News

Fierce Pharma9d
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 ...
BioSpace13d
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 ...
Biotech Stock Ultragenyx Is Falling on Unsatisfying Trial Update
Ultragenyx and partner Mereo BioPharma’s brittle bone disease trial will continue. Investors had wanted data good enough to ...
BioSpace9d
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 ...
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 ...
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 ...
BioPharma Dive9d
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 ...
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 ...
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 ...
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 ...
Fierce Biotech13d
Lack of early win for Ultragenyx, Mereo’s bone disorder med dents stock, but analysts keep faith
Analysts are keeping the faith in Ultragenyx’s brittle bone disease medicines despite the monoclonal antibody failing to score an early outright win in an ongoing phase 3 trail. | Analysts are keeping ...
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA)
Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are ...