NLA101 is an ex-vivo expanded stem and progenitor cell graft derived from umbilical cord blood. The product was developed to improve treatment outcomes in patients receiving cord blood transplants, and in patients with hematologic malignancies at risk for chemotherapy induced neutropenia.
While HCT is a curative treatment option, patients require a matched related or unrelated donor genetically matched at multiple human leukocyte antigens (HLA) to avoid fatal outcomes from graft versus host disease. In contrast, patients undergoing a cord blood transplant (a form of HCT) require matching at fewer HLA loci between patient and cord blood donor, providing increased access to HCT for nearly all patients in need. However, widespread adoption of cord blood transplants as a treatment option is limited by the low cell dose provided by a cord blood graft. As a result, patients are at known risk of delayed engraftment or graft failure and higher early transplant related mortality as compared with other hematopoietic stem cell sources. NLA101 addresses this limitation by generating significantly increased numbers of stem and progenitor cells capable of rapid in vivo repopulation that provide robust early bridging engraftment until engraftment and hematopoietic recovery is achieved by the cord blood donor. As such, NLA101 is intended to reduce morbidity and mortality associated with HCT and improve long term survival. .
Another area of focus for the company is chemotherapy induced neutropenia (CIN) in patients with acute myelogenous leukemia (AML). Current AML chemotherapy approaches are aggressive and associated with significant morbidity and mortality due to prolonged severe neutropenia, thrombocytopenia and immunosuppression. As a result, life-threatening infections are very common in this AML population, leading to lengthy hospitalization and increased reliance on supportive care. Despite the use of growth factors and antibiotics, nearly 30% of patients with hematologic malignancies still experience CIN. To address this unmet medical need, Nohla is developing NLA101, a universal donor expanded progenitor cell product which overcomes the limitations of current standard of care for CIN by providing a donor cell source for rapid, transient, myelopoiesis until autologous recovery can occur. This may ultimately result in significantly improved disease control, reduced morbidity, and better overall patient survival.