BioCentury | Jan 30, 2021
Distillery Therapeutics

Inhibition of fungal splicing factor PRPF8 for cryptococcal infection

DISEASE CATEGORY: Infectious disease INDICATION: Fungal infection Small molecule inhibitors of a Cryptococcus spp. intein-containing intron splicing factor PRPF8 could treat infections by Cryptococcus neoformans. A screen of 300 small molecules yielded a carboxamide-based compound that inhibited PRPF8...
BioCentury | May 16, 2020
Targets & Mechanisms

Gut microbiome manipulation reduces ALS, frontotemporal dementia symptoms

A team from Harvard and the Broad Institute uncovered a link between intestinal bacteria and a pair of neurological diseases associated with pathological DNA repeats, strengthening the connection between the gut microbiome and neurological diseases....
BioCentury | Jan 31, 2020
Distillery Therapeutics

Supplementation with for alcoholic fatty liver disease

DISEASE CATEGORY: Hepatic INDICATION: Liver disease Oral administration of the gut microbe Roseburia intestinalis could treat alcoholic fatty liver disease. In 22 patients with alcoholic liver disease, lower abundances of Roseburia spp. in fecal samples...
BioCentury | Aug 15, 2019
Distillery Therapeutics

TMED9 inhibition for mucin 1 kidney disease

DISEASE CATEGORY: Renal; ophthalmic INDICATION: Renal; retinitis Blocking the vesicular cargo receptor TMED9 could treat mucin 1 kidney disease, a proteinopathy caused by a frameshift mutation in MUC1, and other misfolded protein diseases. The Harvard...
BioCentury | Apr 2, 2019
Distillery Therapeutics

OPN1MW gene therapy for retinitis pigmentosa

DISEASE CATEGORY: Ophthalmic disease INDICATION: Retinitis Mouse studies suggest OPN1MW gene therapy could help treat retinitis pigmentosa. In a mouse model of retinitis pigmentosa, intravitreal injection of the mouse homolog of OPN1MW encoded in an...
BioCentury | Feb 15, 2019
Tools & Techniques

Heartening predictors in diabetes

...The four proteins are NT-proBNP; KIM-1, a protein upregulated by kidney cells after renal injury; Opn...
...KIM-1 - Kidney injury molecule 1 NT-proBNP - N-terminal pro-brain natriuretic peptide Opn (Spp1) - Osteopontin...
BioCentury | Nov 13, 2018
Distillery Therapeutics

Ophthalmic disease

INDICATION: Blindness Mouse studies suggest inhibiting ATXN7 could help treat vision loss in spinocerebellar ataxia type 7 (SCA7), which is caused by gain-of-function mutations in the gene. In a genetic mouse model of SCA7, intravitreal...
BioCentury | Nov 2, 2018
Company News

Ionis grants ProQR rights to QR-1123

Ionis Pharmaceuticals Inc. (NASDAQ:IONS) granted ProQR Therapeutics N.V. (NASDAQ:PRQR) exclusive, worldwide rights to QR-1123 (formerly IONS-RHO-2.5Rx) along with relevant IP. Ionis received $2.5 million in ProQR shares upfront. The partners said the shares were priced...
BioCentury | Oct 26, 2018
Politics & Policy

FDA grants first qualification of a clinical safety biomarker

...healthy volunteers. The markers are kidney injury molecule 1 (KIM-1), clusterin (CLU; APOJ), osteopontin (Opn; Spp1...
BioCentury | Sep 10, 2018
Distillery Techniques

Disease models

...of fibrosis-promoting monocytes in lung, acute alveolitis, poor survival and elevated expression of osteopontin (Opn; Spp1...
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