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Transforming the Future of NGLY1 Deficiency: Grace Science and Andelyn Biosciences Announce Partnership for GS-100 Gene Therapy

Andelyn Biosciences Collaborates with Grace Science to Further Develop GS-100

Transforming the Future of NGLY1 Deficiency: Grace Science and Andelyn Biosciences Announce Partnership for GS-100 Gene Therapy

Grace Science LLC and Andelyn Biosciences Inc., two leading players in the cell and gene therapy industry, have announced a partnership to transfer technology and manufacture GS-100, a novel treatment for NGLY1 Deficiency. The collaboration aims to expedite the development and delivery of this critical therapy to patients who are in dire need of it.

NGLY1 Deficiency is a rare genetic disorder that affects an estimated 1 in 50,000 people worldwide. The disease causes severe symptoms such as muscle weakness, neurological impairment, and organ failure. Currently, there is no cure or approved therapy for NGLY1 Deficiency, making it a life-threatening condition.

GS-100 is a promising gene therapy that uses AAV9 vectors to deliver the full-length human NGLY1 gene to patients’ cells. In 2021, GS-100 received orphan drug designation (ODD) from both the US Food and Drug Administration (FDA) and the European Medicine Agency (EMA). Additionally, GS-100 was granted Rare Pediatric Disease Designation by the FDA in 2021, which may result in a Priority Review Voucher upon approval for marketing and a Fast-Track designation in 2023.

The collaboration between Grace Science and Andelyn Biosciences seeks to increase program efficiency, support ongoing clinical trials, and expedite access to this vital therapy for patients with NGLY1 Deficiency. Through this partnership, Andelyn Biosciences will bring its expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness to bear on accelerating Grace Science’s manufacturing timelines.

Matt Wilsey, CEO of Grace Science expressed his excitement about collaborating with Andelyn Biosciences. He noted that their combined strengths would enable them to provide safe, high-quality drug products for clinical trials and beyond. “We are thrilled about our partnership with Andelyn Biosciences,” said Wilsey. “We believe that their expertise will help us achieve our goal of providing hope for NGLY1 patients.”

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences shared similar sentiments about the collaboration: “We are proud to be working with Grace Science on this important project,” he said. “Our shared values of strong collaboration, customer focus

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