Drug Development

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Mixed headlines have plagued the cell and gene therapy space of late. We believe that a renewed case of optimism is not only warranted but essential if these therapies are to reach their full potential.
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
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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.
Scientists found that SARS-CoV-2 spike protein in the plasma could be a hallmark of long COVID, suggesting that the virus can linger in the body who suffer from prolonged symptoms.
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.
In a filing with the SEC, Abbvie’s decision to terminate came after the two firms jointly conducted a review of their next steps for the drug.
Adverum Biotechnologies is cutting 78 jobs after announcing it had successfully amended its Investigational New Drug application for ADVM-022.to treat wet age-related macular degeneration.