Nanoscope Therapeutics将出席第21届年度高级治疗周会议-动脉网

Nanoscope Therapeutics to Present at the 21st Annual Advanced Therapies Week Conference

CISION 等信源发布 2025-01-20 23:30

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DALLAS

达拉斯

Jan. 20, 2025

2025年1月20日

/PRNewswire/ --

/美通社/ --

Nanoscope Therapeutics Inc.

Nanoscope Therapeutics Inc。

, a biotechnology company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, today announced several presentations by company leadership at the

，一家生物技术公司，致力于通过开发和商业化视网膜退行性疾病的新型基因疗法，在现实世界中恢复盲人患者的视力，今天宣布公司领导层在

圣

Annual Advanced Therapies Week Conference

年度高级治疗周会议

, taking place from January 20-23, 2025 at the Kay Bailey Hutchinson Convention Center in

，于2025年1月20日至23日在凯贝利哈钦森会议中心举行

Dallas, Texas

德克萨斯州达拉斯

. Details for the presentations are as follows:

。演示的详细信息如下：

Title:

标题：

Navigating the Challenges of Gene Therapies for Rare Diseases

应对罕见疾病基因治疗的挑战

Session Title:

会议标题：

Gene Therapy for Rare Disease

罕见疾病的基因治疗

Session Date:

会议日期：

January 21, 2025

2025年1月21日

Session Time:

会话时间：

12:15 p.m. C.T.

12：下午15点C.T。

Presenter:

演示者：

Samarendra Mohanty

萨马伦德拉莫汉塔

, PhD, President & Chief Scientific Officer

，博士，总裁兼首席科学官

Dr. Mohanty will discuss key challenges to modern gene therapy programs for rare disease indications. These will include the general lack of suitable animal models, established clinical endpoints, and validated analytics and manufacturing processes, which in most cases need to be implemented by the gene therapy developer..

莫汉蒂博士将讨论针对罕见疾病适应症的现代基因治疗计划面临的关键挑战。这些将包括普遍缺乏合适的动物模型，建立的临床终点以及经过验证的分析和制造过程，在大多数情况下，这些都需要由基因治疗开发人员实施。。

Title:

标题：

Partnering for Commercialization, a CMC Prospective

商业化合作，CMC前景

Session Title:

会议标题：

Partnering for Gene Therapy Success

基因治疗成功的合作

Session Date:

会议日期：

January 21, 2025

2025年1月21日

Session Time:

会话时间：

4:30 p.m. C.T.

4：下午30点C.T。

Presenter:

演示者：

Raymond Kaczmarek, Chief Technical and Manufacturing Officer

Raymond Kaczmarek，首席技术和制造官

During his presentation, Mr. Kaczmarek will highlight several key internal and external considerations paramount to ensuring a successful CDMO relationship and overall manufacturing program for gene therapy programs.

在演讲中，卡兹马雷克先生将重点介绍几个关键的内部和外部考虑因素，这些因素对于确保成功的CDMO关系和基因治疗计划的整体制造计划至关重要。

Session Title:

会议标题：

International Convergence and Regulatory Alignment for CGT

CGT的国际趋同和监管调整

Session Date:

会议日期：

January 22, 2025

2025年1月22日

Session Time:

会话时间：

11:00 a.m. C.T.

11： C.T.上午00点。

Chair:

主席：

Khandan Baradaran

, PhD, SVP Regulatory and Quality

，博士，监管和质量高级副总裁

Dr. Baradaran will host a fireside chat with Dr.

Baradaran博士将与Dr。

Nicole Verdun

妮可弗登

, Super Office Director of the Office of Therapeutic Products, CBER, discussing how gene and cell therapy developers can forge productive interactions with regulators to ensure an efficient path toward approval.

，CBER治疗产品办公室超级办公室主任，讨论了基因和细胞治疗开发人员如何与监管机构建立有效的互动关系，以确保获得批准的有效途径。

Title:

标题：

Optical Approaches to Non-Viral Redosable Gene Therapies

非病毒可氧化基因治疗的光学方法

Session Title:

会议标题：

Are Redosable Gene Therapies on the Horizon?

可还原基因疗法即将出现吗？

Session Date:

会议日期：

January 22, 2025

2025年1月22日

Session Time:

会话时间：

11:00 a.m. C.T.

