BioCentury | Jan 1, 2021
Targets & Mechanisms

A snapshot of target innovation in 2020

Preclinical publications highlighted in BioCentury’s Distillery this year identified white space target opportunities including untapped E3 ubiquitin ligases, vesicular regulators and ion channels, as...
BioCentury | Dec 31, 2020
Distillery Therapeutics

T cell-specific targeting of KCNA3 for lupus nephritis

DISEASE CATEGORY: Autoimmune disease INDICATION: Lupus Lipid nanoparticle-based T-cell specific inhibition of the potassium channel KCNA3 could treat lupus nephritis. The nanoparticles contain an siRNA against KCNA3 and are coated with a mAb targeting CD45RO,...
BioCentury | Aug 28, 2020
Distillery Therapeutics

Potassium channel-vesicle interaction as a target in ischemic stroke

DISEASE CATEGORY: Neurology INDICATION: Stroke Interrupting the interaction between the potassium channel KCNB1 and VAPA, a vesicle membrane protein, could treat ischemic stroke. In cultured rat cortical...
BioCentury | May 13, 2020
Emerging Company Profile

QurAlis taking three ALS programs toward clinic with $42M series A

After launching with a modest seed round in 2018, neurology company QurAlis is driving toward the clinic with a $42 million series A round to fund development of three therapies to treat amyotrophic lateral sclerosis...
BioCentury | Dec 3, 2019
Company News

Xenon licenses sodium channel inhibitors to Neurocrine to fund more advanced epilepsy programs

Xenon has exclusively licensed its sodium channel inhibitors for epilepsy to Neurocrine to focus on advancing its potassium channel modulators through the clinic. The deal adds a new indication to Neurocrine's neurology portfolio and gives...
BioCentury | Jun 27, 2019
Product Development

Gene therapy’s next frontier lies beyond rare, monogenic diseases

As in vivo gene therapies continue notching approvals for rare monogenic diseases, the next frontier for the modality will be in treating complex indications without obvious genetic drivers. At least seven companies have clinical or...
BioCentury | Jun 4, 2019
Financial News

June 4 Financial Quick Takes: Lung Therapeutics' $36M series C; plus Strand, Hansoh, Inhibrx and more

Lung Therapeutics closes $36M series C Lung Therapeutics Inc. (Austin, Texas) raised $36 million in a series C round led by Bios Partners with participation by undisclosed new and existing investors. Lung has raised $53...
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 | Jan 23, 2019
Distillery Techniques

Disease models

TECHNOLOGY: 3-D models A 3-D human tissue model that mimics beating adult myocardium could help assess the effects of heart disease therapies on myocardial contractility. Generation and use of the biomimetic model involves five steps:...
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