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

New biomarkers of cardiovascular risk could relieve a decade-old bottleneck for diabetes drug development, but validating them will require companies to invest in strategic trial designs, and public and private stakeholders to pool thinking. As...
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

FDA qualified a group of kidney injury biomarkers from the Foundation for the National Institutes of Health (FNIH) Biomarkers Consortium (BC) and the Critical Path Institute (C-Path) Predictive Safety Testing Consortium (PSTC), marking the first...
BioCentury | Sep 10, 2018
Distillery Techniques

Disease models

TECHNOLOGY: Transgenics and knockouts PGK1-knockout mice expressing an SP-C variant could be used to study the biology of idiopathic pulmonary fibrosis (IPF) and screen for therapies. Mice with inducible knockout of PGK1 that systemically expressed...
BioCentury | Aug 29, 2018
Preclinical News

Sequential therapies trigger axon regeneration in spinal cord injury

University of California Los Angeles and Swiss Federal Institute of Technology Lausanne (EPFL) researchers showed delivery of three sequential therapies could trigger axon growth after complete spinal cord injury in rodents, suggesting the combination approach...
BioCentury | Jun 13, 2018
Distillery Therapeutics

Ophthalmic disease

INDICATION: Retinitis; ophthalmic In vitro , mouse and cell culture studies identified a mutant RHO-stabilizing compound that could help treat retinal degeneration and autosomal dominant retinitis pigmentosa (adRP). High throughput screening and testing in a ligand-binding...
BioCentury | Jun 8, 2018
Company News

Opthotech gains gene therapy for ophran vision-loss disease

Ophthotech Corp. (NASDAQ:OPHT) acquired exclusive, worldwide rights to develop and commercialize a preclinical ophthalmic gene therapy from the University of Florida Research Foundation and the University of Pennsylvania. The deal gives the biotech an adeno-associated...
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