BioCentury | Apr 1, 2020
Distillery Therapeutics

FLNA identified as a target for epilepsy associated with focal cortical dysplasia type II

DISEASE CATEGORY: Neurology INDICATION: Epilepsy Inhibiting the actin-binding protein FLNA could prevent or treat epilepsy associated with focal cortical dysplasia type II, a cerebral developmental malformation. Levels of FLNA protein in cortical tissue from 12...
BioCentury | Apr 30, 2019
Distillery Therapeutics

RUNX1, RUNX3, CBFB and PMP22 as targets for neurofibromatosis

DISEASE CATEGORY: Cancer INDICATION: Neurofibromatosis Cell culture and mouse studies suggest promoting PMP22 expression, dual RUNX1/RUNX3 inhibition, or inhibiting the interactions of CBFB with RUNX1 and RUNX3 could help treat neurofibromatosis. In mouse neurofibroma Schwann...
BioCentury | Mar 22, 2019
Targets & Mechanisms

Deconvoluting translation at Scripps

With three institutes now under one roof, Scripps Research has established both its own pipeline and a non-profit funding model to bring candidates through early stage clinical trials. Diverging from the philanthropy and investor-based approaches...
BioCentury | Mar 12, 2015
Distillery Therapeutics

Therapeutics: Core-binding factor β subunit (CBFB; CBF-β); myosin heavy chain 11 smooth muscle (MYH11; SMMHC); runt-related transcription factor 1 (RUNX1)

Cancer INDICATION: Acute myelogenous leukemia (AML) In vitro, mouse and patient sample studies suggest a small molecule protein-protein inhibitor could help treat the inv(16) subtype of AML. The subtype is driven by an oncogenic CBFB-SMMHC...
BioCentury | Sep 13, 2012
Distillery Therapeutics

Indication: Cancer

Indication Target/marker/pathway Summary Licensing status Publication and contact information Cancer Acute myelogenous leukemia (AML); acute lymphoblastic leukemia (ALL) Core-binding factor b-subunit (CBFB; CBFb); runt-related transcription factor 1 (RUNX1) In vitro and mouse studies suggest inhibiting...
BioCentury | May 3, 2012
Distillery Therapeutics

Indication: Autoimmune disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Autoimmune disease Osteoarthritis Filamin A (FLNA); core-binding factor b-subunit (CBFB; CBFb) In vitro and mouse studies suggest the small molecule kartogenin could help treat osteoarthritis. Image-based...
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