Phase II
Nanoscope Therapeutics will make two presentations at the 2022 ASRS meeting on their gene therapy programs to treat degenerative retinal diseases, retinitis pigmentosa and Stargardt 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.
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.
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.
Dynacure has ended its Unite-CNM Phase I/II trial on DYN101, an investigational therapy for the treatment of myotubular and centronuclear myopathies.
Ayala announced positive data for its desmoid tumor treatment, while Vertex and Dyne announced the FDA had lifted the hold on their clinical studies.
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