00:47 , Jun 18, 2019 |  BC Extra  |  Company News

Latest twist in uniQure’s tale: possible sale

Five years after returning to the public markets, uniQure may be on the block. While the Dutch gene therapy play declined to comment on a Bloomberg report citing undisclosed sources that the biotech was exploring...
22:15 , Jun 17, 2019 |  BC Extra  |  Company News

Trial data suggest 80% of Zynteglo patients could meet milestones in outcomes-based pricing model

The most recent long-term data for bluebird bio's Zynteglo β thalassemia gene therapy show that about 80% of patients could hit payment milestones based on details the biotech disclosed about its outcomes-based pricing model on...
23:07 , Jul 20, 2018 |  BioCentury  |  Regulation

FDA’s positive reinforcement

Companies are still sifting through a half dozen gene therapy guidance documents issued by FDA, but so far it appears the recommendations support the paths taken by some of the most advanced clinical candidates for...
21:44 , Jul 10, 2018 |  BC Innovations  |  Distillery Techniques

Biomarkers; other

TECHNOLOGY: Tissue markers; metabolomics; plasma markers; computational models Computational models based on profiles of the fecal metagenome, plasma and urine metabolites and the liver transcriptome could help diagnose hepatic steatosis in non-diabetic obese women. In...
18:15 , May 11, 2018 |  BC Week In Review  |  Clinical News

FDA panel backs volanesorsen approval

FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 12-8 to approve Waylivra volanesorsen from Akcea Therapeutics Inc. (NASDAQ:AKCA) to treat familial chylomicronemia syndrome, a rare lipid disorder that results from lipoprotein lipase deficiency. The company...
22:09 , May 10, 2018 |  BC Extra  |  Company News

FDA panel backs volanesorsen approval

FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted 12-8 to approve Waylivra volanesorsen from Akcea Therapeutics Inc. (NASDAQ:AKCA) to treat familial chylomicronemia syndrome, a rare lipid disorder that results from lipoprotein lipase deficiency. The company...
20:05 , May 8, 2018 |  BC Extra  |  Company News

FDA reviewers question Akcea candidate's safety

FDA reviewers said volanesorsen from Akcea Therapeutics Inc. (NASDAQ:AKCA) will "almost certainly" require a REMS if approved, although even that step "may not be sufficient" to ensure the candidate's safe use due to risks of...
16:48 , Mar 2, 2018 |  BC Week In Review  |  Clinical News

Akcea discloses new thrombocytopenia case for volanesorsen

Akcea Therapeutics Inc. (NASDAQ:AKCA) disclosed an additional case of grade 4 thrombocytopenia in an open-label extension of its volanesorsen (IONIS-APOCIIIRx) to treat familial chylomicronemia syndrome (FCS), a rare lipid disorder that results from lipoprotein lipase...
23:16 , Feb 27, 2018 |  BC Extra  |  Clinical News

Akcea falls on new thrombocytopenia disclosure for volanesorsen

Akcea Therapeutics Inc. (NASDAQ:AKCA) lost $5.78 (26%) to $16.80 on Tuesday after disclosing an additional case of grade 4 thrombocytopenia in an open-label extension of its volanesorsen (IONIS-APOCIIIRx) to treat familial chylomicronemia syndrome (FCS), a...
20:17 , Feb 23, 2018 |  BC Week In Review  |  Clinical News

FDA panel to review volanesorsen from Akcea, Ionis

Akcea Therapeutics Inc. (NASDAQ:AKCA) said FDA's Division of Metabolism and Endocrinology Products will hold an advisory committee meeting to review an NDA for volanesorsen (IONIS-APOCIIIRx) to treat familial chylomicronemia syndrome (FCS), a rare lipid disorder...