Page 35 - SaxoCell Annual Report22/23

Reg. HemRec
Med.

Hemoglobinopathies are one of the most common inherited diseases worldwide. The HemRec
project aims to develop a universal cure strategy for ß-chain hemoglobinopathies. To this end, a
designer recombinase produced in the Buchholz laboratory is being developed to correct genetic
defects in hematopoietic stem cells.

Project lead: Frank Buchholz

Partners: TUD Dresden University of Technology, DKMS Life Science Lab gGmbH

By deactivating a gene that affects the regulation of hemoglobin, the production of fetal
hemoglobin can be stimulated. This genetic correction has the potential to reduce the devastating
effects of diseases such as sickle cell anemia and ß-thalassemia, among others. The cooperation
partners TU Dresden and DKMS Life Science Lab GmbH have the common goal of making this
therapeutic option available worldwide at low cost. The scientific vision is to develop an innovative
genome editing therapy for hemoglobinopathies. Current treatments carry high risks or are not
available to all patients. The targeted recombinase technology promises to provide an effective
and safe treatment for these diseases.

The recombinase technology was pioneered over decades by Frank Buchholz and offers important
advantages over other genome editing methods such as the CRISPR-Cas system. For example,
designer recombinases enable more precise and safer genome editing. Great successes in the
development of designer recombinases led to the foundation of the company Seamless
Therapeutics at the Dresden site in 2022. Start-up funding of 11.8 million euros is intended to
support the further development of the technology platform in order to establish a selection of
therapeutically applicable recombinase candidates and prepare them for clinical use. The
company's know-how could therefore be useful for various projects of the SaxoCell cluster.

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