At Charité hospital in Berlin, a 19-year-old patient with beta-thalassemia has been treated for the first time in Germany with the CRISPR-based drug Exa-cel. The gene therapy was administered in the presence of Nobel laureate Emmanuelle Charpentier, a co-discoverer of CRISPR technology.
Four months after the treatment, the patient no longer needs blood transfusions and is leading a normal life. This marks a significant change in the treatment outcome, as the patient had previously been dependent on transfusions.
The Exa-cel used in the treatment is a CRISPR-based gene therapy. Its use at Charité marks the first such case in Germany.
The case is also notable for its status. It is the world’s first approved CRISPR-based therapy to be used as part of standard medical care.
The case shows that CRISPR technology is no longer solely a tool for research settings, but can also be used as part of approved treatment. What is particularly notable here is not only the use of the technology, but also the fact that four months after treatment, the patient no longer needs blood transfusions.
At the same time, the outcome of a single patient cannot by itself represent all possible results of such treatment. In this case, the four-month course is known, and that period should be regarded as the limit of the documented outcome, without drawing longer-term conclusions beyond it.
