Page 36 - SaxoCell Annual Report22/23

The experimental work within the project has made
significant progress and achieved first promising
results. An important milestone was the identification
of the appropriate target sequence to successfully
deactivate the gene BCL11A, which affects
hemoglobin regulation, in human cells. With the help
of SLiDE, first versions of recombinases with activity
at these target sites were developed. Furthermore,
reporter cells could be generated and first knock-out
experiments were performed, which yielded
promising results. In addition, assays were
established in a relevant cell line for the activation of
fetal hemoglobin (see figure).
Activation of fetal hemoglobin by recombinase
technology

In addition, initial data from a well-founded business case analysis are available, shedding light on
the promising conditions for a possible market entry. The results of this study support the
research efforts in this field and confirm the potential of this innovative approach.

Overall, the progress made so far in the project is extremely promising and gives hope that the
project will be a success. The results and data obtained encourage the team to continue research
and development with the ultimate goal of developing an effective and innovative therapeutic
option for hemoglobinopathies.

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