Gamida Cell Ltd. is internationally well known for its remarkable innovation in the field of cell therapy. It has developed an unrivaled number of platform technologies to expand functional cell populations. The company has applied these platform technologies towards developing an impressive pipeline of cell therapy products for a wide range of clinical applications. Gamida Cell is currently completing enrollment of patients to the Phase III study of its flagship product, StemEx®. Clinical outcome will be available in Q4/2012. The market launch of StemEx is expected in 2014.
The company is developing a pipeline of cell therapeutics in the areas of cancer, hematological diseases, autoimmune diseases, inflammatory conditions and regenerative medicine. Gamida Cell’s therapeutic products are allogeneic and contain adult stem cells selected from non-controversial sources, such as umbilical cord blood, bone marrow and peripheral blood. These cells are then enriched in culture using the company’s proprietary expansion technologies. Gamida Cell was successful in translating these technologies under GMP into robust manufacturing processes, which are cost effective and efficient for commercial production.
Gamida Cell’s technologies and products are protected by more than 20 patents worldwide and numerous patent applications.
StemEx is a graft of stem/progenitor cells isolated and expanded from a portion of a single unit of umbilical cord blood and transplanted in combination with non-expanded cells from the same unit. The portion of umbilical stem cells is expanded using Gamida Cell’s proprietary copper chelator technology.
StemEx is now being tested in an advanced, Phase III, international, pivotal registration clinical trial for hematological malignancies. This is an open study with a historical control cohort.
StemEx is poised to answer a dire unmet clinical need in the field of bone marrow transplantation by supplying alternative cell grafts to patients with hematological malignancies, such as leukemia and lymphoma, who are indicated for transplantation but do not have a matched family related bone marrow donor. Submission to the international regulatory bodies is planned for 2013 and market launch in the US is planned for 2014. The market potential of StemEx is estimated at more than one billion USD. StemEx has orphan drug designation both in the US and in Europe, with 7-10 years market exclusivity, in addition to long term patent protection.
Though several other companies are developing competing technologies to expand cells from cord blood for transplantation in patients with hematological malignancies, StemEx is expected to be the first to market, and years before the market launch of any of the aforementioned competing technologies. In addition, StemEx is the only product that uses a single cord blood unit, rather than the two units required to create the competing products. Since the average cost of a cord blood unit in the US is $40K, StemEx is expected to be a significantly less expensive treatment option.
StemEx is being developed by the Gamida Cell – Teva Joint Venture established in 2005; Gamida Cell owns 50% of this asset. The Joint Venture owns all global rights for the commercialization of StemEx. In July 2008, Gamida Cell signed a licensing agreement with Amgen for the use of a number of proprietary cytokines in the manufacturing of StemEx.
Aside from StemEx, all Gamida Cell pipeline products and the associated strategic rights are owned 100% by Gamida Cell.
NAM Platform and Pipeline Products
Gamida Cell developed a next generation platform technology to expand functional cells in culture, while attenuating the continuous morphological and functional alterations of cells during ex-vivo cultures. The results of multiple studies using several animal models demonstrated that the expanded cell grafts created using NAM technology displayed improved functionality following in vivo infusion, e.g. migration, homing, and durable retention in the target tissues. Based on this platform technology Gamida Cell is now developing a pipeline of allogeneic products for cancer, hematological diseases, autoimmune diseases, inflammatory conditions and regenerative medicine.
NiCord® for hematological diseases, autoimmune diseases and metabolic diseases
Nicord is a graft of stem/progenitor cells isolated and expanded from a unit of umbilical cord blood. It is intended to treat patients with hematological diseases such as thalassemia sickle cell anemia and hematological malignancies, severe autoimmune diseases and metabolic diseases. NiCord is now being tested in a Phase I/II clinical study in the U.S., under IND.
CordBridgeTM for Acute Radiation Syndrome
GC-003 is an off-the-shelf product for IV administration, designed to provide an immediate and efficacious treatment for Acute Radiation Syndrome (ARS) patients. The readily-available bridging cell graft is highly enriched with the cells required to protect against opportunistic infections and uncontrolled bleeding. It is intended to provide protection during the self-recovery time when the patient's own blood cells (autologous recovery) are reforming. GC-003 will mainly serve as a salvage treatment to reduce morbidity and mortality of nuclear terrorism and accident victims both in civilian and military scenarios.
NK Cells for immune therapy of cancer
NK Cells are cytotoxic lymphocytes that have drawn considerable attention in recent years as a promising tool for the treatment of cancer. Multiple clinical trials are evaluating the safety and efficacy of donor NK cell infusion in patients with various refractory hematological malignancies and metastatic solid tumors.
A major challenge in advancing the clinical applicability of adoptively transferred NK cells is to expand in culture NK cells that will display enhanced trafficking, survival and killing activity following in vivo infusion. Using NAM platform technology, Gamida Cell has developed a novel, simple and robust epigenetic process for prompt expansion of more functional NK cells. Our animal studies demonstrated that NK cells expanded with NAM display enhanced trafficking and durable in vivo survival. NAM also down regulated expression of CD200R and PD-1, surface receptors directly involved in tumor immunoevasion. Accordingly, the NK cells cultured with NAM displayed an increased killing activity of primary tumor cells.
Owing to the increased in vivo survival and tumor cytotoxic effect of the NK cells cultured with NAM, this method is expected to increase the clinical efficacy of adoptively transferred NK cells. Based on these intriguing findings Gamida Cell is developing a cell product for adoptive cell-mediated immune therapy to treat metastatic solid tumors and refractory/relapsed hematological malignancies.
Gamida Cell’s management team has many years of experience in the pharmaceutical and biotechnology industry. The company’s innovative and dedicated management team is led by Dr. Yael Margolin, president and CEO of the company. Shareholders include: Elbit Imaging, Clal Biotechnology Industries, Israel Healthcare Venture, Teva Pharmaceuticals, Amgen, Denali Ventures and Auriga Ventures.