Multiple Myeloma (MM) is a hematologic neoplastic disease in which uncontrolled proliferation of clonal plasma cells’ leads to end-organ damage.  MM is the second most common hematologic malignancy and accounts for 10% of all blood cancer cases. The median age for diagnosis is 65 years, with fewer than 3% of patients presenting when they are younger than 40 years. MM disproportionally affects black males. 

It is estimated that 34,470 new cases of MM and 12,640 deaths related to the disease will occur in the US in 2022. The prognosis of patients is poor and the 5-year relative survival is 60%. MM and its treatment are frequently characterized by recurrent relapses and shorter remissions.  

Cellectis’ UCARTCS1 is an allogeneic CAR T product that is being evaluated in the MELANI-01 study in adult patients with relapsed or refractory Multiple Myeloma (r/r MM).  This product candidate has the potential to become an off-the-shelf available therapy for adult patients with r/r MM that uniquely targets CS1. 

Acute myeloid leukemia (AML) is a devastating neoplasm characterized by uncontrolled proliferation and accumulation of leukemic blasts in the bone marrow and peripheral blood. These cells disrupt normal hematopoiesis and rapidly cause bone marrow failure and death. AML is the most common type of acute leukemia in adults, and accounts for the largest number of annual deaths from leukemia in the United States. It is estimated that 20,050 new cases of AML and 11,540 deaths related to the disease will occur in the US in 2022. The prognosis of patients is poor and the 5-year relative survival is only 30%.   

The majority of adult AML patients experience disease relapse with current treatments or are not candidates to hematopoietic stem cell therapy. There is an urgent need for new therapeutic modalities. 

Cellectis’ UCART123 is an allogeneic CAR T product that targets CD123 and is being evaluated in the AMELI-01 study in adult patients with relapsed/refractory acute myeloid leukemia (r/r AML).  This product candidate has the potential to become an off-the-shelf available therapy for adult patients with r/r AML. 

Acute lymphoblastic leukemia (ALL) is a cancer of the lymphoid line of blood cells characterized by the development of large numbers of immature lymphocytes.  Extramedullary involvement is frequent (central nervous system [CNS], lymph nodes, spleen, liver, and testis). Approximately 85% of ALLs are of precursor B-cells.  ALL accounts for .3% of all new cancer cases, and .3% of all cancer deaths.It is estimated that 6,660 new cases of ALL and 1,560 deaths related to the disease occurred in the US in 2022. ALL represents 12% of all leukemia cases progresses rapidly and is typically fatal within weeks or months if left untreated. 

The majority of newly diagnosed ALL patients are pediatric, however there are still a significant number of adults ALL patients. The goal of therapy is to lead patients into a long-enough remission so that an allogeneic hematopoietic stem cell transplantation (HSCT, the only curative approach) could be performed, leading to leukemia-free survival rates of 14% to 43%. There is an urgent need to develop new therapies for ALL for patients who are not candidates for HSCT or relapse after.

Cellectis’UCART22 is an allogeneic CAR T product that is being evaluated in the BALLI-01 study in adult patients with relapsed or refractory Acute lymphoblastic leukemia (r/r ALL). This product candidate has the potential to become an off-the-shelf available therapy for adult patients with r/r ALL that uniquely targets CD22. 

Non-Hodgkin Lymphoma (NHL) are a heterogeneous group of neoplasms that originate in any of the lymphoid cells: B-cells, T-cells, or NK-cells. NHLs encompass a spectrum of lymphoid malignancies, with 85% arising from B lymphocytes. (B-cell NHL). Non-Hodgkin lymphoma (NHL) is one of the most common cancers in the United States, accounting for about 4% of all cancers and one of the more common cancers among children teens, and  young adults. It is estimated that 80,470 new cases of NHL and 20,250 deaths related to the disease will occur in the US in 2022. The 5-year relative survival rate for people with NHL is 73%. 

Despite novel treatment approaches, challenges remain for patients with relapsed or refractory non-Hodgkin Lymphoma (r/r NHL). A significant proportion of patients relapse to CD19 CAR-T. There is an urgent need for new therapeutic modalities, addressing this patient population that represents a potential therapeutic alternative to CD19-directed therapies

UCART20x22 is Cellectis’ first dual allogeneic CAR T product candidate to enter clinical development in the NatHali-01 study in patients with r/r NHL. Dual targeting of CD20 and CD22, both validated targets in B-cell malignancies, has the potential to enhance tumor cell killing and increases the breadth of antigen targeting. These advantages may represent a potential therapeutic alternative to CD19-directed therapies.