Drug Pipeline
29 drugs associated with Severe combined immunodeficiency (SCID) / combined immunodeficiency syndromes
| Drug | Phase | Sponsor | |
|---|---|---|---|
ADA PBSC | unknown | National Human Genome Research Institute (NHGRI) | ↗ |
Alemtuzumab 0.2 mg CAMPATH-1 antigen inhibitor | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
Alemtuzumab 0.3 mg CAMPATH-1 antigen inhibitor | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
Cyclophosphamide Alkylating agents work by three different mechanisms: 1) attachment of alkyl groups to DNA bases, resulting in the DNA being fragmented by repair enzymes in their attempts to replace the alkylated bases, preventing DNA synthesis and RNA transcription from the affected DNA, 2) DNA damage via the formation of cross-links (bonds between atoms in the DNA) which prevents DNA from being separated for synthesis or transcription, and 3) the induction of mispairing of the nucleotides leading to mutations | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
MESNA Mesna reacts chemically with the urotoxic ifosfamide metabolites, acrolein and 4-hydroxy-ifosfamide, resulting in their detoxification. The first step in the detoxification process is the binding of mesna to 4-hydroxy-ifosfamide forming a non-urotoxic 4-sulfoethylthioifosfamide. Mesna also binds to the double bonds of acrolein and to other urotoxic metabolites and inhibits their effects on the bladder. | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
SCID screening | unknown | Nantes University Hospital | ↗ |
Stem Cell Transplantation Removal of necrotic tissue | unknown | Masonic Cancer Center, University of Minnesota | ↗ |
Strimvelis ADA exogenous gene | unknown | Fondazione Telethon | ↗ |
Transduced Lymphocytes ADA exogenous gene | unknown | National Human Genome Research Institute (NHGRI) | ↗ |