#USA #gene therapy #Nanoscope Therapeutics #Dallas, Texas #Optogenetic Therapies

Nanoscope Therapeutics at the 21st Annual Advanced Therapies Week Conference

Nanoscope Therapeutics Inc., a pioneering biotechnology company dedicated to restoring vision for blind individuals, is set to make a significant impact at the upcoming 21st Annual Advanced Therapies Week Conference. This notable event will transpire from January 20-23, 2025, at the Kay Bailey Hutchison Convention Center in Dallas, Texas. The conference will feature a series of presentations by key figures from Nanoscope, highlighting the company's commitment to advancing gene therapies for retinal degenerative diseases.

Presentations Overview

The conference agenda includes the following key sessions featuring Nanoscope's leadership:

1. Navigating the Challenges of Gene Therapies for Rare Diseases

• - Session Date: January 21, 2025
• - Time: 12:15 PM C.T.
• - Presenter: Dr. Samarendra Mohanty, President & Chief Scientific Officer

In this session, Dr. Mohanty will delve into the hurdles faced by contemporary gene therapy programs targeting rare diseases. He will address issues such as the absence of adequate animal models, the need for established clinical endpoints, and the necessity for validated analytics and manufacturing processes essential for successful gene therapy development.

2. Partnering for Commercialization: A CMC Perspective

• - Session Date: January 21, 2025
• - Time: 4:30 PM C.T.
• - Presenter: Mr. Raymond Kaczmarek, Chief Technical and Manufacturing Officer

Mr. Kaczmarek will spotlight important internal and external factors affecting the success of partnerships with Contract Development and Manufacturing Organizations (CDMOs) that are vital for gene therapy manufacturing programs.

3. International Convergence and Regulatory Alignment for CGT

• - Session Date: January 22, 2025
• - Time: 11:00 AM C.T.
• - Chair: Dr. Khandan Baradaran, SVP Regulatory and Quality

In a conversational format, Dr. Baradaran will engage Dr. Nicole Verdun from the Office of Therapeutic Products in a dialogue about how gene and cell therapy developers can effectively collaborate with regulatory agencies to streamline the approval process.

4. Optical Approaches to Non-Viral Redosable Gene Therapies

• - Session Date: January 22, 2025
• - Time: 11:00 AM C.T.
• - Presenter: Dr. Subrata Batabyal, Director of Non-Clinical Development

Dr. Batabyal will explore the limitations present in AAV gene therapies and highlight how Nanoscope's pioneering optical dosing technologies can mitigate these constraints by addressing immunogenicity concerns.

5. First-Line Gene Therapies for Inherited Blindness

• - Session Date: January 22, 2025
• - Time: 4:00 PM C.T.
• - Presenter: Ms. Sulagna Bhattacharya, CEO

During her presentation, Ms. Bhattacharya will update attendees on Nanoscope's recent achievements and future milestones, including the anticipated Biologics License Application (BLA) submission for the flagship product MCO-010, and the progression of the Stargardt program to a Phase 3 trial.

6. Ensuring Flexibility and Scalability in AAV Gene Therapy Manufacturing

• - Session Date: January 22, 2025
• - Time: 5:00 PM C.T.
• - Presenter: Mr. Victor Adeniyi, Senior Director, CMC

Mr. Adeniyi will provide insights into the analytical and scalability challenges faced in gene therapy, emphasizing the importance of incorporating flexibility within the manufacturing processes to adapt to evolving challenges over time.

About Nanoscope Therapeutics

Founded with the mission to create life-changing therapies, Nanoscope Therapeutics is at the forefront of developing sight-restoring optogenetic treatments for millions affected by retinal degenerative conditions. Notably, the company has announced plans to submit a BLA for its lead asset, MCO-010, targeting Retinitis Pigmentosa (RP) in the first quarter of 2025, following a successful Phase 2b trial. Nanoscope is also moving forward with a Phase 3 trial for Stargardt disease, underscoring its commitment to innovation and efficacy in addressing blindness caused by genetic disorders.

With its innovative approaches and focused expertise, Nanoscope Therapeutics is undeniably poised to make substantial contributions to the field of gene therapy and restore vision for those in dire need.