Andelyn Biosciences Selected By Ultragenyx For Late-Stage Process Performance Qualification Manufacturing Of Novel Gene Therapy For Sanfilippo Syndrome
Portfolio Pulse from Happy Mohamed
Andelyn Biosciences has been chosen by Ultragenyx (NASDAQ:RARE) to perform late-stage PPQ manufacturing of UX111, a gene therapy for Sanfilippo Syndrome. UX111 has received multiple designations in the U.S. and EU, and Andelyn has already manufactured GMP supply for patient dosing. The PPQ batches are for future regulatory filings, and the partnership aims to expedite treatment for MPS IIIA patients.
December 18, 2023 | 2:38 pm
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Ultragenyx (NASDAQ:RARE) is advancing its UX111 gene therapy program for Sanfilippo Syndrome by partnering with Andelyn Biosciences for PPQ manufacturing, which is crucial for future regulatory filings.
The news of Andelyn Biosciences being selected for the PPQ manufacturing of UX111 is likely to be viewed positively by investors as it demonstrates progress in the development pipeline of Ultragenyx. The multiple designations received by UX111 in the U.S. and EU suggest a high potential for the therapy, and successful PPQ manufacturing is a critical step towards regulatory approval. This could potentially lead to short-term positive sentiment for Ultragenyx's stock.
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