NLA101 is a universal donor, off-the-shelf, ex vivo expanded hematopoietic stem and progenitor cell product that provides rapid, transient hematopoiesis with long-term benefits. NLA101 was intentionally developed to be a short-term therapy that provides temporary bone marrow function that persists 2-3 weeks until a patient’s immune system recovers while inducing long-term immunologic benefits with the potential for improved survival.
NLA101 is efficiently manufactured ahead of time, cryopreserved, and available for immediate use. NLA101 is differentiated from other autologous or patient-specific allogeneic cell therapies as it does not require tissue matching. NLA101 is currently the subject of two Phase 2 clinical trials to improve treatment outcomes in patients receiving cord blood transplants, and for patients receiving high-dose chemotherapy.
NLA101 for Cord Blood Transplants
Widespread adoption of cord blood transplants is currently limited by the low cell dose provided in a cord blood graft. On average, a cord blood graft contains one tenth the number of stem and progenitor cells compared to other stem cell sources, specifically hematopoietic stem cell grafts from bone marrow or peripheral blood donors. As a result, patients receiving a cord blood transplant are at increased risk for significantly-delayed engraftment or graft failure and experience higher early transplant-related morbidity and mortality compared with patients receiving bone marrow or peripheral blood stem cell transplants.
NLA101 has the potential to address this limitation by instantly providing significantly-increased numbers of stem and progenitor cells capable of rapid production of blood and immune cells. Clinical results to date demonstrate that NLA101 may reduce morbidity and mortality associated with cord blood transplants and improve overall patient survival.
NLA101 following Intensive Chemotherapy
NLA101 is also in clinical development for patients receiving intensive chemotherapy. In these patients, life-threatening infections are very common, leading to lengthy hospitalization and increased reliance on supportive care. Despite the use of growth factors and antibiotics, nearly 40% of patients treated with high-dose chemotherapy still experience infections, which can be life threatening. In addition, patients who experience serious infections are often required to delay the start of additional chemotherapy cycles or have their chemotherapy dose reduced to avoid ongoing complications. These delays or reductions in treatment may lessen chemotherapy’s effectiveness and its ability to induce long-term disease response.
NLA101 provides these patients with additional treatment options by reducing toxicities associated with intensive chemotherapy, allowing for earlier administration of chemotherapy which could lead to improved patient outcomes.