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MODALITY
2025
Rare diseases – congenital thrombotic thrombocytopenic purpura (cTTP)
Monoclonal antibody
2024
HAE
Rare diseases - hemophelia B
Gene therapy
Cancer - various
Small molecule
Neuorlogical - Huntington's disease
2022
Antibody Drug Conjugate
Cell therapy (Engineered B-cell)
2020
Various
Cancer
Lysosomal diseases - Gaucher
Allergic diseases such as asthma
Rare diseases - thrombotic thrombocytopenic purpura (TTP)
Rare diseases - hereditary angioedema (HAE)
2019
Oncology - acute myeloid leukemia (AML)
Oncology - gastrointestinal toxicity
2018
Cancer - gastrointestinal (GI) adverse events (AEs)
Lysosomal diseases - acid sphingomyelinase deficiency (ASMD)
2016
Oncology - B cell malignancies including non-Hodgkin's lymphoma (NHL) and acute lymphocytic leukemia (ALL)
2015
Oncology - many diseases, including ataxia telangiectasia (AT), chronic lymphocytic leukemia (CLL) and colorectal cancer (CRC)
Virology - hepatits C virus (HCV)
2014
2012
2011
2010
2009