BioCentury | Aug 27, 2020
Distillery Therapeutics

siRNAs against plaque-destabilizing molecule CAMK2G for atherosclerosis

DISEASE CATEGORY: Cardiovascular INDICATION: Atherosclerosis Nanoparticles carrying siRNAs against CAMK2G, a plaque-destabilizing molecule expressed by macrophages, could treat atherosclerosis. The nanoparticles consist of a poly(lactic-co-glycolic acid) (PLGA) core loaded with an anti-CAMK2G siRNA-cationic lipid complex and...
BioCentury | Jun 26, 2020
Distillery Therapeutics

FMR1 peptide therapy for Fragile X syndrome

DISEASE CATEGORY: Neurology INDICATION: Fragile X syndrome An FMR1-based peptide therapy could treat Fragile X syndrome. The therapy comprises a FMR1 N-terminal fragment -- the first 297 residues -- conjugated to a cell-penetrating peptide derived...
BioCentury | May 15, 2019
Translation in Brief

Lentiviral gene therapy could offer relief to refractory epilepsy patients

Within a year, University College London researchers hope to begin a clinical trial of a gene therapy for epilepsy, a non-monogenic disorder. The hope is that the approach will offer an alternative to brain surgery...
BioCentury | May 6, 2019
Distillery Therapeutics

KCNA1 gene therapy for epilepsy

DISEASE CATEGORY: Neurology INDICATION: Epilepsy Rat studies suggest KCNA1-based gene therapy could help treat epilepsy. The gene therapy consisted of KCNA1 codon-optimized for expression in human cells and harboring the I400V mutation to increase the...
BioCentury | Oct 31, 2018
Distillery Therapeutics


INDICATION: Addiction Rat studies suggest M1 muscarinic receptor (CHRM1)-based chemogenetic inhibition of the prelimbic paraventricular thalamic nucleus pathway could help treat cocaine addiction. The system for chemogenetically inhibiting the pathway is initiated by bilateral injection...
BioCentury | Aug 1, 2018
Distillery Techniques

Drug platforms

TECHNOLOGY: Gene therapy A method enabling BBB entry, and temporal and cell type-specific control of chemogenetic therapies could be used to develop treatments for CNS diseases. The method, dubbed acoustically targeted chemogenetics (ATAC), involves three...
BioCentury | Aug 1, 2018
Distillery Therapeutics

Autoimmune disease; renal

INDICATION: Lupus; renal damage Patient sample and mouse studies suggest inhibiting CAMK4 could help treat lupus nephritis, focal segmental glomerulosclerosis (FSGS) and other podocytopathies. In kidney samples from lupus nephritis and FSGS patients, podocyte levels...
BioCentury | Jul 31, 2018
Translation in Brief


Scientists from the California Institute of Technology have combined ultrasound waves with gene and drug therapies to selectively and non-invasively control neural circuits. The approach could be used to treat neurologic and psychiatric diseases and...
BioCentury | Jul 26, 2018
Translation in Brief

A plasticity problem

New research from the University of Bordeaux suggests the synaptic pathology underlying Alzheimer’s disease is driven by aberrant activation of CAMK2 by β-amyloid oligomers. The findings may point to the missing link in how β-amyloid...
BioCentury | Jun 6, 2018
Distillery Techniques


TECHNOLOGY: Gene profiling A mutation in CAMK2A could help predict the risk of a previously undescribed neurodevelopmental disorder characterized by growth delay, frequent seizures and severe cognitive dysfunction. Genomic profiling of two children with the...
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