News

US FDA declines to approve Ultragenyx's gene therapy for rare genetic disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
William Blair Maintains a Buy on Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best oversold NASDAQ stocks to buy now. On July 14, William Blair ...
US FDA declines to approve Ultragenyx's gene therapy for rare inherited disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
Ultragenyx: The High Stakes Of Binary Events And A Short Cash Runway
Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...
FDA rejects Ultragenyx gene therapy over manufacturing concerns
While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...
FDA Delays Approval For Ultragenyx's Gene Therapy, But Not A Dealbreaker, Says Analyst
Ultragenyx's UX111 gene therapy faces FDA delay due to facility issues; analysts adjust price targets amid uncertain ...
Ultragenyx: 'Hold' The Best Strategy As Stock Tanks On Bone Disease Study Miss
Ultragenyx Pharmaceutical Inc. shares plunged 30% as UX143 trial raises efficacy doubts. Click for more on RARE and my ...
BioSpace2d
Ultragenyx Takes Second Hit as FDA Rejects Gene Therapy for Sanfilippo Syndrome
The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...
Fierce Pharma2d
Ultragenyx takes another hit as FDA snubs AAV gene therapy over manufacturing issues
The manufacturing-related rejection for UX111 is more than a setback for Ultragenyx, as it also delays a key test of the new ...
Ultragenyx Pharmaceutical, Apple And Other Big Stocks Moving Lower In Monday's Pre-Market Session
Ultragenyx received a Complete Response Letter from the FDA for UX111 AAV gene therapy to treat Sanfilippo Syndrome Type A ...
BioSpace6d
Ultragenyx, Mereo Plummet After Late-Stage Trial for Rare Genetic Disorder Appears To Fall Short
Partners Ultragenyx and Mereo BioPharma saw their stocks drop by 21% and 30%, respectively, after announcing that the Phase ...
STAT2d
FDA declines to approve Ultragenyx gene therapy
In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...