生物技术公司Hubble Therapeutics完成730美元A轮融资，用于开发LCA16的基因疗法-动脉网

Hubble Therapeutics closes Series A Financing to develop gene therapy cure for LCA16

CISION 等信源发布 2025-03-25 22:36



可切换为仅中文







HANOVER, N.H.

新罕布什尔州，汉诺威

，

March 25, 2025

2025年3月25日

/PRNewswire/ --

Hubble Therapeutics LLC

哈勃治疗学有限责任公司

a patient-focused biotechnology company developing a gene therapy to cure pediatric blindness, announces the successful closing of a

一家专注于患者的生物技术公司，正在开发一种基因疗法以治愈儿童失明，宣布成功结束了一项

$7.3 million

730万美元

Series A funding round, which will advance its lead candidate, HUB-101, into human clinical trials.

A轮融资，这将推动其先导候选药物HUB-101进入人体临床试验。

Hubble Therapeutics' gene therapy, HUB-101, targets two rare and devastating eye disorders: Leber Congenital Amaurosis (LCA16) and Snowflake Vitreoretinal Degeneration (SVD). This innovative treatment, developed by Dr.

霍布尔治疗学公司的基因疗法 HUB-101 针对两种罕见且严重的眼部疾病：莱伯先天性黑蒙症（LCA16）和雪花状玻璃体视网膜变性（SVD）。这项创新疗法由博士开发。

Bikash Pattnaik

比卡什·帕特奈克

at the

在

University of Wisconsin-Madison School of Medicine

威斯康星大学麦迪逊分校医学院

and Public Health and licensed from the Wisconsin Alumni Research Foundation (WARF), aims to address the root cause of these conditions. LCA16 and SVD are severe, early-onset retinal dystrophies caused by mutations in the KCNJ13 gene, which is crucial for the function of a vital ion channel in the retina's pigment epithelium cells.

公共卫生和威斯康星校友研究基金会（WARF）许可，旨在解决这些疾病的根源。LCA16 和 SVD 是由 KCNJ13 基因突变引起的严重早发性视网膜营养不良，该基因对视网膜色素上皮细胞中重要的离子通道功能至关重要。

These disorders typically manifest in children before the age of five, leading to progressive retinal degeneration over the following two decades and ultimately resulting in blindness..

这些疾病通常在五岁前的儿童中显现，随后的二十年间会导致视网膜退化，并最终导致失明。

'For over the past 20 years, my lab has developed

“在过去的20多年里，我的实验室开发了

in vitro

体外

and

和

in vivo

体内

models of ion channelopathy to develop curative gene therapies for several devastating pediatric blindness diseases,' Dr.

离子通道病模型，以开发针对几种毁灭性儿童盲病的治愈性基因疗法，”

Bikash Pattnaik

比卡什·帕特奈克

, professor in the Departments of Pediatrics, Ophthalmology and Visual Science at the University of Wisconsin–Madison, Daniel M. Albert Chair McPherson Eye Research Institute and scientific co-founder of Hubble Therapeutics LLC, 'The development of the HUB-101 gene therapy for LCA16 represents not just the scientific breakthrough, but hope for patients and families affected by such devastating conditions caused by defective ion channels.

威斯康星大学麦迪逊分校儿科、眼科和视觉科学系教授，丹尼尔·M·阿尔伯特主席麦克弗森眼科研究所和Hubble Therapeutics LLC的科学共同创始人表示：“HUB-101基因疗法针对LCA16的开发不仅代表了科学突破，还为受缺陷离子通道影响的患者及其家庭带来了希望。"

As we push these therapeutic boundaries, we empower a future where innovative ion channel therapies in our lab address genetic diseases at their core.'.

随着我们突破这些治疗界限，我们正在赋能一个未来，在这个未来中，我们实验室中的创新离子通道疗法能够从根本上解决遗传疾病。

To date, HUB-101 has received Rare Pediatric Disease Designation and Orphan Disease Designation from the FDA for this program. It has partnered with Andelyn Biosciences in

迄今为止，HUB-101 已经获得了 FDA 对该计划的罕见儿科疾病认定和孤儿病认定。它已与 Andelyn Biosciences 合作。

Columbus, Ohio

俄亥俄州哥伦布市

, and Virscio in

，以及Virscio在

New Haven, Connecticut

康涅狄格州纽黑文市

, to develop, manufacture, and conduct IND-enabling studies. Hubble Therapeutics expects to enter the clinic to conduct Phase I / II trials in 2026 to demonstrate the safety and efficacy of HUB-101.

，进行开发、制造和开展新药临床试验（IND）所需的研究。Hubble Therapeutics 预计将于 2026 年进入临床阶段，进行 I/II 期试验，以证明 HUB-101 的安全性和有效性。

'This groundbreaking research aims to restore vision in patients affected by a rare eye disease that ultimately results in complete blindness, with WARF's investment in Hubble Therapeutics as the Series A co-lead investor, we are thrilled to continue our support for the development of a disease-modifying treatment,' says .

