8:00 am Check-In & Coffee Networking
8:25 am Chair’s Opening Remarks
Addressing Off-Target Toxicity to Improve Vector Efficacy & Safety
8:30 am Exploring In Vitro & In Vivo Models to Better Understand AAV Vector Specificity
Synopsis
- Appreciating the advancements made to getting to CNS tissue but realizing the opportunity to use effective gene editing techniques to enhance tissue targeting
- Utilizing in vitro and in vivo models to test AAV vectors specificity for CNS delivery
- Discussing future thoughts: how can more human-based in vitro models improve testing in specific indications and thus enhance translatability?
9:00 am Utilizing Payload to Harness Tissue Specificity for CNS Vector Therapies
Synopsis
- Discussing the strategies used to regulate transgene expression cassettes for AAV vectors targeting the CNS
- Exploring how fine-tuning payload expression enhances tissue tropism, safety, and efficacy for CNS indications
- Revealing some of the considerations needed to increase the tissue tropism of viral vectors
9:30 am Exploring Enzymatic Conjugation as a Capsid Design Approach to Improve Vector Targeting
Synopsis
- Understanding the approach of enzymatically conjugating chemicals or biologics to the AAV capsid
- Highlighting how this rational design approach can increase tissue tropism and eliminate liver targeting
- Emphasizing how increasing specificity also improves potency and limits toxicity
10:00 am Morning Break & Networking
Revolutionizing Vector Production for Maximum Impact
11:00 am Discovering AAV Capsids for Ocular & CNS Gene Therapy by NAVIGATE Directed Evolution
Synopsis
- Exploring strategies for performing capsid directed evolution and considerations for on- vs off-target activity and toxicity
- Understanding how directed evolution of capsids in NHPs can facilitate targeting ocular tissues by non-invasive suprachoroidal space administration
- Emphasizing how capsids engineered in vitro permit crossing of the blood-brain barrier by targeting the transferrin receptor
11:30 am Round Table: Enhancing Vector Manufacturing to Improve Quality & Cut Costs
Synopsis
- Demonstrating how reproducible manufacturing processes can improve vector production
- Highlighting the need to produce high quality vectors to lower doses and enhance safety
- Emphasizing the importance of improved quality and quantity vectors to reduce costs
12:30 pm Lunch Break & Networking
Harnessing Learnings From AAV Vectors to Progress Other Vector Therapies
1:30 pm Rethinking Vector Choice: Utilizing Optimized HSV to Enhance Safety & Efficacy
Synopsis
- Reflecting on the lack of efficient tissue targeting impacting AAV vector development
- Demonstrating that HSV vectors have excellent cellular tropism biodistribution shedding characteristics with regional administration
- Highlighting how regional administration minimizes off-target effects, improves safety, efficacy, and limits immunogenicity
2:00 pm Leveraging Parvovirus Vectors to Combat Immunogenicity, Off-Target Effects, & Limited Payload Capacity
Synopsis
- Highlighting how selecting a vector with limited pre-existing human immunity and excellent tissue tropism improves vector efficacy and safety
- Exploring the benefits of additional cargo space to treat a wider range of indications
- Consolidating learnings and regulations from AAV vectors to facilitate parvovirus vector development
2:30 pm Afternoon Break & Networking
Strategically Injecting Capital into Gene Therapy Vectors
3:00 pm Panel Discussion: Advancing Gene Therapy Vectors with More Insightful Investment
Synopsis
- Discussing the importance of investment into the gene therapy vector space to characterize attractive investment prospects
- Refocusing investment strategies to primarily propel vector therapy innovation
- Determining how workflows and mindsets from the antibody therapy space may hinder vector therapies from reaching their potential