11： C.T.上午00点。

Presenter:

演示者：

Subrata Batabyal

Subrata 巴塔比亚尔

, PhD, Director, Non-Clinical Development

，博士，非临床开发总监

During his presentation, Dr. Batabyal will discuss the dosing limitations of AAV gene therapies, and how Nanoscope's non-viral, optical dosing technologies can overcome this limitation by eliminating concerns related to immunogenicity.

Batabyal博士在演讲中将讨论AAV基因疗法的剂量限制，以及Nanoscope的非病毒光学剂量技术如何通过消除与免疫原性相关的担忧来克服这一限制。

Title:

标题：

First-Line Gene Therapies for Inherited Blindness

遗传性失明的一线基因疗法

Session Title:

会议标题：

Innovation Series Company Presentations

创新系列公司演示

Session Date:

会议日期：

January 22, 2025

2025年1月22日

Session Time:

会话时间：

4:00 p.m. C.T.

4：下午00点C.T。

Presenter:

演示者：

Sulagna Bhattacharya

苏拉尼亚 Bhattacharya

, CEO

，首席执行官

During her talk, Ms. Bhattacharya will provide a corporate update on Nanoscope's recent and upcoming progress across pipeline programs. She will specifically cover Nanoscope's lead asset MCO-010, which is slated for BLA submission, as well as the advancement of Nanoscope's Stargardt program to a registrational Phase 3 trial. .

在演讲期间，Bhattacharya女士将向公司介绍Nanoscope最近和即将在管道项目方面取得的进展。她将专门介绍Nanoscope的主要资产MCO-010，该资产计划提交给BLA，以及Nanoscope的Stargardt计划进入注册3期试验的进展。。

Title:

标题：

Ensuring Flexibility and Scalability in AAV Gene Therapy Manufacturing

确保AAV基因治疗制造的灵活性和可扩展性

Session Title:

会议标题：

Bioprocess Workflows & Analytics for Flexibility and Scalability

生物过程工作流程和分析具有灵活性和可扩展性

Session Date:

会议日期：

January 22, 2025

2025年1月22日

Session Time:

会话时间：

5:00 p.m. C.T.

5：下午00点C.T。

Presenter:

演示者：

Victor Adeniyi

维克多阿德尼

, Senior Director, CMC

，CMC高级总监

During his presentation, Adeniyi will provide an overview of the landscape of analytical and scalability challenges in gene therapy, and discuss the value of designing and incorporating flexibility to address these challenges over time.

在他的演讲中，Adeniyi将概述基因治疗中分析和可扩展性挑战的前景，并讨论设计和整合灵活性以应对这些挑战的价值。

About Nanoscope Therapeutics Inc.

关于Nanoscope Therapeutics Inc。

Nanoscope Therapeutics is developing gene-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases, for which no cure currently exists. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (.

Nanoscope Therapeutics正在为数百万视网膜退行性疾病致盲的患者开发基因不可知的视力恢复光遗传学疗法，目前尚无治愈方法。在RESTORE 2b期多中心，随机，双盲，假对照临床试验的RP（。

NCT04945772

第04945772节

), the company announced its plan to initiate a

)，该公司宣布计划启动

BLA submission for MCO-010 to treat RP

BLA提交MCO-010治疗RP

in Q1 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (

2025年第一季度。该公司已经完成了Stargardt患者MCO-010治疗的第二阶段STARLIGHT试验(

NCT05417126

编号：NCT05417126

) and plans to initiate a

)并计划启动

Phase 3 registrational trial

第三阶段注册试验

in Q1 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical programs include an IND-ready non-viral laser-delivered MCO-020 asset for GA, as well as an AAV asset for Leber Congenital Amaurosis, in IND-enabling studies.

2025年第一季度。MCO-010已获得RP和Stargardt的FDA快速通道指定和FDA孤儿药指定。在IND启用研究中，临床前计划包括用于GA的IND就绪非病毒激光交付MCO-020资产，以及用于Leber先天性黑蒙的AAV资产。

Investor Contact:

投资者联系人：

Argot Partners

阿戈特合作伙伴

212-600-1902

PR@nanostherapeutics.com

SOURCE Nanoscope Therapeutics

源Nanoscope Therapeutics

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