NEW YORK, Oct. 12, 2023 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today the publication of a new research paper in Molecular Therapy – Methods & Clinical Development, demonstrating the efficacy of its TALEN-mediated gene correction of mutated PIK3CD gene in APDS1 T-cells.

纽约，2023年10月12日（GLOBE NEWSWIRE）-Cellectis（“公司”）（Euronext Growth:ALCLS-纳斯达克股票代码：CLLS），一家临床阶段生物技术公司，利用其开创性的基因编辑平台开发挽救生命的细胞和基因疗法，今天宣布发表一篇关于分子疗法-方法和临床开发的新研究论文，证明了其TALEN介导的APDS1 T细胞中突变的PIK3CD基因的基因校正的功效。

The research work described in this article was jointly conducted by Imagine Institute and Cellectis teams. About APDS1: Activated phosphoinositide 3-kinase δ syndrome (also known as APDS type 1 or APDS1) is a rare but devastating disease caused by gain-of-function mutations in the PIK3CD gene and resulting in a combined immunodeficiency.

本文描述的研究工作由Imagine Institute和Cellectis团队共同完成。关于APDS1：活化的磷酸肌醇3-激酶δ综合征（也称为APDS 1型或APDS1）是一种罕见但具有破坏性的疾病，由PIK3CD基因的功能获得性突变引起并导致联合免疫缺陷。

Approved treatments for APDS1 consist in prophylactic measures including long term antibiotics and Ig (immunoglobulin) replacement therapy. Allogeneic hematopoietic stem/progenitor cell (HSPC) transplantation has been proposed as a definitive treatment for APDS1. However, the lack of compatible donor as well as graft failure, graft instability, and poor graft function are still major challenges that must be overcome to reach a positive therapeutic outcome.

批准的APDS1治疗包括预防措施，包括长期抗生素和Ig（免疫球蛋白）替代治疗。异基因造血干/祖细胞（HSPC）移植已被提议作为APDS1的确定性治疗方法。然而，缺乏相容的供体以及移植物衰竭，移植物不稳定和移植物功能差仍然是获得积极治疗结果必须克服的主要挑战。

Thus, so far there are neither optimal nor long-term therapeutic solutions for APDS1 patients and new alternative treatments are highly regarded. The study published here aims at exploring an alternative therapeutic strategy by correcting the mutated PIK3CD gene associated to APDS1 by gene editing. This article describes a TALEN®-mediated gene insertion strategy that allows targeted correction of the dominant gain-of-function mutation of the PIK3CD gene by insertion of a functional sequence in a precise manner.

因此，到目前为止，还没有针对APDS1患者的最佳或长期治疗方案，并且高度重视新的替代疗法。此处发表的研究旨在通过基因编辑纠正与APDS1相关的突变PIK3CD基因，从而探索另一种治疗策略。本文介绍了一种TALEN®介导的基因插入策略，该策略允许通过精确插入功能序列来靶向校正PIK3CD基因的主要功能获得性突变。

Results show efficien.

结果显示效率。