Andelyn Biosciences, a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapies, has joined forces with Grace Science, a dedicated gene therapy company, to advance the development and manufacturing of GS-100, a potentially life-saving gene therapy for NGLY1 Deficiency.

NGLY1 Deficiency is a severe, life-threatening condition characterized by developmental delay, cognitive impairment, movement disorders, and other debilitating neurological symptoms. With no approved therapy available, patients face a lifetime of challenges. GS-100, a recombinant AAV9 vector encoding the human NGLY1 gene, holds promise as a groundbreaking treatment for this rare disease.

In a strategic partnership, Grace Science will leverage Andelyn's expertise in AAV processes and late-stage manufacturing to accelerate the production of GS-100 for Phase I/II/III clinical trial material. The collaboration aims to maximize program efficiency, support ongoing clinical trials, and expedite the delivery of this critical therapy to patients in need.

GS-100 received orphan drug designation from both the FDA and the European Medicine Agency (EMA) in 2021, emphasizing its potential to address unmet medical needs. Additionally, the therapy was granted Rare Pediatric Disease Designation by the FDA, offering incentives for expedited review and approval.

Grace Science recently achieved a significant milestone by dosing the first NGLY1 Deficiency patient with GS-100 in February 2024, with plans to dose the second patient in May of the same year. The partnership with Andelyn is poised to enhance manufacturing capabilities, ensuring the delivery of safe and high-quality drug products for clinical trials and beyond.

Matt Niloff, Chief Commercial Officer at Andelyn Biosciences, expressed enthusiasm for the collaboration, emphasizing the shared commitment to quality and patient-centric focus. He noted, "We are privileged to work with a remarkable gene therapy company like Grace Science, whose tremendous dedication to rare diseases is an inspiration."

Matt Wilsey, CEO and Co-Founder of Grace Science echoed the sentiment, highlighting the importance of Andelyn's expertise in advancing the GS-100 program. "Andelyn's deep expertise and experience in AAV gene therapy manufacturing will assure that our NGLY1 patients have access to safe, high-quality drug products for our clinical trial and beyond," Wilsey stated.

With a focus on advancing client discoveries and manufacturing life-altering therapies, Andelyn Biosciences continues to drive innovation in the field of cell and gene therapies. Founded on its extensive experience in AAV development and manufacturing, Andelyn is committed to delivering hope and transformative treatments for rare and prevalent diseases.