Hematopoietic stem cell transplantation

Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Retrieved on: 
Thursday, April 18, 2024
Community, Eurostat, Civil service commission, Diagnosis, CD7, Graft-versus-host disease, Cell, Bone marrow, Disease, Patient, Committee, Extrapyramidal system, Tissue, Knowledge, Antibody, Regulation, EMA, IRIS, Protein, CD3, BV, European, Skin, WT1, COMP, Oxygen, Immune system, European Commission, Safety, Hematopoietic stem cell transplantation, Stomach, Liver, Pharmaceutical industry

Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Key Points: 


Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positive

Akari Therapeutics Reports Full-Year 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, April 1, 2024
Diagnosis, Civil service commission, Research, AATD, Growth, Akari, C5, Patient, Choroidal neovascularization, Sale, B4, PRV, Mortality, Hot game, Clinical trial, Therapy, Priority review, Safety, NEI, European Commission, ADC, Geographic atrophy, Hematopoietic stem cell transplantation, CNV, Pharmaceutical industry

BOSTON and LONDON, April 01, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the full year 2023 as well as recent company highlights.

Key Points: 
  • BOSTON and LONDON, April 01, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the full year 2023 as well as recent company highlights.
  • The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX.
  • Akari is continuing to recruit patients into the Part A portion of the Phase 3 clinical trial that has treated 10 patients to date.
  • During 2023, Akari also advanced the long-acting version of nomacopan (PASylated nomacopan) into the final stages of pre-clinical development as a treatment for geographic atrophy (GA).

Molecular Partners Reports Corporate Highlights From Q4 2023 and Key Financials for Full Year 2023

Retrieved on: 
Thursday, March 14, 2024
CD33, GSK, Patient, Macrophage, Society, CD3, CD47, DLL3, Therapy, Molecular Partners, TME, Partner, DRS, ASH, AML, SNMMI, Partnership, RDT, Radionuclide, Tetra, Novartis, HSCT, MHS, FTE, Exposition, Delta, Safety, SITC, Amgen, Hematopoietic stem cell transplantation, FAP, Disease, Nuclear medicine, Immunotherapy, District court, Neoplasm, EANM, Alpha particle, JNJ, Tumor microenvironment, Gene family, CD70, AACR, CD40, CHF, CD123, NK, Engineering, Antigen, Molecule, Annual general meeting, Toxicity, CD16, B cell, Conference, MBA, DARPin, Data, Molecular imaging, Pharmaceutical industry

ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- Ad hoc announcement pursuant to Art. 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced its corporate highlights and audited financial results for the full year 2023.

Key Points: 
  • 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced its corporate highlights and audited financial results for the full year 2023.
  • “2023 was a year of successful innovation and execution on our strategy, focusing on novel mechanisms that we believe only DARPin therapies can deliver.
  • In addition to the above updates, Molecular Partners continued to progress its RDT portfolio of projects in partnership with Novartis.
  • In the financial year 2023, Molecular Partners recognized total revenues and other income of CHF 7.0 million (2022: CHF 189.6 million) and incurred total expenses of CHF 68.1 million (2022: CHF 73.0 million).

Omeros Corporation Reports Fourth Quarter and Year-End 2023 Financial Results

Retrieved on: 
Monday, April 1, 2024
Mental Health, Research, Clinical Trials, Biotechnology, Other Health, Health, Pharmaceutical, Science, Oncology, PIN, PNH, Patient, Sale, CUD, Graft-versus-host disease, Investment, Clinical trial, Hemolysis, Calcineurin, Emory University, ASH, Risk, L-DOPA, Thumbay Hospital, Behavior informatics, Trial of the century, Myelodysplastic syndrome, Cancer, Safety, Transplant, LID, DRI, BLA, Corporation, Paroxysmal nocturnal hemoglobinuria, Tardive dyskinesia, Parkinson's disease, Publication, DSM-IV codes, Retirement, FDA, PDE7, LDH, Hematology, Mortality, Hematopoietic stem cell transplantation, Bone marrow failure, Abstract, Society, Pharmacology, Pharmaceutical industry

Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.

