Rapid Gene Therapy for NGLY1 Deficiency: Andelyn Biosciences Teams Up with Grace Science to Accelerate Manufacturing and Deliver Hope

By Eleanor Harrison Mar25,2024
Andelyn Biosciences Collaborates with Grace Science to Further Develop GS-100

In a bid to expedite the availability of crucial gene therapy for NGLY1 Deficiency, Andelyn Biosciences Inc. has teamed up with Grace Science LLC to transfer technology and manufacture GS-100. GS-100 is an AAV NGLY1 gene therapy aimed at treating NGLY1 Deficiency, a serious and life-threatening disease without any approved therapy currently available.

NGLY1 Deficiency is a debilitating condition that afflicts patients with life-long symptoms. The collaboration between Andelyn Biosciences and Grace Science seeks to increase program efficiency, support ongoing clinical trials, and provide hope to patients in need.

GS-100 is a recombinant AAV9 vector containing the full-length human NGLY1 gene. In 2021, it received orphan drug designation (ODD) from the FDA and the European Medicine Agency (EMA). The FDA also granted Rare Pediatric Disease Designation to GS-100 in 2021, potentially leading to a Priority Review Voucher upon approval for marketing and a Fast-Track designation in 2023. Grace Science successfully administered the first dose of GS-100 to an NGLY1 Deficiency patient in February of this year and plans to treat a second patient in May 2024.

Through this partnership, Grace Science will leverage Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness to expedite its manufacturing timelines, offering hope to patients with NGLY1 Deficiency. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed gratitude for the opportunity to collaborate with Grace Science. He highlighted their shared values of strong collaboration, customer-centric focus, and a quality-first mindset, which instills confidence and hope in the success of the GS-100 program. Matt Wilsey, CEO and Co-Founder of Grace Science, emphasized his excitement about the partnership with Andelyn Biosciences. He noted that Andelyn’s expertise and experience in AAV gene therapy manufacturing would ensure that NGLY1 patients receive safe, high-quality drug products for clinical trials and beyond.

The collaboration between these two organizations represents an important step forward in bringing hopeful treatment options to those affected by NGLY1 Deficiency. With their combined expertise and commitment to excellence, they are working together towards delivering on their shared mission: providing innovative solutions for complex medical conditions like NGLY

By Eleanor Harrison

As a content writer at newseasoning.com, I infuse flavor into words, crafting compelling stories that captivate and inform our audience. With a keen eye for detail and a passion for creativity, I strive to create content that not only engages but also inspires. Whether I'm concocting a savory blog post or whipping up a spicy product description, I pour my heart and soul into every piece I write. Join me on this flavorful journey as we explore the tantalizing world of content creation together.

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