Charles River Laboratories International has entered into a manufacturing collaboration with the Faculty of Medicine at Gazi University to support the development of a gene therapy programme focused on a rare genetic disorder.
查爾斯河實驗室國際公司與加齊大學醫學院建立了製造合作關係，以支持開發專注於一種罕見遺傳病的基因治療計劃。
The partnership will provide plasmid DNA for adeno-associated virus (AAV) production and in vitro efficacy studies.
該合作將為腺相關病毒（AAV）生產和體外功效研究提供質粒DNA。
The collaboration centres on research into hyperphosphatemic tumoral calcinosis (HTC), a rare inherited condition characterised by elevated phosphate levels in the blood and painful calcium phosphate deposits in soft tissues, particularly around major joints. These deposits can cause inflammation, pain, and restricted movement.
合作的重點是對高磷血癥性腫瘤樣鈣質沉着症 (HTC) 的研究，這是一種罕見的遺傳性疾病，其特徵是血液中磷酸鹽水平升高，並在軟組織（尤其是主要關節周圍）形成疼痛的磷酸鈣沉積。這些沉積物可能引發炎症、疼痛和活動受限。
Researchers at Gazi University have identified defects in the GALNT3 gene as a key cause of the disorder..
加齊大學的研究人員已確定GALNT3基因的缺陷是該疾病的一個關鍵原因。
As part of the agreement, Charles River will supply pre-manufactured, research-grade AAV plasmids to support early-stage gene therapy development. These plasmids are produced using animal component-free processes and are designed to be ready for immediate use. The approach is intended to help academic researchers advance programmes more efficiently while managing development timelines and costs..
根據協議，查爾斯河將提供預先製造的、研究級別的AAV質粒，以支持早期基因治療開發。這些質粒採用無動物成分的工藝生產，並設計為可立即使用。該方法旨在幫助學術研究人員更高效地推進項目，同時管理開發時間和成本。
The partnership brings together academic expertise in rare disease genetics with industrial capabilities in cell and gene therapy manufacturing. It is expected to support the translation of laboratory research into potential therapeutic solutions for patients with limited treatment options.
該合作將罕見病遺傳學的學術專業知識與細胞和基因治療製造的工業能力相結合。預計它將支持實驗室研究轉化為針對治療選擇有限的患者的潛在治療方案。
The company provides integrated solutions across the development lifecycle, from preclinical research through to commercial-scale production of plasmid DNA, viral vectors, and cell-based therapies.
該公司提供貫穿開發生命周期的綜合解決方案，從臨牀前研究一直到質粒DNA、病毒載體和細胞療法的商業化規模生產。
Charles River continues to expand its cell and gene therapy CDMO offering, which spans discovery support, safety assessment, biologics testing, and manufacturing services.
查爾斯河繼續擴展其細胞和基因治療CDMO服務，涵蓋發現支持、安全性評估、生物製品測試及生產服務。
Source: criver.com
來源：criver.com