Andelyn to tech transfer and manufacture AAV gene therapy for Grace

The deal will see contract development manufacturing organization (CDMO) Andelyn carry out the tech transfer and manufacturing for Grace’s suspension process AAV N-Glycanase 1 (NGLY1) gene therapy GS-100 for Phase I, II, and III clinical trial materials.

“We are privileged to work with a remarkable gene therapy company like Grace Science, whose tremendous dedication to rare diseases is an inspiration. Both Andelyn and Grace Science value strong collaboration and share a customer-centric focus and quality-first mindset. These synergies provide patients and their families confidence and hope for success of the GS-100 program,” said Matt Niloff, chief commercial officer at Andelyn.

The recombinant AAV9 vector works by encoding a full-length version of the human NGLY1 gene. GS-100 has received orphan drug designation (ODD) by the Food and Drug Administration (FDA) in June 2021 and by the European Medicine Agency (EMA) in September 2021. Additionally, the FDA granted the firm with Fast Track Designation in 2023.

Under the terms of the deal, of which financials have not been disclosed, Grace will use Andelyn’s knowledge in AAV processes and capabilities in late-stage production and commercialization. The aim of the collaboration is to advance manufacturing timelines and deliver the product to patients with an NGLY1 Deficiency.

Niloff told us there is no time limit associated with the partnership and “the established upstream and downstream process will be transferred to Andelyn to produce GMP material for the ongoing clinical trial.”

Grace said it had successfully dosed the first NGLY1 Deficiency patient with GS-100 in February and it expects to dose the second patient in May.