Key Points: 
  • Net loss from continuing operations was $39.3 million for the 2023 fourth quarter compared to $51.7 million for the prior quarter.
  • Total operating expenses for the fourth quarter of 2023 were $39.8 million compared to $48.2 million for the third quarter of 2023.
  • Interest expense during the fourth quarter of 2023 was $7.1 million compared to $7.9 million during the prior quarter.
  • During the fourth quarter of 2023, we earned $3.4 million in interest and other income compared to $4.4 million in the third quarter.

Forge Biologics’ Novel AAV Gene Therapy FBX-101 for Patients with Krabbe Disease is Granted UK’s Innovation Passport Designation

Retrieved on: 
Tuesday, March 19, 2024
Biotechnology, Pharmaceutical, Genetics, Health, Infant, AAV, MAA, Disease, Patient, FBX, Innovation, HSCT, EMA, Krabbe disease, GMP, European Medicines Agency, MHRA, Education minister, Clinical trial, Safety, FDA, Neurodegenerative disease, Hematopoietic stem cell transplantation, Medication, Pharmaceutical industry

FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two.

Key Points: 
  • FBX-101 was created for the treatment of patients with Krabbe disease, a rare neurodegenerative disease that is usually fatal in untreated patients by age two.
  • FBX-101 has been granted Orphan Drug Designation and Priority Medicines (PRIME) designation by the European Medicines Agency (EMA), allowing Forge to advance and expedite the development of FBX-101 as the leading worldwide gene therapy for patients with Krabbe disease.
  • In the US, the FDA has granted FBX-101 Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation.
  • Through this designation Forge has expanded global regulatory experience to include interactions with the MHRA in the UK.

Global Cord Blood & Tissue Banking Industry Market Size, Segmentation & Forecast Report 2024 - ResearchAndMarkets.com

Retrieved on: 
Friday, March 15, 2024
Health, Biotechnology, Catalent, Transplant, Cell, Patient, UCB, LifeCell International, Microsoft Exchange Server, Organization, Regenerative medicine, Cryo-Cell International, Spectrum, Disorder, Umbilical cord, Population growth, Stemcell Technologies, Certification, HSCT, NIH, GMP, Cord blood, The Cooper Companies, Safety, Hematopoietic stem cell transplantation, Artivion, DSM-IV codes, Tissue, Research, Acquisition, Cancer, Leukemia, CBR, Cryopreservation, Cerebral palsy, FDA, CBUS, Sickle cell disease, ClinicalTrials.gov, Registry, Amniotic fluid, Hearing loss, GCBC, MNC, Geography

The "Global Cord Blood & Tissue Banking Industry Report - Market Size, Segmentation, & Forecasts, 2024" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Cord Blood & Tissue Banking Industry Report - Market Size, Segmentation, & Forecasts, 2024" report has been added to ResearchAndMarkets.com's offering.
  • There are now over 800,000 cord blood units stored in public cord blood banks and more than 6.75 million cord blood and tissue units stored within private banks worldwide.
  • Today, nearly all U.S. cord blood banks and approximately one-third of global cord blood banks offer cord tissue storage.
  • The report presents findings on the following topics:
    Rate per cord blood unit in the U.S. and Europe
    Number of clinical trials, number of published scientific papers, and amount of NIH funding for cord blood research
    Number and types of patents for cord blood, cord tissue, and placental products
    The report presents a comprehensive analysis of the global cord blood and tissue banking market.

MaaT Pharma Announces Positive Review from the DSMB on the Ongoing Phase 1 Clinical Trial Evaluating MaaT033 in Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Thursday, February 29, 2024
Research, Genetics, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, Oncology, Hematopoietic stem cell transplantation, SLA, Microbiota, HSCT, Safety, MET, Bone marrow, Charcot disease, Patient, Clinical trial, Survival, DSMB, Cancer, Pharmaceutical industry

The DSMB recommended that the trial continue without modifications.

