Grace Science LLC and Andelin Biosciences Inc. have partnered to develop and manufacture GS-100, a promising gene therapy for the treatment of NGLI1 deficiency. NGLI1 is a rare and debilitating disease with no approved therapy, affecting patients worldwide. The partnership aims to maximize the effectiveness of the program, support ongoing clinical trials, and accelerate critical therapy for patients suffering from this life-threatening disease.

GS-100 is a recombinant AAV9 vector encoding a full-length version of the human NGLI1 gene. It has been granted Orphan Drug Designation (ODD) by the FDA and EMA in 2021, along with Rare Pediatric Disease Designation by the FDA. Grace Science successfully dosed the first NGLI1-deficient patient with GS-100 in February of this year and plans to dose a second patient in May 2024.

In this partnership, Grace Science will leverage Andelin’s expertise in AAV processes and late-stage manufacturing and commercial readiness capabilities to accelerate its manufacturing timelines. Matt Niloff, chief commercial officer at Andelin Biosciences, emphasized the strong collaboration and customer focus shared by both companies, providing confidence and hope in the success of the GS-100 program.

Matt Wilsey, CEO and co-founder of Grace Science, expressed his excitement about the partnership, stating that Andelin’s expertise in AAV gene therapy manufacturing will ensure that NGLI1 patients have access to safe, high-quality medicines for their clinical trials going forward. The partnership between Andelin Biosciences and Grace Science aims to bring hope and relief to patients suffering from NGLI1 deficiency through the development and production of GS-100.

By Samantha Johnson

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