CGT specialist Andelyn Biosciences has begun commercial manufacturing of Ultragenyx Pharmaceutical’s Fayuvi gene therapy at its facility in Columbus, Ohio, following US FDA approval of the treatment for Sanfilippo syndrome type A.
The milestone marks the first FDA-approved gene therapy manufactured using Andelyn’s AAV Curator Platform, according to the CDMO.
Fayuvi approval follows manufacturing hurdles
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf) last Thursday as a one-time intravenous gene therapy to treat neurological manifestations of mucopolysaccharidosis type IIIA (MPS IIIA) in paediatric patients with preserved neurodevelopmental function.
Fayuvi, previously known as UX111, uses an AAV vector to deliver a functional copy of the SGSH gene to patients’ cells. According to Ultragenyx, around 3000 to 5000 people in the developed world are affected by Sanfilippo syndrome type A.
Last week's FDA approval follows an earlier regulatory setback for Ultragenyx.
In 2025, the agency declined to approve Fayuvi due to manufacturing-related issues involving Ultragenyx’s own gene therapy production facility and a third-party manufacturer.
Ultragenyx subsequently resubmitted the application in April 2026, with the FDA granting it priority review.
The company has said Fayuvi is now manufactured at its Bedford, Massachusetts facility and by Andelyn Biosciences in Ohio, making Andelyn’s role in commercial supply a key part of the programme’s progression to approval.
For Andelyn, FDA approval marks the firm's transition from clinical-stage manufacturing to commercial supply.
It said its AAV Curator Platform uses a configurable, data-driven approach that combines process development, modular manufacturing strategies and analytics to support AAV gene therapy programmes from development through commercialisation.
The build-up towards commercial gene therapy manufacturing
The milestone follows a series of moves by Andelyn to expand its gene therapy manufacturing capabilities.
In May, the company received full certification under Dark Horse Consulting’s Initiative for Certification of Manufacturing Capabilities (ICMC) programme, which assesses manufacturing systems and capabilities against criteria based on global regulatory standards for commercial advanced-therapy manufacturing.
More recently, Andelyn was selected by Genprex to support process optimisation, scale-up, analytical development and cGMP manufacturing for the company’s AAV-based diabetes gene therapy programme.
The collaboration is intended to support IND-enabling studies and subsequent clinical development.
Andelyn has also continued to apply its Curator Platform to earlier-stage programmes, including an AAV9 gene therapy being developed with Queen’s University for GM2 gangliosidoses.
The company said the Fayuvi milestone demonstrates its ability to now support gene therapy programmes across development, clinical manufacturing and commercial supply, while positioning its Curator Platform for further late-stage programmes
“We congratulate Ultragenyx on this significant achievement and recognise the MPS IIIA community, investigators and everyone who contributed to advancing this programme,” said Wade Macedone, CEO of Andelyn Biosciences.
“We are proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator Platform process."
This milestone reflects the purpose behind Andelyn’s founding and underscores the importance of building commercial readiness through rigorous science, accountable execution and quality at every stage.
"We look forward to supporting our other late-stage clients and the industry with our FDA-stamped commercial capabilities.”
