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Schanz, J.

Publications and source records attributed to Schanz, J..

2 recordsLinked to original sources

Welding PROxAb Shuttles: A modular approach for generating bispecific antibodies via site-specific protein-protein conjugation

Targeted protein degradation is an innovative therapeutic strategy to selectively eliminate disease-causing proteins. Exemplified by proteolysis-targeting chimeras (PROTACs), it has shown promise in overcoming drug resistance and targeting previously undruggable proteins. However, PROTACs face challenges such as low oral bioavailability and limited selectivity. The recently published PROxAb Shuttle technology offers a solution enabling targeted delivery of PROTACs using antibodies fused with PROTAC-binding domains derived from camelid single-domain antibodies (VHHs). Here, a modular approach to quickly generate PROxAb Shuttles by enzymatically coupling PROTAC-binding VHHs to off- the-shelf antibodies was developed. The resulting conjugates retained their target binding and internalization properties and incubation with BRD4-targeting PROTACs resulted in formation of defined PROxAb-PROTAC complexes. These complexes selectively induced degradation of the BRD4 protein, resulting in cytotoxicity specifically in cells expressing the antibodys target. The chemoenzymatic approach described here provides a versatile and efficient solution for generating antibody-VHH conjugates for targeted protein degradation applications but could also be used to combine antibody and VHH binders to generate bispecific antibodies for further applications.

biochemistry↗

Implications of TP53 Allelic State for Genome Stability, Clinical Presentation and Outcomes in Myelodysplastic Syndromes

TP53 mutations are associated with poor clinical outcomes and treatment resistance in myelodysplastic syndromes. However, the biological and clinical relevance of the underlying mono- or bi-allelic state of the mutations is unclear. We analyzed 3,324 MDS patients for TP53 mutations and allelic imbalances of the TP53 locus and found that 1 in 3 TP53-mutated patients had mono-allelic targeting of the gene whereas 2 in 3 had multiple hits consistent with bi-allelic targeting. The established associations for TP53 with complex karyotype, high-risk presentation, poor survival and rapid leukemic transformation were specific to patients with multi-hit state only. TP53 multi-hit state predicted risk of death and leukemic transformation independently of the Revised International Prognostic Scoring System, while mono-allelic patients did not differ from TP53 wild-type patients. The separation by allelic state was retained in therapy-related MDS. Findings were validated in a cohort of 1,120 patients. Ascertainment of TP53 allelic state is critical for diagnosis, risk estimation and prognostication precision in MDS, and future correlative studies of treatment response should consider TP53 allelic state.

genomics↗