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...

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...

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...

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...

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...

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...

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....

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...

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...

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...