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VisitWhich gene therapy will be first approved for Diamond-Blackfan anemia by end of 2025?
Universal gene therapy • 33%
Mutation-specific gene therapy • 33%
No approval • 34%
FDA announcements and press releases
CAR-T Therapy Shows Promise for Lupus Amid Serious Side Effects
Nov 30, 2024, 01:46 PM
Chimeric Antigen Receptor T-cell (CAR-T) therapy, initially approved for certain blood cancers, is showing promise in early tests for treating autoimmune diseases such as lupus. This innovative therapy is designed to eliminate entire classes of immune cells, potentially addressing conditions like lupus. However, the treatment poses risks of serious side effects. Researchers are also exploring other gene and immune therapies, including a universal gene therapy for Diamond-Blackfan anemia, which aims to correct the disorder regardless of the patient’s specific mutation. Additionally, a drug approved for heart failure has demonstrated potential in killing aggressive ovarian cancer cells and resensitizing relapsed tumors to chemotherapy in preclinical studies. These advancements highlight significant progress in the use of gene and immune-based treatments for various diseases.
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Yes • 50%
No • 50%
Yes • 50%
No • 50%
Gene therapy • 25%
Conventional drug therapy • 25%
Stem cell transplant • 25%
Other • 25%
NTLA-2002 • 25%
Editas Medicine Therapy • 25%
Beam Therapeutics Therapy • 25%
Other • 25%
Safety warning update • 25%
Clinical trial halt • 25%
No action • 25%
Other action • 25%
Successful • 25%
Partially Successful • 25%
Unsuccessful • 25%
No Data Released • 25%
1-5 countries • 25%
6-10 countries • 25%
11-15 countries • 25%
More than 15 countries • 25%
Yes • 50%
No • 50%
Adaptimmune (Afami-cel) • 25%
Checkpoint Therapeutics (Cosibelimab) • 25%
Mirati Therapeutics (Adagrasib-Cetuximab) • 25%
Other • 25%
Mild side effects • 25%
No major side effects • 25%
Severe side effects • 25%
Moderate side effects • 25%