BioCentury | Jun 10, 2020
Emerging Company Profile

Autobahn: fine-tuning thyroid agonism to remyelinate axons

...Allergan plc in 2006-15. Finney pointed to a rare genetic disorder cause by mutations in SLC16A2...
...binding cassette subfamily D member 1 FAAH - Fatty acid amide hydrolase SLC16A2 (MCT8) - Solute carrier family 16 member 2...
BioCentury | Dec 20, 2019
Clinical News

Stealth cites endpoints in Phase III mitochondrial disease miss

Shares of Stealth BioTherapeutics sank 65% to $4.75 on Friday after the company announced a Phase III miss for its lead mitochondria-targeted therapy in primary mitochondrial myopathy, just two months after Alexion obtained an option...
BioCentury | Jul 18, 2019
Distillery Therapeutics

Inhibition of BACH1 or the genes it controls for metastatic lung cancer

DISEASE CATEGORY: Cancer INDICATION: Lung cancer Inhibiting the pro-metastatic transcription factor BACH1 or its gene targets MCT1, HMOX1, HK2 and GADPH could treat metastatic lung cancer. In patients with metastasized lung cancer, levels of BACH1...
BioCentury | Mar 29, 2019
Targets & Mechanisms

Next-wave targets at AACR 2019 start filling the blanks in the tumor microenvironment

New and emerging targets at AACR 2019 show cancer researchers are gaining granularity on how to manipulate the tumor microenvironment, a top priority goal for the field. The targets point to emerging strategies for modulating...
BioCentury | Sep 28, 2018
Targets & Mechanisms

Cell metabolism’s migration

As cancer metabolism undergoes a renaissance in the clinic, with compounds targeting growth factor and nutrient sensing pathways, basic research on metabolic processes in all cells is shining light on pathways that could yield new...
BioCentury | Aug 31, 2018
Clinical News

FDA clarifies path to submission for Ultragenyx's rare disease therapy

FDA has given Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) a clear path to an early NDA submission next year using existing data for UX007 to treat long-chain fatty acid oxidation disorders (LC-FAOD), CFO and EVP Shalini Sharp...
BioCentury | Aug 29, 2018
Company News

FDA clarifies path to submission for Ultragenyx's rare disease therapy

FDA has given Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) a clear path to an early NDA submission next year using existing data for UX007 to treat long-chain fatty acid oxidation disorders (LC-FAOD), CFO and EVP Shalini Sharp...
BioCentury | May 25, 2018
Clinical News

Allergy Therapeutics reports Phase II data for grass pollen therapy

Allergy Therapeutics plc (LSE:AGY) said GrassMATA MPL met the primary endpoint of establishing a "dose-response relationship" in improving allergy symptoms as measured by the change in total symptom score (TSS) following a conjunctival provocation test...
BioCentury | Jun 8, 2017
Translation in Brief

Barrier gateways

...The second study, published in Cell Stem Cell in May, showed mutations in solute carrier family 16 member 2 (; MCT8...
...intellectual and motor disabilities that characterize Allan-Herndon-Dudley syndrome - limit transport across patient-derived iBMECs. While SLC16A2-mutant...
...barrier deficits.” Cell Reports (2017); Vatine, G., et al. “Modeling psychomotor retardation using iPSCs from MCT8-deficient...
BioCentury | Jun 7, 2017
Distillery Techniques

Disease models

...triiodothyronine (T3) uptake and apical-to-basolateral transport that resulted from mutations in solute carrier family 16 member 2 ( SLC16A2 ; MCT8...
...Irvine University of Wisconsin-Madison ATP-binding cassette sub-family B member 1 (ABCB1) (MDR1) (PGP) (CD243) Fibroblast growth factor 2 (FGF2) Solute carrier family 16 member 2 (SLC16A2) (MCT8) Triiodothyronine...
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