Gamida Cell, an Israel-based provider of stem cell expansion technologies and therapeutic products, has signed an investment and option agreement with Novartis Pharma. Novartis will invest $35 million in Gamida Cell and in return will receive 15% equity and an option to fully acquire Gamida Cell. The option is exercisable for a limited period of time following achievement of certain milestones connected to the development of NiCord, anticipated to be met during 2015.

Novartis will pay the other shareholders in Gamida Cell cash payments of approximately $165 million. In addition, the sellers will be entitled to potential future payments, which can reach a total of $435 million, depending on certain development and regulatory milestones and on sales of Gamida Cell's products.

Gamida Cell currently is engaged in a phase I/II study of NiCord as an investigational therapeutic treatment for hematological malignancies such as leukemia and lymphoma. In this study, NiCord is being used as the sole stem cell source. NiCord is derived from a single cord blood unit and expanded and enriched with stem cells using Gamida Cell’s proprietary NAM technology.

In a standard individual cord blood unit, the limited number of stem cells compromises successful engraftment in an adult patient. To circumvent this obstacle, physicians currently provide patients with two cord blood units to achieve therapeutically meaningful cell numbers. The positive clinical results of a phase I/II clinical study using a double cord protocol, NiCord (the expanded cord blood unit) along with an un-manipulated unit, showed early and durable engraftment with the un-manipulated unit disappearing in most of the patients.

These results were the basis for studying NiCord as a single expanded unit without co-infusion of a second un-manipulated cord. NiCord may provide a single unit of expanded cord blood with clinical results comparable to those seen in a double cord setting, thus introducing a paradigm shift in treatment practice. The current phase I/II single cord study of NiCord is soon to be followed by a phase III study planned to begin at the end of 2015.

Additionally, recruitment continues for Gamida Cell’s phase I/II study of NiCord for pediatric sickle cell disease (SCD). SCD affects 90,000 to 100,000 in the U.S. alone. Symptoms range in type and severity. However, SCD can be fatal. To date, the only known cure for SCD is stem cell transplantation from a family related matched donor.