8:00 am Preconference Virtual Networking

8:50 am Chair’s Opening Remarks

9:00 am Slow Progression of Dry AMD: Small Molecule Approaches

  • Leonide Saad President & CEO , Alkeus Pharmaceuticals, Inc.

Synopsis

• Dry AMD treatment with minerals, antioxidants, and other natural
compounds.
• Current challenges and therapeutic approaches for the treatment of
Geographic Atrophy: drug delivery, animal models, study designs
• Visual cycle: to slow or not to slow? Use of ALK-001 as vitamin A
replacement therapy for the treatment of dry-AMD or Geographic
Atrophy

Targeting Gene Therapy at Retinal Diseases

9:20 am “AAV Engineering for Retina Diseases in Mouse, Dog, and NHP”

  • David Schaffer Co-Founder, Chief Scientific Officer & Co-Chairman of The Board of Directors , 4D Molecular Therapeutics

9:40 am Live Presenter Q&A

  • Leonide Saad President & CEO , Alkeus Pharmaceuticals, Inc.
  • David Schaffer Co-Founder, Chief Scientific Officer & Co-Chairman of The Board of Directors , 4D Molecular Therapeutics

9:50 am Virtual Networking Break

Optimizing Therapeutics Targeting Oxidative Stress in Dry AMD

10:50 am Photobiomodulation as a Novel Treatment for Dry AMD Patients

Synopsis

  • LumiThera, Inc. is the leading company developing Photobiomodulation (PBM) for the treatment of Dry AMD
  • The Valeda Light Delivery System is the only EU-approved treatment for dry AMD and has shown to improve clinical outcomes such as visual acuity and contrast sensitivity and reduce drusen pathology
  • PBM is a mitochondrial-targeted approach that addresses underlying disease processes that contribute to age-related degenerative eye disease

11:10 am Targeting Mitochondrial Oxidative Stress: Getting Drugs to the Retinal Tissue Mitochondria in Dry AMD

  • Karl G. Csaky T. Boone Pickens Director, Clinical Center of Innovation for AMD, Chief Executive and Medical Officer , Retina Foundation of the Southwest

Synopsis

• Evidence to support mitochondrial damage in AMD
• Understanding the role of mitochondrial redox imbalance
• Importance of retinal mitochondrial pharmacokinetics as a critical step
in potential dry AMD therapeutics

11:30 am Targeting Iron Induced Oxidative Stress and BRB Permeability in Dry AMD

  • Joshua Dunaief Adele Niessen Professor of Ophthalmology Scheie Eye Institute, University of Pennsylvania

Synopsis

• Mechanisms of iron accumulation in AMD retinas, including BRB
compromise even in dry AMD
• Cases of hereditary iron overload that can cause maculopathy with
features of AMD
• Retina-protective effects of iron chelators in preclinicial models

11:50 am Live Presenter Q&A

  • Karl G. Csaky T. Boone Pickens Director, Clinical Center of Innovation for AMD, Chief Executive and Medical Officer , Retina Foundation of the Southwest
  • Joshua Dunaief Adele Niessen Professor of Ophthalmology Scheie Eye Institute, University of Pennsylvania
  • Clark Tedford Co-Founder, LumiThera

Slowing Down Vision Loss Through Retinal Prosthesis Technologies

12:00 pm Creating a World of Bionic Vision for Those Who Have Lost Their Sight

Synopsis

• Dry-AMD and Geographic Atrophy what is it, incidence, prevalence,
target market for retinal implantation
• Mechanism of action of novel retinal implant
• Clinical outcomes today

12:20 pm Live Presenter Q&A

12:25 pm Virtual Networking Break

Emerging Therapeutics for the Treatment of AMD

1:00 pm Targeting Innate Immune Pathways in Intermediate AMD

  • Qian Huang Head of Ocular Inflammation, Ophthalmology , Novartis

Synopsis

• Discuss novel innate immune pathway identification and validation

1:20 pm Antagonism of Retinal Binding Protein 4 as a Therapeutic Strategy for Dry AMD Treatment

Synopsis

10.20 Antagonism of Retinal Binding Protein 4 as a Therapeutic
Strategy for Dry AMD Treatment
• Pharmacological Inhibition of Lipofuscin Accumulation in the Retina as a
Therapeutic Strategy for Dry AMD Treatment
• The recent clinical findings of RBP4 plasma concentration and its
correlation with disease progression in dry AMD patients
• A non-retinoid antagonist of Retinal Binding Protein 4 as a potential
therapeutic target in dry AMD

1:40 pm Live Presenter Q&A

  • Irene Wang Chief Operating Officer , Belite Bio
  • Qian Huang Head of Ocular Inflammation, Ophthalmology , Novartis

1:50 pm Virtual Networking Break

2:30 pm Cell-based Therapies for Blinding Diseases

  • Dang Dao Director - Research , Astellas Pharma Inc

Synopsis

• Discuss the new approaches to cell therapy for retinal diseases

2:50 pm Advanced Cell-based Therapies for Macular Degeneration

  • Kapil Bharti Senior Investigator , National Institutes of Health

Synopsis

• An autologous replacement therapy for AMD using an RPE-tissue
developed from patient-specific induced pluripotent stem (iPS) cells.
• Confirmed the safety and efficacy of this replacement patch in animal
models as part of a Phase I Investigational New Drug (IND)-application
• This work has formed the basis for future RPE/photoreceptor and RPE/
choroid transplants

3:10 pm Live Presenter Q&A

  • Dang Dao Director - Research , Astellas Pharma Inc
  • Kapil Bharti Senior Investigator , National Institutes of Health

3:20 pm Virtual Networking Break

3:30 pm Panel Discussion: Developing, Commercializing & Delivering Dry AMD Therapeutics into the Hands of Physicians & Patients in Need

  • Leonide Saad President & CEO , Alkeus Pharmaceuticals, Inc.
  • Brian M. Culley Chief Executive Officer , Lineage Cell Therapeutics, Inc
  • Karl G. Csaky T. Boone Pickens Director, Clinical Center of Innovation for AMD, Chief Executive and Medical Officer , Retina Foundation of the Southwest
  • Pooya Hemami Healthcare & Supervisory Analyst , Edison Group

Synopsis

During this discussion we will summarise the learnings of the meeting by addressing:

Strengths
What are the strengths of existing dry AMD
therapeutic approaches?

Opportunities
What is the marketing opportunity versus unmet
patient clinical need that needs to be addressed by
emerging therapeutic approaches for dry AMD? As
we are working outside of an orphan disease state,
what will the cost of treatment look like and what will
the patient share of costs be?

 

 

5:00 pm Chair’s Closing Remarks

5:15 pm End of Conference