03:42 , Mar 9, 2018 |  BioCentury  |  Emerging Company Profile

Reviving lixivaptan

Single-asset play Palladio Biosciences Inc. is repurposing lixivaptan as a safer alternative to Jinarc tolvaptan, a competing vasopressin 2 (V2) receptor antagonist associated with hepatotoxicity in patients with autosomal dominant polycystic kidney disease. ADPKD is...
02:10 , Dec 8, 2017 |  BC Innovations  |  Targets & Mechanisms

Epiphenomena at ASH

While it’s no surprise that cell therapy research is dominant in hematology, the emergence of epigenetics as an equally large focus at this year’s ASH meeting is more notable, reinforcing the resurgence of a field...
19:22 , Jul 28, 2017 |  BioCentury  |  Regulation

Ganging up in rare disease

A joint proposal from EMA and FDA that encourages companies to join multidrug platform trials for pediatric Gaucher’s disease came too late to serve its original purpose. But the proposal could provide a template for...
18:46 , Jul 21, 2017 |  BC Week In Review  |  Company News

NICE final guidance recommends Sanofi's Cerdelga for Type I Gaucher's

Last month, the U.K.'s NICE issued final guidance recommending the use of Cerdelga eliglustat from the Sanofi Genzyme unit of Sanofi (Euronext:SAN; NYSE:SNY) to treat Type I Gaucher’s disease in adults who are cytochrome P450...
20:25 , Feb 16, 2017 |  BC Week In Review  |  Clinical News

GZ/SAR402671: Ph II MOVES-PD started

Sanofi’s Genzyme Corp. unit began the double-blind, placebo-controlled, dose-escalation, international Phase II MOVES-PD trial to evaluate once-daily oral GZ/SAR402671 in about 231 patients heterozygous for the glucocerebrosidase (GBA; GCase) gene mutation associated with PD. Sanofi...
22:38 , Jan 20, 2017 |  BioCentury  |  Emerging Company Profile

Aiming for durability

Avrobio Inc. is developing cell therapies that are transduced with lentivirus ex vivo to express therapeutic proteins. The approach could provide long-lasting treatments for patients with monogenic rare diseases or cancer. Data from Phase I...
07:00 , May 25, 2015 |  BC Week In Review  |  Clinical News

GZ/SAR402671: Phase IIa started

Sanofi began an open-label, international Phase IIa trial to evaluate once-daily oral GZ/SAR402671 for 6 months in 9 treatment-naive adult male patients. The trial includes a 2-year extension study. Sanofi (Euronext:SAN; NYSE:SNY), Paris, France  ...
07:00 , May 21, 2015 |  BC Innovations  |  Targets & Mechanisms

Intersection in marrow

An unlikely connection between Gaucher's disease and multiple myeloma has been uncovered by a U.K. group that found a common node in the pathways responsible for the bone disease symptoms that accompany both diseases. Suppression...
07:00 , May 4, 2015 |  BC Week In Review  |  Clinical News

GZ402671 regulatory update

FDA granted Fast Track designation to GZ/SAR402671 from Sanofi’s Genzyme Corp. company to treat Fabry’s disease. The glucosylceramide synthase (GCS) inhibitor is in a Phase IIa trial for the indication. Sanofi (Euronext:SAN; NYSE:SNY), Paris, France...
07:00 , May 4, 2015 |  BioCentury  |  Emerging Company Profile

Nixing Niemann-Pick

Vtesse Inc. is hoping to change the course of disease in Niemann-Pick type C, a fatal ultra-Orphan lysosomal storage disorder, using a compound plucked from NIH's Therapeutics for Rare and Neglected Diseases repurposing project. Niemann-Pick...