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Zacks Investment Research on MSN2 天
Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA
Ultragenyx Pharmaceuticals RARE announced that the FDA has issued a Complete Response Letter (CRL) for its biologics license ...
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 ...
Fierce Pharma3 天
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 ...
BioPharma Dive3 天
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 ...
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 ...
The Pharma Letter3 天
Ultragenyx hit with FDA delay for gene therapy targeting rare childhood disorder
Ultragenyx Pharmaceutical (Nasdaq: RARE) has been dealt a regulatory setback, as the American medicines regulator has ...
Bank of America Securities Reiterated a Buy Rating on Ultragenyx Pharmaceutical (RARE)
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the Best 52-Week Low Stocks to Buy According to Analysts. On July 14, ...
STAT3 天
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 ...
The Manila Times6 天
Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat ...
Complete Response Letter (CRL) cited specific chemistry, manufacturing and controls (CMC) related observations that are ...
GlobalData on MSN3 天
Ultragenyx receives complete response letter for UX111 BLA
The FDA's request stems from observations made during recent inspections of Ultragenyx's manufacturing facilities.