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Holzleitner, N.

Publications and source records attributed to Holzleitner, N..

2 recordsLinked to original sources

Synthesis and Preclinical Development of a Novel 68Ga/89Zr-Labelled α{nu}β6-Integrin Targeting Trimer

The v{beta}6 integrin has emerged as a valuable target for theranostic applications in nuclear medicine with high applicability across a variety of cancers, including head-and-neck, lung, breast, and pancreatic carcinomas. [Ga]Ga-Trivehexin is a prominent example of a diagnostic tracer targeting this integrin. In this work, we aimed to expand on this concept by developing FSC(PEG4-v{beta}6), a novel tracer that retains the Trivehexin design, but features PEGylated spacers and replaces the TRAP chelator with Fusarinine C (FSC), enabling labelling with Zirconium-89 in addition to Gallium-68. Preclinical characterization of [Ga]Ga/[Zr]Zr-FSC(PEG4-v{beta}6) included affinity determination towards the v{beta}6 integrin and cellular uptake studies in v{beta}6-positive H2009 cells. A subcutaneously xenografted H2009 tumor model was used to assess the PET imaging potential and biodistribution at early time points with the Gallium-68-labelled compound, and at later time points (up to 6 days post-injection) with the Zirconium-89-labelled version. While [Ga]Ga-FSC(PEG4-v{beta}6) exhibited moderate binding to v{beta}6, its affinity, cellular internalization, and tumor uptake in vivo were lower compared to [Ga]Ga-Trivehexin. Notably, this decreased target engagement was associated with reduced nonspecific binding, which we primarily attributed to the incorporation of PEGylated linkers. Despite indication of in vivo degradation of [Zr]Zr-FSC(PEG4-v{beta}6), still a meaningful evaluation of pharmacokinetics and biodistribution at extended time points was feasible, indicating its suitability for prolonged imaging studies.

cancer biology↗

Engineering de novo binder CAR-T cell therapies with generative AI

Chimeric antigen receptor T cell (CAR-T) therapies have revolutionized cancer treatment, with six CAR-T products currently in clinical use1-4. Despite their success, high resistance rates due to antigen escape remain a major challenge5,6. In silico design of de novo binders (DNBs) has the potential to accelerate the development of new binding domains for CAR-T, possibly enabling personalized therapies for cancer resistance7,8. Here, we show that DNBs can be used for CAR-T, targeting clinically relevant cancer antigens. Using a DNB against the epidermal growth factor receptor (EGFR), we demonstrate comparable cytotoxicity, cytokine secretion, long-term proliferation, and lysis of primary patient-derived cancer organoids with single-chain variable fragment (scFv)-based and DNB-based CAR-T cells. Moreover, we use generative artificial intelligence (AI) guided binder design with RFdiffusion9 to target the B cell maturation antigen (BCMA), a key antigen in multiple myeloma treatment10-17. We confirmed the activity of our AI-designed BCMA CAR-T in short- and long-term effector readouts, including a xenograft mouse model of multiple myeloma. Notably, our AI-guided CAR-T approach also successfully targets a mutated BCMA protein variant resistant to the clinically used bispecific antibody teclistamab. In sum, we demonstrate a proof-of-concept for engineering new, bespoke cellular immunotherapies targeting cancer resistance with the help of generative AI. This approach may further accelerate the development of new CAR-T therapies addressing cancer resistance.

bioengineering↗