PORTEFEUILLE

Currently approved stem cell gene therapies require each patient’s cells to be collected, corrected outside the body (ex-vivo), and reinfused after intensive chemotherapy, creating a 6-to-12-month journey with serious side effects, mortality risk, limited scalability, and very high costs. The main reason behind such procedure is that adding an entire gene inside patients’ body (in-vivo) is not feasible. Immitra Bio’s innovation eliminates the need for gene insertion, enabling the development of a one-time, off-the-shelf in-vivo gene-editing therapy that corrects blood stem cells directly inside the patient, without chemotherapy or individualized cell manufacturing. This innovative shift from ex-vivo cell processing to in-vivo correction shortens treatment from months to days, improve safety and access, and make curative gene-editing therapies scalable for patients with severe inherited blood disorders.

At project start, Immitra Bio will focus on optimizing and validating its in-vivo blood stem cell gene-editing approach in relevant preclinical models, with the first interim report expected to cover lead optimization, assay development, and initial safety and efficacy readouts. The project closes a key gap between promising in-vitro proof-of-concept and a clinically translatable in-vivo therapy by generating the data needed to de-risk delivery, editing performance, and functional benefit. The project builds directly on the ETH spin-off Immitra Bio GmbH and is intended to advance its therapeutic platform toward IND-enabling studies. Industrial partnerships are being pursued, and evaluation of the delivery technology and downstream development is planned, including collaborations with specialized LNP delivery providers and future pharma partners. After completion, the program will continue through seed financing, non-dilutive grants, and strategic partnerships to support larger preclinical studies, regulatory preparation, and eventual clinical translation.