Gamida Cell Ltd.

Core Business and Technology Platform

The fundamental pillar of Gamida Cell???s operation is its proprietary Nicotinamide (NAM) technology platform. This innovative platform leverages the biological properties of nicotinamide, a form of Vitamin B3, to enhance and expand multiple cell types ex vivo (outside the body). This process allows the company to rapidly generate and maintain highly functional and potent therapeutic cells, which are then administered to patients.

The NAM-enabled cell expansion technology has been successfully applied to:

• Hematopoietic Stem Cells (HSCs): By treating allogeneic stem cells???often sourced from umbilical cord blood???with the NAM technology, Gamida Cell is able to significantly increase the number of cells and their quality. This enhancement is designed to accelerate neutrophil and platelet engraftment after transplantation, a critical measure for patient recovery and reducing the risk of infection.
• Natural Killer (NK) Cells: The technology is also used to expand and enhance the function of innate natural killer cells, a critical component of the body???s immune system, for use in cancer immunotherapy. The NAM platform allows for a scalable and Good Manufacturing Practice (GMP)-compliant culture method that yields NK cells with demonstrated ability to kill cancerous cells.

Gamida Cell???s focus is on providing allogeneic therapies, meaning the therapeutic cells come from a donor rather than the patient themselves. This approach is vital for patients whose own cells may be compromised by their disease or prior treatment, or for those who cannot find an ideal sibling or unrelated donor.

Products and Services Offered

Gamida Cell's offerings include an FDA-approved commercial product and a promising clinical-stage pipeline candidate, both built upon the proprietary NAM technology.

1. Omisirge® (omidubicel-onlv)

Omisirge is Gamida Cell???s lead and first commercially available product. It represents a significant advance in allogeneic hematopoietic stem cell transplantation (HSCT).

• Product Description: Omisirge is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy. It is derived from a single unit of umbilical cord blood which is then expanded and enriched using the NAM technology.
• Indication: Omisirge is approved by the U.S. Food and Drug Administration (FDA) for use in adults and pediatric patients aged 12 and older with hematologic malignancies who are planned for an umbilical cord blood transplantation following myeloablative conditioning.
• Therapeutic Benefit: The primary therapeutic benefit of Omisirge is its ability to accelerate the recovery of neutrophils (a type of white blood cell critical for fighting infection) and platelets following a stem cell transplant. For patients who have limited or no access to a fully matched donor, Omisirge provides a valuable treatment option by mitigating the life-threatening risks associated with prolonged neutropenia and delayed engraftment, such as severe infections. The drug essentially provides a new source of high-quality stem cells for patients who have exhausted other options for bone marrow transplant.
• Service Component: The company provides the service of processing the donor cord blood unit using their NAM expansion process and delivering the final, ready-to-infuse Omisirge product to the transplant center, ensuring a pharmaceutical-grade, consistently manufactured cell therapy.

2. GDA-201

GDA-201 is a key product candidate in Gamida Cell???s pipeline, representing the company???s focus on leveraging the NAM platform for immune cell therapies.

• Product Description: GDA-201 is an intrinsic natural killer (NK) cell immunotherapy candidate. The NAM technology is used to expand and enhance NK cells, significantly increasing their numbers and boosting their ability to target and kill cancer cells.
• Targeted Indications: The candidate is being investigated in clinical trials for the treatment of various hematologic malignancies, with a primary focus on non-Hodgkin lymphoma (NHL). There is also preclinical exploration into its potential use for solid tumors.
• Mechanism of Action: GDA-201's mechanism involves using an intrinsic cell source (NK cells) that is expanded ex vivo to create a potent, allogeneic cell therapy that is ready-to-use. This approach aims to provide an off-the-shelf treatment that harnesses the patient???s or a donor's immune system to attack cancer.

Corporate Status

Gamida Cell, founded in the late 1990s, has navigated the complex landscape of biotech development. While the company was formerly publicly traded, recent capital constraints led to a significant restructuring. In 2024, the company successfully completed a restructuring transaction and transitioned to private ownership under the management of Highbridge Capital Management, LLC. This influx of capital and strategic change was explicitly aimed at stabilizing the company and ensuring the continued commercialization and expansion of Omisirge, as well as providing support for ongoing research and development activities for pipeline candidates like GDA-201. The company maintains its commitment to advancing potentially curative cell therapies for cancer patients.