Grace Science LLC, a leading biotechnology company, has recently partnered with Andelyn Biosciences Inc., a Contract Development and Manufacturing Organization (CDMO) specializing in cell and gene therapy. Together, the companies aim to tech transfer and manufacture GS-100, a suspension process AAV NGLY1 gene therapy for Phase I/II/III clinical trial material. GS-100 is aimed at treating NGLY1 Deficiency, a serious and life-threatening disease with no approved treatment currently available. Patients with this disease experience debilitating symptoms throughout their lifetime.
The partnership between Andelyn Biosciences and Grace Science aims to maximize program efficiency, support ongoing clinical trials, and accelerate the availability of this critical therapy for patients suffering from NGLY1 Deficiency. GS-100 is a recombinant AAV9 vector that encodes the human NGLY1 gene. In 2021, GS-100 received orphan drug designation from both the FDA and the European Medicine Agency, as well as Rare Pediatric Disease Designation from the FDA.
As part of the partnership, Grace Science will benefit from Andelyn’s expertise in AAV processes and capabilities in late-stage manufacturing and commercial readiness. This collaboration will help accelerate manufacturing timelines, bringing hope to patients in need of treatment. Matt Niloff, Chief Commercial Officer at Andelyn Biosciences expressed excitement about working with Grace Science and highlighted both companies’ commitment to quality and patient-centric focus. Matt Wilsey CEO of Grace Science also emphasized the importance of this partnership stating that Andelyn’s expertise in AAV gene therapy manufacturing will ensure NGLY1 patients have access to safe and high-quality drug products.
Andelyn Biosciences is known for its excellence in providing CDMO services that enable biopharmaceutical clients to bring innovative therapies to market faster while maintaining regulatory compliance standards. The company’s team comprises highly skilled scientists and engineers with extensive experience in developing cell culture processes, optimizing protein purification protocols, designing bioprocessing systems, validating analytical methods and ensuring cGMP compliance standards are met.
Grace Science has been at the forefront of researching rare genetic diseases since its founding in 2013. The company’s mission is to develop new treatments for rare genetic diseases by leveraging cutting-edge science technology while focusing on patient outcomes first.
The successful dosing of the first NGLY1 Deficiency patient with GS-100 earlier this year marks an important milestone for both companies. Grace Science is planning to dose the second patient in May 2024 as part of their ongoing clinical trials.
In conclusion, this partnership between two leading biotechnology companies showcases their commitment towards innovation
