Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells characterized by rapid growth of abnormal white blood cells that accumulate in the bone marrow. Although AML is rare, there were an estimated 19,520 new U.S. cases of AML in 2018 with an estimated five-year survival rate of 25%. Current treatment of relapsed or refractory AML with chemotherapy or hematopoietic stem cell transplantation is associated with low rates of complete response and considerable complications.

BPDCN is an ultra-rare disease and aggressive blood cancer with 500-1,000 patients per year in the U.S. and a median overall survival of 9-12 months.

CD123 is overexpressed on AML blasts and leukemic stem cell-enriched cell subpopulations compared to normal hematopoietic stem cells and myeloid progenitors, making CD123 an attractive target for T cell-based adoptive immunotherapy.

GBM is the most common brain and central nervous system (CNS) cancer, accounting for 45.2% of malignant primary brain and CNS tumors, 54% of all gliomas, and 16% of all primary brain and CNS tumors. There were an estimated 12,390 new GBM cases predicted in 2017 in the U.S. Malignant brain tumors are the most common cause of cancer-related deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19-year-olds in the U.S. Although GBM is a rare disease (2-3 cases per 100,000 persons per year in the US and EU), it is quite lethal, with five-year survival rates historically under 10%. Standard of care therapy consists of maximal surgical resection, radiation and chemotherapy with temozolomide, which, while rarely curative, is shown to extend median overall survival from 4.5 to 15 months. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies.

MM is a type of blood cancer that develops within bone marrow and affects plasma cells. Normal plasma cells produce antibodies that attack infections and diseases. When plasma cells become cancerous, however, they accumulate in the bone marrow and can cause severe pain and damage to the bone marrow. In addition, cancerous plasma cells will produce faulty antibodies, which make it harder for the body to fight off infections and diseases. Despite great advances in treatment, MM remains an uncured malignancy of plasma cells.

There are several forms of NHL, including follicular lymphoma, mantle cell lymphoma, marginal zone lymphoma, lymphoplasmacytic lymphoma and small lymphocytic lymphoma, which account collectively for ~45% of all cases of NHL. Most types of NHL are incurable with available therapies, except for allogenic hematopoietic stem cell transplant (allo-SCT). More than 70,000 new cases of B-cell NHL are diagnosed each year in the United States, and more than 19,000 patients die annually due to this group of diseases.

GBM is the most common brain and central nervous system (CNS) cancer, accounting for 45.2% of malignant primary brain and CNS tumors, 54% of all gliomas and 16% of all primary brain and CNS tumors. There were an estimated 12,390 new GBM cases predicted in 2017 in the U.S. Malignant brain tumors are the most common cause of cancer-related deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19-year-olds in the U.S. Although GBM is a rare disease (2-3 cases per 100,000 persons per year in the U.S. and E.U.), it is quite lethal, with five-year survival rates historically under 10%. Standard of care therapy consists of maximal surgical resection, radiation and chemotherapy with temozolomide, which, while rarely curative, is shown to extend median overall survival from 4.5 to 15 months. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies.

HER2/neu (HER2) is a growth-promoting protein on the outside of all breast cells. HER2-positive (HER2+) breast cancer cells tend to grow and spread faster than other breast cancers. Breast cancer is the most commonly diagnosed cancer in women, with over 40,000 women in the U.S. expected to die from advanced metastatic disease annually. Approximately 20% to 25% of breast cancers overexpress HER2, which is an established therapeutic target of both monoclonal antibodies (mAbs) and receptor tyrosine kinase inhibitors.

