LONDON--(BUSINESS WIRE)--AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN).

伦敦--（商业新闻短讯）--AviadoBio是一家开创性的基因治疗公司，致力于开发和提供潜在的神经退行性疾病转化药物，今天宣布，第一名患者已在1/2期ASPIRE-FTD试验中接受治疗，该试验评估了AviadoBio的研究性基因治疗AVB-101，用于额颞叶痴呆（FTD）伴颗粒蛋白前体（GRN）基因突变（FTD-GRN）的患者。

Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States..

临床试验地点目前在波兰、西班牙和荷兰开放，预计将在包括美国在内的多个国家开设更多地点。。

“AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” said Lisa Deschamps, CEO. “We are thankful for the dedication of the ASPIRE-FTD clinical investigators studying AVB-101 and immensely grateful for the families who participate in clinical trials for new treatment options that may change the future for generations of families living with FTD-GRN.”.

首席执行官丽莎·德尚（LisaDeschamps）表示：“AviadoBio致力于为FTD-GRN提出一种创新的基因治疗方法，这一时刻标志着FTD社区和我们公司的一个重要里程碑。”。“我们感谢研究AVB-101的ASPIRE-FTD临床研究人员的奉献精神，并非常感谢参与临床试验的家庭提供新的治疗选择，这些选择可能会改变FTD-GRN家庭的未来。”。

FTD is a devastating form of early-onset dementia that typically leads to death within three to 10 years from diagnosis.1,2 People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.3 It is a leading cause of dementia in people under the age of 65 and is often misdiagnosed.4 People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein.

FTD是一种破坏性的早发性痴呆，通常会在诊断后的三到十年内导致死亡[1,2]。FTD患者通常会出现人格改变，行为障碍，语言丧失，冷漠和活动能力下降[3]。这是65岁以下人群痴呆的主要原因，常被误诊[4]。患有致病性GRN突变的FTD患者产生的颗粒蛋白前体数量减少。

AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain..

AVB-101是一种潜在的一次性疗法，旨在通过提供GRN基因的功能拷贝来阻止疾病进展，从而将适当的颗粒蛋白前体水平恢复到大脑的受影响区域。。

“There is a critical need for new treatments for people living with FTD-GRN. The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families who don’t currently have any available treatment options for this devastating and progressive disease,” said David Cooper, M.D., Chief Medical Officer.

首席医疗官大卫·库珀（DavidCooper）医学博士说：“FTD-GRN患者迫切需要新的治疗方法。由于缺乏有效的疾病缓解治疗方法，对于目前尚无任何治疗这种毁灭性和进行性疾病的选择的患者及其家属来说，这种诊断尤其困难。”。

“Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101’s potential, which has already shown promise in preclinical studies.”.

“在ASPIRE-FTD中治疗第一名患者标志着在理解AVB-101潜力方面迈出了重要的一步，这在临床前研究中已经显示出前景。”。

AVB-101 is delivered using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families.

AVB-101使用微创立体定向神经外科手术直接输送到称为丘脑的大脑部分。丘脑与大脑的其他部分有着广泛的联系，包括额叶和颞叶，额叶和颞叶在FTD以及影响患者及其家人的症状中起着至关重要的作用。

This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required and thereby limiting any potential systemic exposure..

这种靶向递送方法旨在安全有效地穿过血脑屏障，直接向大脑提供靶向治疗，以恢复最需要的额叶和颞叶皮层中的颗粒蛋白前体水平，同时最大程度地减少所需剂量，从而限制任何潜在的全身暴露。。

The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in Europe currently performing MRI-guided infusions of gene therapies. “Our team is excited to participate in this important scientific research and to perform the first intrathalamic administration of AVB-101 in the ASPIRE-FTD global trial,” said Prof.

Mazowiecki Szpital Bródnowski医院的介入神经治疗中心（INC）是欧洲目前第一个也是唯一一个在MRI引导下进行基因治疗输注的中心。“我们的团队很高兴参与这项重要的科学研究，并在ASPIRE-FTD全球试验中首次进行丘脑内AVB-101给药，”教授说。

Mirosław Ząbek, M.D., Ph.D., the chairperson of the department of neurosurgery at INC..

Mirosław Ząbek，医学博士，博士，INC.神经外科系主任。。

“As a neurologist and founder of the Neuro-Care Clinic, I’ve seen the difficult struggles of my patients with dementia and their families,” said Gabriela Klodowska, M.D., Ph.D. “It means a lot to our patients with FTD to bring this type of study offering a potential one-time treatment for patients with a GRN mutation to Poland and to be the first to enroll patients in this global study.”.

