Conference Agenda

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Wednesday, Nov. 13

Opening Keynote: State of gene therapy in ophthalmology
9 – 10:15am 

Eric Pierce, MD, PhD, will kick off the conference with an overview of the current state of gene therapy research for ophthalmic diseases and disorders. He will discuss the latest advancements, key challenges and future directions in the field. This opening session sets the stage for the conference, providing valuable context and highlighting the significance of ongoing research and clinical trials in advancing ophthalmic gene therapy. 

Presentations

• Welcome and introduction to the conference — Priya Chaudhary, PhD
• State of Gene Therapy for Ophthalmic Clinical Care — Eric Pierce, MD, PhD, FARVO

Delivery systems including gene transfer strategies, viral vectors and their manufacturing
10:30 – 11:45am

Explore the critical aspects of delivery systems in gene therapy, focusing on gene transfer strategies, viral vectors and their manufacturing processes. This session will delve into the various methods used to deliver genetic material to target cells, highlighting the advantages and challenges of each approach. Attendees will gain an in-depth understanding of the different types of viral vectors (e.g., AAV, lentivirus and adenovirus) and their specific applications in ophthalmic gene therapy. The session will cover also the manufacturing processes required to produce high-quality, safe and effective viral vectors, addressing issues such as scalability, regulatory compliance and quality control.

Presentations

• Advancing Intravitreal Gene Therapy: Developing Novel AAV Capsids Through the scAAVengr Platform — Laura Campello, PhD
• Developing lipid nanoparticle-mediated gene editing therapeutics for inherited retinal degeneration — Renee Christine Ryals, PhD
• Intravitreal, subretinal, and suprachoroidal delivery: Common vectors, risks, and benefits for each approach — Thomas A. Ciulla, MD, MBA, FASRS
• Optogenetics: A mutation-agnostic approach — Samarendra Mohanty, MSc, PhD

Acceleration of AAV gene therapy for ocular diseases: Improving delivery and quality
Noon – 1:15pm

Attendees will be introduced to key concepts, themes and issues involved in AAV gene therapy and ocular disease treatment, providing a solid foundational understanding of these research areas. You will delve deeper into the practical applications and challenges of AAV gene therapy plus explore advanced techniques, lessons learned and new methodologies to enhance your research and operational practices.

Presentations

• Development and implementation of the recombinant baculovirus-Sf9 Cell (rBV-Sf9) platform technology in AAV Vector GMP production for human ocular gene therapy — Shengjiang Shawn Liu, PhD
• Acceleration of AAV Gene Therapy for Ocular Diseases: Enhancing Delivery & Quality — Amicia Elliott, PhD
• The Accelerating Medicines Partnerships® (AMP®) Bespoke Gene Therapy Consortium (BGTC) Regulatory Playbook — Brad Garrison, MBA, PMP
• Optimized Pre-Clinical Study Designs for Regulatory Compliance in AAV-Based Ophthalmic Drug Development — Julia Carrier, PhD
• Development of genome-modified AAV vectors for enhanced transgene expression — Arun Srivastava, PhD

Potential strategies to improve the outcome of gene therapy in the clinical application
1:30 – 2:45pm 

This session introduces three approaches that could enhance the safety and efficacy of voretigene neparvovec gene therapy in clinical practice. You will cover new paradigms in retinal rehabilitation after gene therapy, explore the potential of additional corticosteroid treatment to reduce inflammatory side effects in children following voretigene neparvovec therapy, and examine the possibility of central nervous system rehabilitation improving the outcomes of retinal therapies.

Presentations

• Retinal rehabilitation after gene therapy - new paradigms — Katarina Stingl, MD
• Can additional corticosteroid treatment reduce inflammatory side effects in children after voretigene neparvovec? — Maximilian Gerhardt
• Can central nervous rehabilitation improve the outcome of retinal therapies? — Pierre Safar, MD

Thursday, Nov. 14

From lab to life: A patient’s journey through gene therapy
9 – 10:15am

Delve into the transformative potential of gene therapy in ophthalmology with Paul Yang, MD, PhD, and one of his patients as they recount their shared journey through gene therapy. Discover firsthand the challenges, triumphs and hope that come with pioneering treatments for vision restoration.

Genetic counselor Kari Branham will present next on best practices for discussing gene therapy options and clinical trials with patients. She will provide insights on how to effectively communicate complex genetic information, manage patient expectations and provide compassionate guidance throughout their decision-making process. 

Presentations

• Talking with patients about gene therapies — Kari E. Branham, MS
• Interview: Doctors and patients with Paul Yang, MD, PhD

Advances in gene therapies and clinical endpoints for Leber Congenital Amaurosis (LCA)
10:30 – 11:45am 

This session will include a comprehensive description of development of gene therapy approaches to treat the patients affected by LCA. After the approval of voretigene neparvovec, there remain several LCA IRDs which are unaddressed (i.e., not supported by pre-clinical investigations that would lead to trials). We will cover progress in clinical trials for other forms of LCA and endpoint development. 

Presentations

• Genomic medicine at the forefront of gene therapy for LCA — Bikash R. Pattnaik, PhD, MPhil, FARVO
• Full-field Stimulus Testing (FST) as a clinical endpoint in severe vision loss — Artur V. Cideciyan, PhD, FARVO
• Twelve-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) — Dan Y. Yoon, MD
• AAV8-hLCA5 subretinal delivery to the macula in lca5-associated LCA: Preliminary safety and efficacy results of an ongoing Phase I/ II gene therapy trial — Tomas S. Aleman, MD, FARVO

Clinical trial design and endpoints
Noon – 1:15pm 

Get an understanding of the critical components involved in trial design, including patient selection, endpoint determination, regulatory considerations and ethical challenges. Attendees will gain insights into the latest methodologies and best practices for developing robust clinical trials that can lead to successful regulatory approvals and improved patient outcomes. This session is ideal for researchers, clinicians and industry professionals seeking to enhance their knowledge and skills in ophthalmic gene therapy trial design.

Presentations

• Ocular gene therapy trials: Patient perspectives — Ian M. MacDonald, MD, CM, FARVO
• Lessons learned from Academia — Rachel M. Huckfeldt, MD, PhD
• Lessons learned from Industry — Daniel C. Chung, DO, MA

How to get your gene therapy idea off the ground?
1:30 – 2:45pm

This is an opportunity for attendees to get feedback about their specific research projects and ideas. The session will have an informal structure where everyone can learn from each other. Come prepared to ask your questions and share your insights.  

Panelists

• Shengjiang Shawn Liu, PhD
• Ashwath Jayagopal, PhD, MBA, FARVO