NK Cell and CAR-NK Cell Program
The company’s Natural Killer (NK) cell program utilizes Nohla’s cell therapy platform technology. Through the use of the company’s proprietary engineered Notch ligand, this platform technology promotes the self-renewal or directed differentiation of cord blood stem and progenitor cells by modulating the Notch signaling pathway. Here, the Notch signaling pathway is leveraged to maximize production of NK cells which can be generated ex vivo, resulting in significant numbers of NK cells that can be cryopreserved and stored in multiple doses as a potential off-the-shelf universal cell therapy. The company has demonstrated the ability of these NK cells to kill cancerous cells both in vitro and in mouse models. The company has also demonstrated the feasibility of genetically modifying these cells to express chimeric antigen receptors (CAR) without negatively impacting the growth process and cell yield. The company is in the process of exploring multiple potential candidates for advancement to preclinical development as oncology therapeutics.
Enhanced HSC Expansion Platform
Nohla is also developing an enhanced cell growth platform for Hematopoietic Stem Cells, or HSCs, based on the two-dimensional methods used to manufacture dilanubicel for clinical development. This enhanced ex vivo expansion platform will focus on the use of a novel three-dimensional technology that could result in a significant improvement in maintenance of primitive HSCs without differentiation during the expansion phase. Once optimized, the company envisions a powerful technology to be used for the expansion of genetically modified cells, which may lead to increased efficacy and reduced costs for gene therapy-based products.
Dilanubicel (NLA101) is a universal donor, off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell product that does not require tissue matching. It can be manufactured ahead of time, cryopreserved, and made available for immediate use.
It has been evaluated in two Phase 2 clinical trials for patients undergoing a cord blood transplant and patients receiving high-dose chemotherapy. Nohla is further analyzing results from an interim analysis in AML patients and is currently seeking companies interested in in-licensing the program.