Dive Brief:

潜水简介：

A profoundly deaf baby who received Regeneron’s experimental gene therapy was able to hear normally within six months, the company announced Wednesday.

该公司周三宣布，一名接受Regeneron实验性基因治疗的重度失聪婴儿在六个月内能够正常听力。

The data, presented at a medical conference, build on initially promising research released in October. The baby was dosed at 11 months of age, one of the youngest children in the world to receive gene therapy for genetic deafness, Regeneron said. A second patient, treated at age 4, also showed hearing improvements at a six-week assessment, the company said..

在一次医学会议上公布的数据是建立在10月份发布的最初有希望的研究基础上的。雷杰隆说，这名婴儿在11个月大时服用了药物，是世界上接受基因治疗遗传性耳聋的最小儿童之一。该公司表示，第二名患者在4岁时接受治疗，在为期六周的评估中也显示出听力改善。。

Both children suffer from a type of blindness caused by mutations in a gene known as otoferlin. The initial research on Regeneron’s DB-OTO therapy is part of an ongoing Phase 1/2 trial known as CHORD enrolling infants and children in the U.S., U.K. and Spain.

两个孩子都患有一种由称为otoferlin的基因突变引起的失明。Regeneron DB-OTO治疗的初步研究是正在进行的1/2期试验的一部分，该试验在美国、英国和西班牙被称为CHORD登记婴儿和儿童。

Dive Insight:

潜水洞察：

Regeneron’s announcement follows news that Eli Lilly’s competing experimental gene therapy gave an 11-year-old boy who was born deaf the ability to hear. The treatment, which Lilly acquired in a buyout of biotechnology company Akouos, also targets otoferlin.

Regeneron宣布这一消息之前，有消息称礼来公司（EliLilly）的竞争性实验性基因疗法使一名天生聋哑的11岁男孩具备了听力。礼来公司在收购生物技术公司Akouos的过程中收购了该疗法，该疗法也针对otoferlin。

The results suggest gene therapy may become a viable therapeutic option for people with congenital deafness. The field has attracted a great deal of attention, as evidenced in abstracts of data presented at the American Society of Gene and Cell Therapy’s yearly meeting. One, from a group of researchers based in China and the U.S., found hearing recovery in 10 out of 11 patients..

结果表明，基因治疗可能成为先天性耳聋患者可行的治疗选择。正如美国基因与细胞治疗学会年会上提交的数据摘要所证明的那样，该领域引起了极大的关注。来自中国和美国的一组研究人员发现，11名患者中有10名听力恢复。。

Regeneron acquired its entrant in the field with its $109 million purchase of Decibel Therapeutics in August. The deal includes potential payments that could add up to $104 million more for Decibel shareholders for reaching certain milestones.

8月，Regeneron斥资1.09亿美元收购了分贝治疗公司（DecibelTherapeutics），收购了该领域的进入者。这笔交易包括可能为达到某些里程碑的分贝股东额外支付1.04亿美元。

The success with such a young child in the Regeneron trial of DB-OTO may offer hope for other developmental milestones, such as speech, said Lawrence Lustig, a study investigator and chair of the department of otolaryngology – head and neck surgery at Columbia University.

哥伦比亚大学（Columbia University）耳鼻咽喉头颈外科系主任兼研究研究员劳伦斯·卢斯蒂格（Lawrence Lustig）表示，在DB-OTO的Regeneron试验中，如此年幼的孩子获得成功，可能会为其他发展里程碑带来希望，例如言语。

“The opportunity of providing the full complexity and spectrum of sound in children born with profound genetic deafness is a phenomenon I did not expect to see in my lifetime,” Lustig said in Regeneron’s statement. “We are excited to see how this translates into an individual’s development.”

鲁斯蒂格在Regeneron的声明中说：“有机会为出生时患有严重遗传性耳聋的儿童提供完全复杂和频谱的声音，这是我一生中从未见过的现象。”。“我们很高兴看到这如何转化为个人的发展。”

DB-OTO is designed to deliver a working copy of the otoferlin gene with an injection into the cochlea after patients are sedated with general anesthesia. Like Lilly’s experimental treatment, Regeneron’s medicine uses a specially engineered virus to deliver the gene.

DB-OTO的设计目的是在患者全身麻醉镇静后，通过耳蜗注射将耳铁蛋白基因的工作拷贝传递到耳蜗中。就像莉莉的实验治疗一样，Regeneron的药物使用一种特殊的工程病毒来传递基因。

Regeneron’s therapy has received orphan drug, rare pediatric disease and fast track designations from the Food and Drug Administration.

Regeneron的治疗已获得食品和药物管理局的孤儿药、罕见儿科疾病和快速通道指定。