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 Innovations | Jun 27, 2019
Distillery Techniques

Patient-derived liver organoids for screening steatohepatitis

TECHNIQUES CATEGORY: Disease models TECHNOLOGY: 3D models Patient-derived liver organoids could be used to model steatohepatitis and screen therapies for the disease. The organoids were generated by differentiating human pluripotent stem cells into foregut spheroids;...
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...
BC Extra | May 6, 2019
Company News

Lundbeck’s $250M Abide takeout lands Tourette compound, discovery hub

Lundbeck will obtain its first pipeline candidate to treat Tourette syndrome via a deal to acquire Abide. The deal will give the Danish pharma a California-based discovery site, and could also help it implement a...
BC Week In Review | Oct 12, 2018
Clinical News

Phase II data for Merck's Btk inhibitor highlighted at ECTRIMS

Merck KGaA (Xetra:MRK) reported 24-week data from a Phase II trial to treat relapsing multiple sclerosis showing that evobrutinib (M2951) reduced the mean total number of gadolinium-enhancing T1 lesions by up to 2.7 over placebo....
BC Extra | Oct 12, 2018
Clinical News

Phase II data for Merck's Btk inhibitor highlighted at ECTRIMS

Merck KGaA (Xetra:MRK) reported 24-week data from a Phase II trial to treat relapsing multiple sclerosis showing that evobrutinib (M2951) reduced the mean total number of gadolinium-enhancing T1 lesions by up to 2.7 over placebo....
BC Week In Review | Aug 31, 2018
Clinical News

Priority Review for Alexion's long-acting PNH therapy

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) said FDA accepted and granted Priority Review to its BLA for ALXN1210 to treat paroxysmal nocturnal hemoglobinuria. Its PDUFA date is Feb. 18, 2019. ALXN1210 is a long-acting humanized mAb targeting...
BC Extra | Aug 20, 2018
Company News

Priority Review for Alexion's long-acting PNH therapy

Alexion Inc. (NASDAQ:ALXN) said FDA accepted and granted Priority Review to its BLA for ALXN1210 to treat paroxysmal nocturnal hemoglobinuria. Its PDUFA date is Feb. 18, 2019. ALXN1210 is a long-acting humanized mAb targeting complement...
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...
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