BioCentury | Jan 31, 2020
Distillery Therapeutics

Targeting SMPD1 to treat norovirus

DISEASE CATEGORY: Infectious disease INDICATION: Norovirus Inhibiting the sphingomyelinase SMPD1, by preventing its activation or blocking its release from lysosomes, could treat norovirus infection. Norovirus infect cells via uptake by endosomes, which undergo acidification --...
BioCentury | Sep 12, 2019
Distillery Therapeutics

SMPD1 gene therapy for Niemann-Pick disease type A

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Metabolic SMPD1, a sphingomyelinase, encoded in viral vector could treat Niemann-Pick disease type A. The lysosomal storage disorder is caused by loss-of-function mutations in SMPD1 and is characterized by motor impairments,...
BioCentury | Aug 23, 2019
Translation in Brief

New Therapeutic Targets and Biomarkers: July 2019

New Therapeutic Targets and Biomarkers: July 2019 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database in July. Therapeutic targets are defined as any protein, gene or other molecule...
BioCentury | Jul 11, 2019
Distillery Therapeutics

SMPDL3b inhibition or C1P supplementation for diabetic kidney disease

DISEASE CATEGORY: Renal INDICATION: Diabetic nephropathy Inhibition of SMPDL3b or supplementation with the bioactive sphingolipid ceramide-1-phosphate (C1P) could help prevent diabetic kidney disease. In human podocytes, which are injured in diabetic kidney disease, SMPDL3b knockdown...
BioCentury | Apr 10, 2019
Distillery Therapeutics

Inhibiting production of PD-L1-loaded exosomes for prostate, colorectal cancers

...DISEASE CATEGORY: Cancer INDICATION: Prostate cancer; colorectal cancer Mouse studies suggest inhibiting RAB27A or SMPD3 alone...
...and colorectal cancers. In a mouse model of prostate cancer, tumor-specific knockout of RAB27A or SMPD3...
...to treat prostate or colorectal cancer. TARGET/MARKER/PATHWAY: RAB27A member RAS oncogene family (RAB27A); sphingomyelin phosphodiesterase 3 (SMPD3; NSMASE2...
BioCentury | Apr 8, 2019
Preclinical News

Tumors release PD-L1 exosomes to evade antitumor immunity

...block exosome release by deleting two genes associated with exosome production in cancer mouse models, SMPD3...
...cell death 1 ligand 1; RAB27A - RAB27A member RAS oncogene family; SMPD3 (NSMASE2) - Sphingomyelin phosphodiesterase 3 Sandi...
...California San Francisco Programmed cell death 1 ligand 1 (PD-L1) (B7-H1) (CD274) RAB27A member RAS oncogene family (RAB27A) Sphingomyelin phosphodiesterase 3 (SMPD3) (NSMASE2) Colorectal...
BioCentury | Oct 29, 2015
Translation in Brief

Microglia strike again

...study, the team used a commercially available tool compound that inhibits sphingomyelin phosphodiesterase 3 ( SMPD3 ; NSMASE2...
...of its work, but Ikezu thinks a company could synthesize a patentable analog of the SMPD3...
BioCentury | Oct 29, 2015
Distillery Therapeutics

Therapeutics: Sphingomyelin phosphodiesterase 3 (SMPD3; NSMASE2); microtubule-associated protein τ (tau; MAPT; FTDP-17); colony-stimulating factor 1 receptor (C

...INDICATION: Alzheimer's disease (AD) Mouse studies suggest depleting microglial cells in the brain or inhibiting SMPD3...
...normal SMPD3 expression or vehicle, respectively. Next steps include testing whether PLX3397 or inhibition of SMPD3...
...tumors and in Phase II testing for brain, breast and other cancers. TARGET/MARKER/PATHWAY: Sphingomyelin phosphodiesterase 3 (SMPD3; NSMASE2...
BioCentury | Jul 18, 2013
Distillery Therapeutics

Indication: Neurology

This week in therapeutics Indication Target/marker/pathway Summary Licensing status Publication and contact information Neurology Depression Sphingomyelin phosphodiesterase 1 acid lysosomal (SMPD1; ASM) Mouse and cell culture studies suggest inhibiting SMPD1 could be useful for treating...
BioCentury | Jul 18, 2013
Targets & Mechanisms

Depressing sphingolipids

European researchers have obtained the most compelling evidence yet that targeting sphingolipid metabolism could help treat depression. 1 The team has shown that two known antidepressants inhibit sphingolipid metabolism and now is planning to screen...
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