INSC01 - Cord Blood Stem Cell Expansion Product

“Grounded in half a century of research, the study of hematopoietic stem cells is one of the most exciting and rapidly advancing disciplines in biomedicine today…Key issues for tapping the potential of hematopoietic stem cells will be finding ways to safely and efficiently expand the numbers of transplantable human HSCs in vitro or in vivo.”

NATIONAL INSTITUTE OF HEALTH WEB SITE

Cord Blood	Proprietary 8-day cord                           blood stem cell                        expansion product	Meets growing demand for   hematopoietic stem cell              transplants

This experience has been applied toward the development of INSC01, a proprietary closed system bioreactor that amplifies hematopoietic (i.e. blood) stem cells from cord blood ex vivo.

INSC01 is directed towards the treatment of adult and pediatric patients with hematologic diseases (e.g. leukemia, lymphoma, thalassemia, sickle cell disease) for whom a bone marrow match is not available. INSC01 provides a 14-23 fold expansion of blood progenitor cells from a cord blood sample over a relatively short period of time.

INSC01 addresses significant limitations commonly associated with the use of cord blood stem cells in transplants. The majority of cord blood transplants are currently performed on children, largely due to the limited number of stem cells that can be harvested from a typical cord blood sample. Moreover, the time to engraftment for cord blood stem cells is on average 10 days longer than for bone marrow stem cells. During this period, the patient is at great risk for acquiring opportunistic infections which often contribute to transplant related mortalities.

A growing body of clinical data has established that the number of cord blood stem cells used in a transplant correlates directly with improved engraftment and survival (Ballen, Blood; 105: 3786-3792). There is also a minimum number of cord blood stem cells that is required to achieve favorable outcomes in adult transplant recipients. Most cord blood units do not provide this minimum number of cells. Newer strategies such as double cord blood transplants have shown improvement in clinical outcomes, however, it is generally believed that development of technologies to expand stem cells in cord blood beyond two-fold are needed to enable routine adult cord blood transplants and improved clinical outcomes.

INSC01 is a cellular product that is categorized as “more-than-minimally manipulated” by the FDA, and a Biologics License Application is required for its eventual marketing. Insception has received approval from the CBER (Center for Biologics Evaluation and Research) unit of the FDA for a combined phase I/II trial whereby safety and efficacy will be determined within the time-frame of the trial (2 years).