UCBT

Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote

Retrieved on: 
Monday, January 29, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Mortality, Death, HSCT, Patient, Legislation, GALC, Advisory Committee, AAV, Psychosine, ESGCT, Data, Gene, DSM-IV codes, Neurodegenerative disease, Disease, Safety, Central nervous system, Sphingolipid, Physician, Krabbe disease, Cell, Human services, Parent, Nervous system, Clinical trial, Forge, Peripheral nervous system, Hematopoietic stem cell transplantation, Brain, UCBT, Pharmaceutical industry

Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.

Key Points: 
  • Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.
  • Previously published data have demonstrated that patients with Krabbe treated with HSCT demonstrate increased lifespan and stabilization of neurodegenerative disease in the central nervous system.
  • FBX-101, an investigational adeno-associated viral (AAV) gene therapy, has been designed to address the peripheral nerve disease not corrected by HSCT.
  • As a result, after a year of patient and foundation advocacy, Krabbe disease is again being voted on for potential inclusion on the RUSP.

Forge Biologics Reports Positive Clinical Data on Brain Development and Motor Function from the RESKUE Novel Phase 1/2 Gene Therapy Trial in Patients with Krabbe Disease at the SSIEM Annual Symposium

Retrieved on: 
Tuesday, August 30, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, GALC, Muscle weakness, Locomotion, Hematopoietic stem cell transplantation, Lipid, Peripheral neuropathy, Lists of diseases, Central nervous system, Krabbe disease, Brain, Entrepreneurship, Disease, Death, Peripheral nervous system, CGMP, Cell, Multimedia, Neurodegeneration, CET, Irritability, Hearing loss, Psychosine, UCBT, Clinical trial, Environment, Society, AAV, Patient, Pharmaceutical industry, Vaccine, Krabbe, Forge, Hearth, Metabolism

The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.

Key Points: 
  • The data will be presented on August 31, 2022, at the annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) in Freiburg, Germany.
  • The first patients data from the RESKUE clinical trial demonstrate that intravenous FBX-101 following UCBT has been safe and well tolerated through Day 180.
  • Notably, the data demonstrate an absence of humoral immune response against the vector and significantly increased GALC enzyme activity.
  • Through Day 180, the patient exhibited improved motor activity and normal brain development compared to previously reported transplanted patients with Krabbe disease.

Gamida Cell Presents Updated One-Year Post-Transplant Follow Up Data from Phase 3 Study of Omidubicel at 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings

Retrieved on: 
Wednesday, April 27, 2022
Biotechnology, Health, Clinical Trials, Cardiology, Oncology, UCB, HSCT, Transplant, Poster, Duke Cancer Institute, Aplastic anemia, U.S. Securities and Exchange Commission, COVID-19, Lymphatic system, Twitter, Security (finance), Infection, Private Securities Litigation Reform Act, Therapy, Risk, OS, Patient, Cell, LinkedIn, Trial of the century, Mortality, Technology, Phenotype, SEC, TCT, Safety, Instagram, NK, Hematopoietic stem cell, NAM, Facebook, FDA, BLA, UCBT, Hematology, B cell, Graft-versus-host disease, Cancer, Pharmaceutical industry, Vaccine

The data showed sustained clinical benefits in the first-year post-transplant with omidubicel, as demonstrated by significant reduction in infectious complications.

Key Points: 
  • The data showed sustained clinical benefits in the first-year post-transplant with omidubicel, as demonstrated by significant reduction in infectious complications.
  • There was a continued trend toward improved OS in favor of the omidubicel arm over time (73% vs. 60%).
  • The overall and sustained clinical benefit of omidubicel makes it an important addition to the options for allogeneic HSCT.
  • For additional information on Gamida Cell, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn , Twitter , Facebook or Instagram at @GamidaCellTx.