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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 ...
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 ...
Fierce Pharma5d
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 ...
BioSpace5d
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 ...
Pharmabiz5d
Ultragenyx receives CRL from US FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
Novato, California Monday, July 14, 2025, 16:00 Hrs [IST] ...
BioPharma Dive5d
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 inherited disorder
The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...
The Manila Times7d
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 ...
The Pharma Letter5d
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 ...
Nasdaq5mon
Ultragenyx Announces New Data For UX111 Gene Therapy In ... - Nasdaq
(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo ...
Business Insider1y
Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in ...
NOVATO, Calif., Feb. 06, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced data demonstrating treatment with UX111 (ABO-102) AAV gene therapy resulted in rapid ...
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 ...