Key Points: 
  • The DSMB recommended that the trial continue without modifications.
  • The DSMB, composed of 4 independent experts, including an ALS patient association representative, concluded that safety was good.
  • More precisely, it should be noted that no serious or severe adverse events were observed, and no infectious events could be related to MaaT033.
  • The preliminary results reinforce confidence in the safety of MaaT033, a drug candidate produced by combining the microbiota from multiple donors using a "pooling" process.

Orca Bio Presents Promising Data on Orca-T in Two Oral Presentations at the 2024 Tandem Meetings of ASTCT® and CIBMTR®

Retrieved on: 
Friday, February 23, 2024
Research, Infectious Diseases, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, Oncology, MAC, AML, Syndrome, MD, Assistant, Physician, RFS, NRM, MMF, Weill Cornell Medicine, Risk, Thiotepa, Graft-versus-host disease, Bone marrow, Society, BFT, MDS, CNI, Patient, Hematopoietic stem cell transplantation, NewYork-Presbyterian Hospital, Survival, Transplant, Acute myeloid leukemia, Weill Cornell Medical Center, Cancer, Busulfan, Pharmaceutical industry

In the first of two oral presentations, findings from a retrospective analysis compared historical outcomes between Orca-T and a standard of care allogeneic stem cell transplant (alloHSCT) plus post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis in patients with hematologic malignancies receiving myeloablative conditioning (MAC).

Key Points: 
  • In the first of two oral presentations, findings from a retrospective analysis compared historical outcomes between Orca-T and a standard of care allogeneic stem cell transplant (alloHSCT) plus post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis in patients with hematologic malignancies receiving myeloablative conditioning (MAC).
  • Within the non-randomized cohorts of varying conditioning regimens and disease types, Orca-T demonstrated favorable results across clinical outcomes including relapse-free survival (RFS), non-relapse mortality (NRM) and overall survival (OS).
  • The analysis included patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) who had a median follow-up time of 15 months and 12 months in the Orca-T and PTCy cohorts, respectively.
  • The outcomes at 12 months are summarized in the table below:

CareDx Showcases Digital Solutions and Pipeline in Cellular Transplant and Therapy Monitoring at the 2024 Tandem Meetings

Retrieved on: 
Wednesday, February 21, 2024
Technology, Health, General Health, Public Relations, Investor Relations, Communications, Health Technology, Medical Devices, Genetics, Internet, Mobile, Wireless, Clinical Trials, Hematopoietic stem cell transplantation, Caregiver, Patient, AML, Digital Solutions, ALL, HLA, Chimera, HCT, High, Medicine, Transplant, Prognosis, Cell, HA2, Nucleic acid thermodynamics, MDS, University, Antigen, HA1, Accuracy and precision, Medical device

“Cellular transplant centers are recognizing the value of our digital and patient monitoring solutions, and we look forward to sharing our latest advancements as we work to expand our presence in this dynamic ecosystem,” said Kashif Rathore, Chief of Patient and Digital Solutions at CareDx.

Key Points: 
  • “Cellular transplant centers are recognizing the value of our digital and patient monitoring solutions, and we look forward to sharing our latest advancements as we work to expand our presence in this dynamic ecosystem,” said Kashif Rathore, Chief of Patient and Digital Solutions at CareDx.
  • CareDx will be showcasing its broad portfolio of digital health solutions that address needs in the cellular transplant and therapy ecosystem including workflow management, regulatory reporting, medication management, and remote patient monitoring.
  • Product solution areas include:
    Ottr® Cellular: Designed as a workflow management solution for hematopoietic cellular transplant and cellular therapy centers.
  • Poster 299: Analytical Validation of AlloCell cfDNA - a Highly Sensitive, Precise, and Accurate Cell-Free DNA-Based Test for Allogeneic Cell Therapy Monitoring.

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.