Human pluripotent stem-cells (HPSCs)—including human embryonic stem-cells and human induced pluripotent stem-cells—are reshaping the landscape of regenerative medicine.1,2 In Parkinson's disease, ...
Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...
Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development ...
If approved, Ojemda® (tovorafenib) is expected to be the first and only targeted medicine in European Union for children with ...
Federal Circuit revives REGENXBIO’s suit against Sarepta, reversing a Delaware ruling that found asserted host-cell claims ...
For too long, the promise of personalized therapies has been tantalizingly close, yet frustratingly out of reach for ...
In the wake of the germline editing controversy, when Chinese scientist He Jianku illegally edited the embryos of human twins ...
Gothenburg, February 27, 2026 - Elicera Therapeutics AB (publ), a clinical stage cell and gene therapy company developing next-generation cancer treatments based on its proprietary iTANK platform, tod ...
New FDA guidelines for personalized genomic drug development are a step forward in advancing new therapies for ultrarare diseases, but industry leaders need further clarification.
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