Liver

Calliditas Therapeutics to Present Nefecon Data at the ISN World Congress of Nephrology April 13 - 16 in Buenos Aires

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Monday, April 8, 2024

The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.

Key Points: 
  • The ISN World Congress of Nephrology will be held in Buenos Aires, Argentina April 13-16, 2024.
  • Presentations will highlight the eGFR results found in patients on Nefecon as well as the data on quality of life during the trial.
  • There will also be a presentation on the subanalysis evaluating benefits of Nefecon for patients with lower levels of UPCR.
  • The congress will include a symposium, Evolving Landscape of eFGR and Proteinuria Surrogate Markers in IgA Nephropathy, moderated by KOL Richard Lafayette, M.D., F.A.C.P.

Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia [...]

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia A, 13/04/2022 Positive

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia A, 13/04/2022 Positive

Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

Key Points: 


Orphan designation: Alisitol, retinol palmitate, zinc gluconate Treatment of microvillus inclusion disease, 13/11/2020 Positive

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII Treatment of haemophilia A, 25/05/2018 Withdrawn

Orphan designation: Florilglutamic acid (18F) Diagnosis of hepatocellular carcinoma, 21/03/2016 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Florilglutamic acid (18F) Diagnosis of hepatocellular carcinoma, 21/03/2016 Withdrawn

Key Points: 


Orphan designation: Florilglutamic acid (18F) Diagnosis of hepatocellular carcinoma, 21/03/2016 Withdrawn

Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene Treatment of primary hyperoxaluria type 1, 21/03/2012 Withdrawn

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024

Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Key Points: 


Orphan designation: Adeno-associated viral vector containing porphobilinogen deaminase gene Treatment of acute intermittent porphyria, 29/04/2009 Withdrawn

Chemomab Awarded New European Patent for CM-101, Its First-in Class Monoclonal Antibody in Phase 2 Clinical Development for Primary Sclerosing Cholangitis

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Monday, March 25, 2024

TEL AVIV, Israel, March 25, 2024 (GLOBE NEWSWIRE) -- Chemomab Therapeutics Ltd. (Nasdaq: CMMB), (Chemomab), a clinical stage biotechnology company developing innovative therapeutics to treat rare fibro-inflammatory diseases with high unmet need, today reported that the European Patent Office has granted a new patent for CM-101, Chemomab’s first-in-class monoclonal antibody that neutralizes CCL24, a novel disease target that has been shown to play a critical role in the processes that drive fibrosis and inflammation. CM-101 is currently being assessed in the global Phase 2 SPRING trial for the treatment of primary sclerosing cholangitis (PSC). Patient enrollment in the trial has been completed, with a topline data readout expected midyear 2024.

Key Points: 
  • CM-101 is currently being assessed in the global Phase 2 SPRING trial for the treatment of primary sclerosing cholangitis (PSC).
  • Patient enrollment in the trial has been completed, with a topline data readout expected midyear 2024.
  • The new European patent covers the use of CM-101 and sequence-related anti-CCL24 antibodies for the treatment of hepatic (liver) diseases, including PSC.
  • Unlike other drugs in development for PSC, CM-101 has a unique dual mechanism of action that simultaneously blocks fibrosis and inflammation.

AIM ImmunoTech Announces Publication of Positive Findings from a Study Evaluating Ampligen® in the Treatment of Pancreatic Cancer in the Journal Clinical Cancer Research

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Monday, March 25, 2024

The manuscript titled “ Rintatolimod in Advanced Pancreatic Cancer enhances Anti-Tumor Immunity through Dendritic Cell-Mediated T Cell Responses ,” appears in the journal Clinical Cancer Research, one of oncology’s most prestigious journals.

Key Points: 
  • The manuscript titled “ Rintatolimod in Advanced Pancreatic Cancer enhances Anti-Tumor Immunity through Dendritic Cell-Mediated T Cell Responses ,” appears in the journal Clinical Cancer Research, one of oncology’s most prestigious journals.
  • Ampligen is a dsRNA product candidate that acts via the TLR-3 receptor present on several immune cells, epithelial cells and tumors.
  • Post-Ampligen, the increased peripheral abundance of BTLA+XCR1+ cDC1s and CD4+SELL+ T cells correlated with improved clinical outcomes.
  • Additionally, the changes in the tumor microenvironment we see in pancreatic cancer are similar to those we have seen in triple-negative breast cancer, ovarian cancer and colorectal cancer metastatic to the liver.