Drug Pipeline

29 drugs associated with Severe combined immunodeficiency (SCID) / combined immunodeficiency syndromes

DrugPhaseSponsor
ADA PBSC
unknownNational Human Genome Research Institute (NHGRI)↗
Alemtuzumab 0.2 mg

CAMPATH-1 antigen inhibitor

unknownMasonic Cancer Center, University of Minnesota↗
Alemtuzumab 0.3 mg

CAMPATH-1 antigen inhibitor

unknownMasonic 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

unknownMasonic 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.

unknownMasonic Cancer Center, University of Minnesota↗
SCID screening
unknownNantes University Hospital↗
Stem Cell Transplantation

Removal of necrotic tissue

unknownMasonic Cancer Center, University of Minnesota↗
Strimvelis

ADA exogenous gene

unknownFondazione Telethon↗
Transduced Lymphocytes

ADA exogenous gene

unknownNational Human Genome Research Institute (NHGRI)↗