi-Center Pediatric Kidney Transplant Study

Retrieved on: 
Monday, December 13, 2021
Dialysis, Solution, Population, Collaborative Study on the Genetics of Alcoholism, SAN, Nasdaq, Kidney, Caregiver, Pediatric Transplantation, Transplant, CEO, Research, Patient, DNA, Chronic kidney disease, MD, Natural history, GLOBE, Partnership, Precision medicine, Dentistry

Data will be collected from participating centers by NAPRTCS for five years or until the study concludes the utility of dd-cfDNA, whichever is sooner.

Key Points: 
  • Data will be collected from participating centers by NAPRTCS for five years or until the study concludes the utility of dd-cfDNA, whichever is sooner.
  • The objectives of the study are to characterize the use of dd-cfDNA testing and measure longitudinal outcomes following transplantation.
  • We thank CareDx for their ongoing collaboration with NAPRTCS and look forward to the results of this study and the guidanceit providesus in caring for theseyoungpatients.
  • CareDx offers testing services, products, and digital healthcare solutions along the pre- and post-transplant patient journey and is the leading provider of genomics-based information for transplant patients.