- Data show that AXV-101 halts photoreceptor and outer nuclear retinal layer (ONL) degeneration in a dose-dependent manner - - Company to initiate clinical studies in 1H 2025 to treat blindness and progress AXV-101 program to also treat obesity by 2026 - LONDON, May 10, 2024 (GLOBE NEWSWIRE) -- Axovia Therapeutics Ltd., a biotechnology company developing therapies to address the genetic causes of blindness and obesity, announced the presentation of preclinical data supporting lead program AXV-101, which is being developed to address blindness and obesity associated with Bardet-Biedl Syndrome (BBS).

-数据显示，AXV-101以剂量依赖的方式停止光感受器和外核视网膜层（ONL）变性-该公司将于2025年1月开始临床研究，以治疗失明，并在2026年前推进AXV-101计划，以同时治疗肥胖-伦敦，2024年5月10日（环球通讯社）-Axovia Therapeutics Ltd.，一家开发治疗失明和肥胖遗传原因的生物技术公司，宣布推出临床前数据支持领导计划AXV-101，该计划正在开发中，以解决与Bardet-Biedl综合征（BBS）相关的失明和肥胖问题。

These data are being presented today by Co-Founder and Chief Scientific Officer, Dr. Victor Hernandez, at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held in Baltimore, MD from May 7-11, 2024. “Today’s presentation highlights that AXV-101 has demonstrated efficacy, safety and durability to halt retinal degeneration in the BBS1 animal model,” said Dr.

这些数据今天由联合创始人兼首席科学官维克多·埃尔南德斯博士在2024年5月7日至11日于马里兰州巴尔的摩举行的美国基因与细胞治疗学会（ASGCT）第27届年会上发表。“今天的演讲强调了AXV-101在BBS1动物模型中已经证明了阻止视网膜变性的有效性、安全性和耐久性，”Dr。

Hernandez. “BBS patients carrying biallelic mutations in the BBS1 gene begin to experience vision loss in childhood leading to blindness before 20 years of age. Our novel gene therapy utilizes an adeno-associated virus (AAV9) to deliver a functional copy of the faulty BBS gene and these data give us further confidence in the therapeutic potential of our program.” “We are very encouraged by these data which establish that AXV-101 acts in a dose dependent manner and we are diligently advancing this program toward clinical study initiation in the first half of 2025,” said Professor Phil Beales, Chief Executive Officer and Co-Founder at Axovia Therapeutics.

埃尔南德斯。“携带BBS1基因双等位基因突变的BBS患者在20岁之前就开始经历儿童时期的视力丧失，导致失明。我们的新型基因疗法利用腺相关病毒（AAV9）来传递有缺陷的BBS基因的功能拷贝，这些数据使我们对我们计划的治疗潜力有了进一步的信心。”“我们对这些数据感到非常鼓舞，这些数据表明AXV-101以剂量依赖的方式起作用，我们正在努力推动该计划在2025年上半年开始临床研究，”Axovia Therapeutics首席执行官兼联合创始人菲尔·比尔斯教授说。

“These data suggest that our BBS1 novel gene therapy can modify the underlying disease of BBS, including rescuing vision loss by halti.

“这些数据表明，我们的BBS1新型基因疗法可以改变BBS的潜在疾病，包括通过halti挽救视力丧失。