“这项开创性的研究旨在恢复受一种罕见眼疾影响的患者的视力，这种疾病最终会导致完全失明。WARF作为Hubble Therapeutics的A轮融资共同领投方，我们很高兴能够继续支持这种疾病改善性治疗的开发，”他表示。

Greg Keenan

格雷格·基南

, Senior Director of WARF Ventures & Accelerator and Hubble Therapeutics Board of Director. 'The collaboration between Hubble Therapeutics and the pioneering research of UW-Madison inventor, Dr.

WARF风险投资与加速器高级总监，哈勃治疗公司董事会成员。 “哈勃治疗公司与威斯康星大学麦迪逊分校发明家、博士的开创性研究之间的合作。

Bikash Pattnaik

比卡什·帕塔奈克

, exemplifies the transformative potential of academic-industry partnerships and we are honored to continue our support in advancing this critical research alongside the dedicated team at Hubble Therapeutics.'

，体现了学术与产业合作的变革潜力，我们很荣幸能够继续支持这一关键研究，并与哈勃治疗学公司的专业团队携手共进。'

'We are grateful for our investors, leading scientists, industry experts, best-in-class partners, patient families, and the

“我们感谢我们的投资者、领先的科学家、行业专家、一流的合作伙伴、患者家庭，以及

University of Wisconsin-Madison

威斯康星大学麦迪逊分校

who share our commitment to realizing the promise of improving vision for patients suffering from LCA16 and SVD,' said

“他们与我们一样，致力于实现改善LCA16和SVD患者视力的承诺，”他说。

Jeff Sabados

杰夫·萨巴多斯

, Founder and President of Hubble Therapeutics LLC. Sabados continued, 'with our new financing and the establishment of key partnerships, we hope to scale up HUB-101 manufacturing and conduct additional IND-enabling studies to enter the clinic within the next two years to realize our steadfast, unwavering promise to support families throughout the world impacted by this devastating genetic disease.'.

Hubble Therapeutics LLC创始人兼总裁。萨巴多斯继续说道：“通过我们的新融资和关键合作伙伴关系的建立，我们希望能够扩大HUB-101的生产规模，并进行更多的IND支持性研究，以便在未来两年内进入临床阶段，履行我们坚定不变的承诺，支持全球受这种毁灭性遗传疾病影响的家庭。”

About Hubble Therapeutics, LLC

关于哈勃治疗学有限责任公司

Hubble Therapeutics LLC ('HubbleTx') is a patient-focused biotechnology company developing research from Professor

哈勃治疗学有限责任公司（“HubbleTx”）是一家以患者为中心的生物技术公司，致力于开发来自教授的研究成果。

Bikash Pattnaik's

比卡什·帕特奈克的

lab at the

实验室在

University of Wisconsin-Madison

威斯康星大学麦迪逊分校

. Our primary focus is on developing novel gene therapy for Leber Congenital Amaurosis Type 16 (LCA16) and Snowflake Vitreoretinal Degeneration (SVD), two debilitating retinal diseases that lead to severe vision loss in adult and pediatric patients. Our mission is to restore vision and improve the quality of life for affected individuals and their families by harnessing the power of gene delivery technology to target the underlying genetic mutation responsible for the disease.

我们的主要重点是开发针对Leber先天性黑蒙16型（LCA16）和雪花状玻璃体视网膜变性（SVD）的新型基因疗法，这两种使人衰弱的视网膜疾病会导致成人和儿童患者严重视力丧失。我们的使命是通过利用基因递送技术的力量，靶向导致疾病的潜在基因突变，从而恢复患者的视力并改善受影响个人及其家庭的生活质量。

Our team of leading scientists, industry experts, and best-in-class partners are committed to rigorous research and clinical development, ensuring that our therapies are safe, effective, and accessible. HubbleTx strives to bring hope to those affected by this condition through collaboration and innovation.

我们由顶尖科学家、行业专家和一流的合作伙伴组成的团队致力于严格的研究和临床开发，确保我们的疗法安全、有效且可及。HubbleTx 力求通过合作与创新，为受此疾病影响的人们带来希望。

Together, we aim to illuminate the path to a brighter future for patients battling LCA16 and SVD..

我们一起致力于为LCA16和SVD患者照亮通往更光明未来的道路。

For Hubble media inquiries, please contact:

对于哈勃媒体咨询，请联系：

jsabados@hubbletx.com

Logo -

标志 -

https://mma.prnewswire.com/media/2644246/Hubble_Tx_Logo.jpg

WANT YOUR COMPANY'S NEWS

希望贵公司的新闻

FEATURED ON PRNEWSWIRE.COM?