Clinical research
Verve Therapeutics dosed its first patient with VERVE-101, a one-time treatment that aims to permanently lower low-density lipoprotein (LDL) cholesterol in patients and prevent heart disease.
uniQure gave a 12-month update on AMT-130, the first-ever AAV gene therapy for HD to enter clinical trials. BioSpace spoke with the company’s president of R&D, Dr. Ricardo Dolmetsch.
Exelixis’ Cabometyx, in combination with two BMS-owned drugs, hit the primary endpoint of progression-free survival but missed the secondary endpoint of overall survival in renal cell carcinoma.
Tonix Pharmaceuticals announced a mid-stage study for major depressive disorder, which followed the June disclosure of a Phase II study for a cocaine intoxication therapy.
The experimental drug, being developed to treat immunoglobulin A nephropathy, is IONIS-FB-L Rx, which hit the mark in a Phase II study showing a change in urinary protein after 29 weeks of treatment.
Sanofi announced positive data for its therapeutic, fitusiran, for treating patients with hemophilia A and B, as well as efanesoctocog alfa therapy for treating hemophilia A.
The study was assessing the investigational drug enoblituzumab as part of a combination therapy for head and neck cancer.
Weeks after BridgeBio presented positive biomarker data in Canavan Disease, gene therapy company Myrtelle added its own data to the emerging research space.
Imago’s early failed experiments investigating the potential of LSD1 in sickle cell disease and thalassemia have turned out to be fortuitous for patients with myeloproliferative neoplasms.
Seagen recently presented positive pivotal Phase II data from its trial of Tukysa (tucatinib) in combination with trastuzumab in HER2-positive metastatic colorectal cancer.
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