BioCentury | Oct 28, 2020
Product Development

Angelman trial pause for antisense oligo comes with efficacy data that lift Ultragenyx shares

GeneTx and Ultragenyx will look to adjust dosing and administration in their Phase I/II trial of GTX-102 to treat Angelman syndrome after patients experienced a serious adverse event thought...
BioCentury | Oct 24, 2020
Translation in Brief

UNC Chapel Hill Angelman gene therapy rescues motor function; plus data backing planned Pacylex, Kronos clinical trials and more

AAV gene editing therapy for Angelman syndromeUniversity of North Carolina at Chapel Hill scientists have created a CRISPR gene therapy for Angelman syndrome that ameliorated motor function deficits in a mouse model of the maternally...
BioCentury | Nov 15, 2019
Company News

Sarepta partners with StrideBio to enable viral gene therapy re-dosing

To enable repeat dosing of adeno-associated virus gene therapies, Sarepta is partnering with StrideBio to use the latter’s capsid technology in up to eight CNS and neuromuscular programs. The immunogenicity of viral antigens limits the...
BioCentury | Sep 19, 2019
Tools & Techniques

Atomwise's JV spree

Drug developers signing up to partner with Atomwise for hit discovery and lead optimization said deal structures made it attractive to take the plunge on the AI company's emerging technology. Last week, Atomwise Inc. announced...
BioCentury | Aug 14, 2019
Company News

Patient access, antisense oligo key to Angelman deal for Ultragenyx

Ultragenyx's partnership and option deal with Angelman syndrome company GeneTX delivers two components key to success in the rare disease: a candidate that targets the root cause of the disease, and a direct link to...
BioCentury | Jan 5, 2019

Ready to launch

The proposed $74 billion takeout of Celgene Corp. by Bristol-Myers Squibb Co. will not shift buysider attention away from upcoming and maturing drug launches heading into 2019, with plenty to track following a record year...
BioCentury | Nov 30, 2018
Targets & Mechanisms

Revving up metabolism at ASH 2018

Cancer metabolism is a prominent theme at this year’s ASH meeting, with researchers broadening the tent from metabolic targets that drive hematologic malignancies to those that intersect with other hot topics, notably immuno-oncology and epigenetics....
BioCentury | Nov 30, 2018
Targets & Mechanisms

Adhesion targets emerge at ASH 2018

The next wave of targets in blood cancers and hematology will come from adhesion molecules, innate immune cells and regulators of gene expression, according to BioCentury’s analysis of emerging targets at ASH 2018. BioCentury’s analysis...
BioCentury | Nov 1, 2018
Translation in Brief

New Therapeutic Targets and Biomarkers: October 2018

New Therapeutic Targets and Biomarkers: October 2018 Select top therapeutic targets and biomarkers covered by BioCentury or added to the BCIQ database during October. Therapeutic targets are defined as any protein, gene or other molecule...
BioCentury | Oct 3, 2018
Distillery Therapeutics


INDICATION: Melanoma Patient sample, cell culture and mouse studies suggest inhibiting UBE2N, UBE2V1, UBE2V2 or FOSL1 could help treat metastatic melanoma. In tumor samples from patients, levels of UBE2N mRNA were higher in metastatic tumors...
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