“作为一名神经科医生和神经护理诊所的创始人，我看到了我的痴呆症患者及其家人的艰难挣扎，”医学博士加布里埃拉·克洛多夫斯卡（GabrielaKlodowska）说。“将这种类型的研究带给波兰，为GRN突变患者提供潜在的一次性治疗，并成为第一个在这项全球研究中招募患者的人，对我们的FTD患者意义重大。”。

In November 2023, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to AVB-101. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. This designation is intended to bring new medicines to patients earlier.

2023年11月，美国食品和药物管理局（FDA）授予AVB-101快速通道名称。快速通道是一个旨在促进开发和加快新药审查的过程，以治疗严重疾病并满足未满足的医疗需求。该名称旨在尽早为患者带来新药。

It has also received orphan drug designation by both the FDA and European Commission..

它还被FDA和欧盟委员会指定为孤儿药。。

About ASPIRE-FTD

关于ASPIRE-FTD

ASPIRE-FTD is an open-label, multi-center, Phase 1/2 dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, patients will receive a single administration of AVB-101 delivered as a one-time infusion into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States..

ASPIRE-FTD是一项开放标签，多中心，1/2期剂量递增研究，旨在评估AVB-101在FTD-GRN患者中的安全性和初步疗效。在该研究中，患者将在欧洲和美国的专家神经外科中心通过立体定向神经外科手术一次性输注AVB-101。。

About AVB-101

关于AVB-101

An investigational gene therapy, AVB-101 contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 will be delivered as a one-time infusion directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center..

作为一种研究性基因疗法，AVB-101包含正确（非突变）版本的GRN基因。它旨在恢复大脑中颗粒蛋白前体的水平，可能会减缓或阻止FTD-GRN的进展。AVB-101将通过微创外科手术直接一次性输注到大脑中，由专业神经外科中心的研究神经外科医生进行。。

About Frontotemporal Dementia (FTD) and FTD with GRN Mutations (FTD-GRN)

关于额颞叶痴呆（FTD）和具有GRN突变的FTD（FTD-GRN）

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.1,2 People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.3

FTD是一种破坏性的早发性痴呆，通常在症状发作后7至13年内和诊断后3至10年内导致死亡。1,2 FTD患者通常会经历人格改变，行为障碍，语言丧失，冷漠和行动不便

FTD is a leading cause of dementia in people under the age of 654 with an estimated prevalence at any one time of up to 4.6 cases per 1,000 people.5 FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age.6,7 Given the early onset, FTD can have a substantially greater impact on work, family, and finances than Alzheimer’s disease.8 Genetic FTD cases account for about one-third of cases and are associated with autosomal dominant mutations in three genes, including the GRN (progranulin) gene.9 Approximately 11,000 people in the U.S.

FTD是654岁以下人群痴呆的主要原因，估计每1000人中有4.6例患病率。FTD通常比阿尔茨海默氏病发病年龄小，大多数FTD病例发生在45至68岁之间。6,7鉴于FTD发病较早，FTD对工作，家庭和财务的影响可能比阿尔茨海默氏病大得多。8遗传性FTD病例约占病例的三分之一，与包括GRN（颗粒蛋白原）基因在内的三个基因的常染色体显性突变有关。9美国约有11000人。

and EU5 are living with FTD-GRN with approximately 2,200 new cases per year.1,10 Some FTD cases may be misidentified, and diagnostic delay is common. As disease education, genetics knowledge, and research and treatment options grow, these numbers are expected to increase..

EU5患有FTD-GRN，每年约有2200例新病例。1,10一些FTD病例可能被错误识别，诊断延迟很常见。随着疾病教育、遗传学知识以及研究和治疗选择的增长，这些数字预计会增加。。

About AviadoBio

关于Aviadobio

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly to overcome the challenges of delivering the right drug to the right place.

在AviadoBio，我们正在不懈地寻求治疗方法，将开创性的科学和精确递送转化为改变生活的药物，用于额颞叶痴呆（FTD）和肌萎缩侧索硬化症（ALS）患者。随着我们对大脑的深入了解以及一套专有的基因治疗平台和传递技术，AviadoBio正在不懈地努力克服将正确的药物传递到正确的地方的挑战。

Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients..

其创新的神经解剖学主导的方法旨在最大限度地发挥基因治疗的治疗潜力，以阻止或潜在地逆转神经退行性疾病。AviadoBio成立于伦敦国王学院和英国痴呆症研究所的开创性研究，拥有一支具有广泛基因治疗开发、交付和商业化经验的领导团队，为公司成功为患者带来变革性药物奠定了独特的地位。