BioCentury | Jun 10, 2020
Emerging Company Profile

Autobahn: fine-tuning thyroid agonism to remyelinate axons

Autobahn, which launched with a $76 million series B Tuesday, joins at least six other companies with disclosed remyelination programs for multiple sclerosis. But the biotech thinks its prodrug, slated to enter the clinic next...
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

With induced pluripotent stem (iPS) cells making inroads into more and more areas of biology, two groups have now used the technology to generate models of the blood-brain barrier and pinpoint new molecular mechanisms contributing...
BioCentury | Jun 7, 2017
Distillery Techniques

Disease models

TECHNOLOGY: Cell models iPS cell-derived brain microvascular endothelial cells could serve as BBB models for HD or Allan-Herndon-Dudley syndrome. The cells were generated by co-culturing iPS cells derived from patients in endothelial cell-conditioned media with...
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