BioCentury | Sep 7, 2019
Product Development

Regeneron’s evinacumab data could make it the therapy of choice in HoFH

With a cleaner safety profile and better efficacy than marketed agents, Regeneron’s first-in-class evinacumab is poised to become the standard of care for homozygous familial hypercholesterolemia and other rare lipid disorders, including for the hardest...
BC Extra | Jun 18, 2019
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...
BC Extra | Jun 17, 2019
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...
BioCentury | Jul 20, 2018
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...
BC Innovations | Jul 10, 2018
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...
BC Week In Review | May 11, 2018
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...
BC Extra | May 10, 2018
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...
BC Extra | May 8, 2018
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...
BC Week In Review | Mar 2, 2018
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...
BC Extra | Feb 27, 2018
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...
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