BioCentury | Sep 19, 2020
Translation in Brief

Gene therapies from Passage Bio, Locanabio; Lava’s antitumor bispecific, health disparities in Alzheimer’s and more

...and guide RNA targeting CUG repeats eliminated toxic RNA with microsatellite repeat expansions in the DMPK...
...Bio Inc. University of Pennsylvania Locana Inc. University of California San Diego University of Florida Lava Therapeutics B.V. NIH Granulin (GRN) Progranulin (PGRN) (PCDGF) Dystrophia myotonica-protein kinase (DMPK) CD1D Neurology Musculoskeletal Cancer Frontotemporal...
BioCentury | Jun 26, 2020
Distillery Therapeutics

CDK12 identified as myotonic dystrophy target

...elongation regulator CDK12 could treat DM1, which is caused by CTG repeat expansions in the DMPK...
...healthy volunteers. In a human DM1 fibroblast cell line with one wild-type and one mutant DMPK...
...inhibitor GSK3186899 in Phase I testing for visceral leishmaniasis. TARGET/MARKER/PATHWAY: Cyclin dependent kinase 12 (CDK12); dystrophia myotonica-protein kinase (DMPK)...
BioCentury | Nov 18, 2019

Microtubule inhibitors including colchicine for myotonic dystrophy

...microtubule inhibitors could treat DM1, which is caused by expansions of CTG repeats in the DMPK...
...in animal models. Colchicine, a generic microtubule polymerization inhibitor, is marketed for gout. TARGET/MARKER/PATHWAY: Tubulin; dystrophia myotonica-protein kinase (DMPK)...
...of Medicine, Gainesville, Fla.; State University of New York, Albany, N.Y. email: aberglund@albany.edu Claire Quang University of Florida Tubulin Dystrophia myotonica-protein kinase (DMPK)...
BioCentury | Aug 1, 2019
Product Development

NeuBase takes antisense oligos to more targets in more places

...applying the concept to hairpins that form in mutant versions of mRNAs encoding HTT and DMPK...
...NeuBase’s first two preclinical programs target unique hairpins in mutant HTT mRNA and in mutant DMPK...
...Co. Ltd. (Tokyo:4502), Tokyo, Japan Wave Life Sciences Ltd. (NASDAQ:WVE), Cambridge, Mass. Targets DMPK - Dystrophia myotonica-protein kinase...
BioCentury | May 3, 2019
Product Development

With a deal and a debut, targeted oligo delivery gets a boost from ADCs

...in the DMPK gene. Both compounds are designed to knock down the mutant version of DMPK...
...SIX:ROG; OTCQX:RHHBY), Basel Switzerland Takeda Pharmaceutical Co. Ltd. (Tokyo:4502; NYSE:TAK), Tokyo, Japan Targets DMPK - Dystrophia myotonica-protein kinase Allison...
...Dystrophia myotonica-protein kinase Allison Johnson, Staff Writer Alnylam Pharmaceuticals Inc. Avidity Biosciences LLC Dyne Therapeutics Inc. Ionis Pharmaceuticals Inc. Roche Takeda Pharmaceutical Co. Ltd. Dystrophia myotonica-protein kinase (DMPK)...
BioCentury | Apr 23, 2019
Company News

Fulcrum gets rights to GSK's shelved p38 kinase inhibitor

...Friedreich ataxia and DMPK in myotonic dystrophy (see "Fulcrum’s Genetic Levers" ). Targets: DMPK - Dystrophia myotonica-protein kinase...
BioCentury | Jan 3, 2018
Product R&D

Expanding small molecule horizons

...the most common adult-onset muscular dystrophy. It is caused by repeated CTG sequences in the DMPK...
...the myotonia that occurs in DM1 pathology. CLCN1 - chloride voltage-gated channel 1; DMPK - dystrophia myotonica-protein kinase...
...CRISPR-associated protein 9 CNBP - CCHC-Type Zinc Finger Nucleic Acid Binding Protein DMPK - Dystrophia myotonica-protein kinase...
BioCentury | Sep 14, 2017
Emerging Company Profile

RNA gets CRISPR

...study focused on myotonic dystrophy type 1 (DM1), in which triplet CTG repeats in the DMPK...
...Diego, La Jolla, Calif. Targets and Compounds Mentioned Cas9 - CRISPR-associated protein 9 DMPK - Dystrophia myotonica-protein kinase...
BioCentury | Jul 11, 2016
Finance

Rethinking risk

...disease," he said. As an example, he pointed to BIIB065 (IONIS-DMPK-2.5Rx). The antisense therapeutic targeting dystrophia myotonica-protein kinase (DMPK)...
...testing to treat myotonic dystrophy type 1 (DM1). "I'm looking forward to seeing the early DMPK...
...ITI-007 Schizophrenia Top-line Ph III data 2H16 Ionis Pharmaceuticals Inc. (NASDAQ:IONS) / Biogen Inc. (NASDAQ:BIIB) IONIS-DMPK-2.5Rx...
BioCentury | Apr 28, 2016
Finance

Virtually biotechs

...probably struggled." Harvard's Keith told BioCentury the accelerator employed CROs for the animal pharmacology and DMPK...
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