BioCentury | Jan 30, 2021
Product Development

ALS looks up as clinical pipeline explodes

...trial of antisense therapy BIIB078, which targets C9orf72...
...disease processes.Research on the roles of mutant C9orf72...
BioCentury | Jan 23, 2021
Emerging Company Profile

Technology showcase: BioCentury’s 2020 class of emerging companies

As emerging therapeutic modalities move toward mainstream, a flood of new companies has arisen to break through the limitations of first-generation technologies and expand the reach of gene and cell therapies,...
BioCentury | Sep 29, 2020
Distillery Therapeutics

Repeat expansion-targeting antisense oligo for ALS and frontotemporal dementia

...researchers showed that an antisense oligonucleotide targeting C9orf72...
...genetic alteration. In a mouse model expressing C9orf72...
...development for ALS and frontotemporal dementia.TARGET/MARKER/PATHWAY: Chromosome 9 open reading frame 72 (C9orf72)EXPERIMENTAL...
BioCentury | Sep 24, 2020
Emerging Company Profile

Libra launches with $29M, three neurodegeneration programs to lower toxic protein levels

...caused by a six-nucleotide repeat expansion in C9orf72.Veinbergs...
...protein production mechanisms it’s pursuing are independent of C9orf72 and...
...at reducing production of repeat expansion-induced aberrant C9orf72...
BioCentury | May 16, 2020
Targets & Mechanisms

Gut microbiome manipulation reduces ALS, frontotemporal dementia symptoms

...presentation in a mouse model of ALS and FTD associated with six-nucleotide repeat expansions in C9orf72...
...three ALS therapies (see “QurAlis Taking Three ALS Programs Toward Clinic” ). Targets C9orf72 - Chromosome 9 open reading frame 72...
...Chromosome 9 open reading frame 72 SOD1 - Superoxide dismutase 1 Sandi Wong, Assistant Editor Harvard University Broad Institute of MIT and Harvard Chromosome 9 open reading frame 72 (C9orf72) gut...
BioCentury | May 13, 2020
Emerging Company Profile

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

...in the TBK1 pathway can be modulated to normalize malfunction in patients with TBK1 or C9orf72...
...Ventures, MP Healthcare Venture Management, Sanford Biosciences CEO: Kasper Roet Patents: Undisclosed Targets C9orf72 - Chromosome 9 open reading frame 72...
BioCentury | Feb 4, 2020
Finance

Co-founded by UPenn’s Wilson, Passage Bio outlines clinical goals in new IPO filing

...genetically-driven forms of disease: PBML04 for metachromatic leukodystrophy in patients with ARSA mutations; PBAL05 for C9orf72-driven...
...Inc. (NASDAQ:BDTX) (see “Arcutis, Black Diamond Gain” ). Targets ARSA - Arylsulfatase A C9orf72 - Chromosome 9 open reading frame 72...
BioCentury | Jan 4, 2020
Product Development

More Waves to come on stereopure antisense oligo tech

...year, likely the C9orf72 program to treat amyotrophic lateral sclerosis and frontotemporal dementia. Mutations in C9orf72...
BioCentury | Dec 13, 2019
Preclinical News

Dec. 12 Preclinical Quick Takes: POC for Aptose blood cancer candidate, plus Penn-Acorn AI, Neurimmune-Biogen and Syndax

...the generation of mAbs against poly-GA repeat-associated non-ATG (RAN) proteins--the result of repeat expansions in C9orf72--to...
...the survival rate in a mouse model of ALS and frontotemporal dementia (FTD) associated with C9orf72...
...leukemias, with initial data due next year. Targets: Btk - Bruton’s tyrosine kinase; C9orf72 - Chromosome 9 open reading frame 72...
BioCentury | Oct 3, 2019
Targets & Mechanisms

ALS platform trial to put neuroinflammation hypothesis to the test

...Neurodegeneratio n”). A 2016 study found signatures of activated microglia and macrophages in mice lacking C9orf72...
...an immune attack. Targets BDNF - Brain-derived neurotrophic factor C5 - Complement 5 C9orf72 - Chromosome 9 open reading frame 72...
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