8:00 am Online Registration & Virtual Coffee Networking

8:50 am Chair’s Opening Remarks

OPTIMIZING VECTOR DESIGN FOR MORE EFFECTIVE TARGETING AND EXPRESSION OF GENE THERAPIES

9:00 am Beyond AAV: Realizing the Potential of Non-Viral Vectors

Synopsis

• Developing non-viral vectors to minimize immune response and re-dose as needed
• Effectively targeting cells using non-viral and minimally invasive delivery approaches
• Delivering larger genetic material to target cells
• Extending the benefits of gene therapy to more common diseases

9:30 am Discovering Novel Ways to Deliver Larger Genes to the Eye

  • Hemant Khanna Associate Professor, University of Massachusetts Medical School

Synopsis

• Delivering cargo to target cells using multiple AAV vectors
• Discussing recent data to show progress on delivery of larger genes to the eye
• Exploring the potential of AAV to deliver larger proteins compared to alternative approaches (e.g. non-viral)

10:00 am Panel Discussion: Discussing the Future of Gene Therapy Delivery

Synopsis

It is evident that effective vector design to target cells both more effectively and safely is a vital aspect of creating a successful gene therapy platform. Delivery to the eye specifically brings unique challenges, and therefore choosing the right vector to deliver the cargo will be central to any ophthalmic program. This panel discussion will address some of the most important considerations in
the vector space, including:
• Development of a delivery platform compared to individual designs for individual therapeutics
• Assessing the safety of viral vectors in the eye – immune response considerations
• Exploring the future of non-viral vectors and their likelihood of success for ophthalmic indications
• Specificity requirements depending on the disease being targeted and how this impacts vector choice

10:30 am Morning Refreshments & Virtual Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

EXPLORING THE LATEST DEVELOPMENTS IN DELIVERY OF GENE THERAPIES TO THE EYE

11:00 am Discussing the Latest Advances in Sub-Retinal Administration of Gene Therapies

Synopsis

• Understanding the impact on patients of sub-retinal delivery
• Exploring the latest technologies to increase effectiveness of sub-retinal delivery
• Investigating the targets for which sub-retinal delivery is the optimal administration route

11:30 am Intravitreal Delivery of Gene Therapy Vectors to Treat Retinal Disease

Synopsis

• Discussing progress of intravitreal delivery of gene therapies as a less invasive procedure for patients
• Understanding challenges of making intravitreal delivery commonplace for gene therapies
• Reviewing ADVM-022: an intravitreal gene therapy product for patients with nAMD

12:00 pm Discovering Suprachoroidal Delivery as a Potential Platform to Deliver Gene Therapies to the Eye

  • Leroy Muya Director, Development, Clearside Biomedical Inc.

Synopsis

• Realizing the potential of suprachoroidal delivery to minimize impact on patients
• Discussing recent data showing progress made using this technique so far
• Exploring challenges associated with translating this technique from animals to humans for gene therapy

12:30 pm PANEL DISCUSSION: Evaluating Advantages and Disadvantages of Different Administration Routes

Synopsis

Delivery is a hot topic in the ophthalmology space. It’s clear that a myriad of factors influence which administration route is chosen, and it’s vital to choose the right one for your indication, taking into account what is best not just for effectiveness of the therapy, but for the patient. This panel discussion will bring together experts pioneering sub-retinal, intravitreal and suprachoroidal administration routes to delve into:
• Advantages of each route
• Challenges associated with different delivery routes and how they are being overcome
• Levels of specialism needed by physicians administering gene therapies
• Patient experience
• Exposure of vectors to their target cells
• Device development for more effective delivery

1:00 pm Lunch Break

DEVELOPING EFFECTIVE PRE-CLINICAL MODELS FOR TRANSLATION TO THE CLINIC

2:00 pm Development of Preclinical Models Supporting CPK850 for RLBP1 Retinitis Pigmentosa

  • Chad Bigelow Associate Director, Ophthalmology Research, Novartis Institutes for BioMedical Research

Synopsis

• Understanding the limitations of preclinical animal models is key to developing a path to the clinic for gene therapies
• Probing multiple species informs the nonclinical safety profile
• A combination of biologically-relevant animal models and translatable biomarkers increases confidence in efficacy-based dose selection

2:30 pm Determining the Right Dose to Deliver to the Eye

  • Dan Chung Therapeutic Area Leader Ophthalmology, Spark Therapeutics

Synopsis

• Understanding the unique challenges of achieving the correct dose in the eye
• Discussing why getting the dose right is not just sufficient but necessary to succeed
• Exploring animal models which will translate well into the clinic

3:00 pm Studying Retinal Degeneration in Preclinical Models for Inherited Retinal Disorders

  • Sandeep Kumar Principal Scientist, Ophthalmology, Absorption Systems

Synopsis

  • Understanding transgenic preclinical models for inherited retinal disorders with either retinal pigmented epithelium cells or photoreceptor specific mutations.
  • Using advanced in vivo techniques like multicolor retinal fundus imaging, spectral domain optical coherence tomography (SD-OCT), optokinetic response (OKR) and electroretinography (ERG) to identify changes in retinal morphology and function.
  • Application of these advanced techniques in the transgenic animal models can provide valuable information to support the development of gene therapies.

3:25 pm Afternoon Break

INCORPORATING PATIENTS INTO GENE THERAPY PROGRAMS

4:00 pm Implementing a Patient Registry to Accelerate Gene Therapy Development

  • Brian Mansfield Executive Vice President, Research & Chief Scientific Officer, Foundation for Fighting Blindness

Synopsis

  • Developing a registry for retinal diseases – how it works
  • Exploring the need for open access genetic testing to accelerate patient diagnosis and identification
  • Supporting clinical trial recruitment by efficiently finding interested parties

4:30 pm The Role of Patient-Oriented Research in Health Technology Assessments for Gene Therapies

  • Chad Andrews Senior Advisor on Policy, Equity, and Access, Fighting Blindness Canada

Synopsis

• Overviewing the history and features of patient-oriented research as a new investigatory paradigm

• Discussing the role that patient-oriented research projects, particularly so-called “burden of illness” studies, play in the health technology assessment (HTA) landscape in Canada and elsewhere

• Considering gene therapies in particular, and the challenges they present for researchers and HTA bodies

5:00 pm Incorporating the Patient Voice

Synopsis

  • Understanding the patient experience so you can better meet them where they are
  • Understanding how to work effectively with a patient advocacy group
  • Sharing real-world experiences of individuals and their families

5:30 pm Chair’s Closing Remarks

5:40 pm End of Day One