GBM is the most common brain and central nervous system (CNS) cancer, accounting for 45.2% of malignant primary brain and CNS tumors, 54% of all gliomas and 16% of all primary brain and CNS tumors. There were an estimated 12,390 new GBM cases predicted in 2017 in the U.S. Malignant brain tumors are the most common cause of cancer-related deaths in adolescents and young adults aged 15-39 and the most common cancer occurring among 15-19-year-olds in the U.S. Although GBM is a rare disease (2-3 cases per 100,000 persons per year in the US and EU), it is quite lethal, with five-year survival rates historically under 10%. Standard of care therapy consists of maximal surgical resection, radiation and chemotherapy with temozolomide, which, while rarely curative, is shown to extend median overall survival from 4.5 to 15 months. GBM remains difficult to treat due to the inherent resistance of the tumor to conventional therapies.

Prostate cancer has an annual incidence of 164,000, pancreatic cancer has an annual incidence of 55,000, gastric cancer has an annual incidence of 26,000 and bladder cancer has an annual incidence of 81,000.

Central nervous system diseases, also known as central nervous system disorders, are a group of neurological disorders that affect the structure or function of the brain or spinal cord, which collectively form the central nervous system (CNS).

Rheumatic diseases, sometimes referred to as musculoskeletal diseases, affect the joints, ligaments, tendons, bones or muscles. There are more than 200 distinct rheumatic diseases.

Severe combined immunodeficiency (SCID) due to complete recombinase-activating gene-1 (RAG1) deficiency is a rare, genetic severe combined immunodeficiency disorder due to null mutations in the RAG1 gene resulting in less than 1% of wild type V(D)J recombination activity. Patients present with neonatal onset of severe, recurrent fungal, viral and bacterial infections, as well as skin rashes, chronic diarrhea, failure to thrive and fever. Immunologic observations include profound T- and B-cell lymphopenia, low or absent serum immunoglobulins, and normal natural killer cell counts. As is the case with other types of SCID, RAG1-SCID is fatal in infancy unless immune reconstitution is achieved with hematopoietic stem cell transplantation (HSCT). RAG1-SCID Gene Therapy, developed by researchers at LUMC, has been exclusively licensed by Mustang Bio. Successful development of RAG1-SCID Gene Therapy may lead to its replacing HSCT in the treatment of the majority of RAG1-SCID patients.

In Vivo CAR T Technology, developed by Larry R. Pease, Ph.D., principal investigator and former director of the Center for Immunology and Immune Therapies at Mayo Clinic, and exclusively licensed by Mustang Bio, is a new platform to administer CAR T therapy using a two-step approach. In the first step, an adenoviral vector is injected intradermally into the patient to drive the activation of resident T cells in nearby lymph nodes. In the second step, a viral CAR construct is injected directly into these lymph nodes. The viral construct infects the activated T cells and creates CAR T cells in vivo in the patient. These CAR T cells then migrate out of the lymph nodes into the circulation to proliferate and effect the killing of tumor cells. Successful development of the In Vivo CAR T Technology may lead to an off-the-shelf product with no need to isolate and expand allogeneic or autologous (patient-derived) T cells ex vivo.

Menkes disease is an X-linked genetic disorder of copper transport caused by mutations of the ATP7A gene. Copper is an essential trace element required for human development and health. In Menkes disease, patients are born without the ability to absorb copper from diet, leading to severe developmental delays and significant neurological symptoms. Without treatment, Menkes disease could lead to death by three years of age. Currently, there is no FDA-approved therapy for treatment of Menkes disease.

TBIs are associated with 33% of all trauma-related deaths. There are no effective therapies to treat secondary brain injury and the post-injury response of central nervous system apoptosis and neuroinflammation.

TBIs are associated with 33% of all trauma-related deaths. There are no effective therapies to treat secondary brain injury and the post-injury response of central nervous system apoptosis and neuroinflammation.

Genetically Driven Cancers - Initially targeting KRAS G12D,which was previously considered un-druggable and plays a significant role in various cancer types with substantial unmet need, including pancreatic and colorectal.

Coronaviruses - Coronaviruses have single-stranded RNA genomes, making them strong targets for ONCOlogues. The company is studying replacement sequences, which could help combat COVID-19 and provide proof-of-concept as a treatment for coronaviruses.