*All times are shown in EST
THE EVOLVING GENE THERAPY FOR BLOOD DISORDERS LANDSCAPE
8:00 am Chair’s Opening Remarks
8:20 am Discussing Gene Therapy in Hemophilia– What Does the Past, Present and Future Look Like?
Synopsis
- Discussing the history, recent advances and potential future developments of gene therapy in hemophilia
- How recent developments in the approval space may influence the future
- The critical importance of solving “Known Unknowns”
8:40 am Towards a Curative Treatment for Hemophilia A Using Lentiviralbased Hematopoietic Stem Cell Gene Therapy
Synopsis
- Over the past decade treatment options for persons with hemophilia A have increased and allow for individualized treatment decisions
- AAV-based gene therapies have advanced clinically, but long-term durability remains to be determined
- Lentiviral-based hematopoietic stem cell gene therapy provides an alternative to AAV-based gene therapy and the potential for sustained and curative fVIII expression
- Advancements in lentiviral vectors and autologous cell product manufacturing, as well as targeted-stem cell conditioning agents, further support this promising gene therapy platform.
9:00 am Optimizing Regulatory Strategy for Gene Therapies: Current Trends, Guidance & Industry Milestones
Synopsis
- Get the latest updates on the dynamic regulatory landscape
- Explore how new and upcoming guidance has reshaped strategy
- Apply lessons learned from key regulatory milestones across the industry
9:20 am Q&A Panel Discussion
9:50 am Speed Networking
Synopsis
Grab a quick cup of tea or coffee from the comfort of your own home or office 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!
10:20 am Morning Break
ADVANCING GENETIC THERAPIES FOR BLOOD DISORDERS TOWARDS THE CLINIC
10:40 am Reactivation of Fetal Hemoglobin Expression via Cas12a Ribonucleoprotein-Mediated Gene Editing for the Potential Treatment of Sickle Cell Disease
Synopsis
- Discussing how reactivating fetal hemoglobin can be used to treat sickle cell disease
- An overview of the preclinical data supporting the development of EDIT-301, an experimental autologous cell therapy for the potential treatment of sickle cell disease
11:00 am Progress with Non-Viral Gene Therapy Technologies
Synopsis
- Utilizing Poseida’s proprietary platform technologies for gene addition
(piggyBac® transposons) and high-fidelity gene-editing (Cas-CLOVER™ CRISPR) - Ex-vivo application for gene-correction of hematopoietic stem cells and in manufacture of CAR-T cells
- In-vivo delivery with nanoparticles for permanent liver-directed treatment of
genetic diseases
11:50 am Gene Editing with Investigational Zinc Finger Nucleases for the Potential Treatment of Sickle Cell Diseas
Synopsis
An overview of the supporting preclinical data of a novel ex vivo gene editing approach to increase fetal haemoglobin in sickle cell disease will be presented
Details of the technology and detailed characterization of the gene editing will be reviewed
Clinical trial design and updates will also be presented
12:10 pm Q&A Panel Discussion
12:40 pm Lunch & Networking
OPTIMIZING CLINICAL TRIAL DESIGN & COLLABORATION
1:40 pm Optimizing the Clinical Development Strategies to Accelerate the Timing of the Submission & Demonstrate Treatment Effect Durability for AAV Platform
Synopsis
• How the recent FDA feedback impacts the clinical development with regards
to surrogate endpoints and clinically meaningful endpoints
• What is the right patient follow up for the accelerated submission?
• How long do we have to follow up with the patients from the scientific,
patients community, and payer points of view?
2:00 pm Anticipating the Commercial Implementation Challenges of Gene Therapies for Hemophilia
Synopsis
- Considering emerging gene therapy for hemophilia with impending transition to commercialisation, discussing the evolving issues and questions that treaters and patients will face with such a new approach to treatment
- Based on the challenges experienced with clinical trials, what strategies and preparations are required to successfully implement gene therapy treatments for hemophilia?
2:20 pm Q&A Panel Discussion
2:50 pm Afternoon Break
OVERCOMING CLINICAL ENROLMENT CHALLENGES TO INCREASE DATA QUALITY & ADHERE TO SHORTER TIMELINES
3:20 pm Improving Awareness, Education & Accessibility for Patients to Improve Enrolment
Synopsis
- Increasing the speed of recruitment for clinical trials when other treatments are available, particularly with shorter timelines, and educating patients on a novel therapeutic
- Addressing the lack of ethnic diversity and social-economic diversity to be more representative of the target patient population
- Improving access to and quality of clinical trial information and access to the clinical trial themselves (diversity of sites, travel reimbursement, appointment timings etc.)
3:40 pm Pediatric Gene Therapy & Medical Ethics: Informed Consent
Synopsis
- Informed consent and pediatric assent for trial participation
- Novel considerations in operationalizing consent in gene therapy
- Emerging ethical dilemmas in pediatric gene therapy research
4:00 pm Jumping into Icy Water: A Patient Perspective on Enrolment Challenges for Gene Therapy Trials
Synopsis
- Informing the patient community through the patient associations
- Emphasizing “informed” in the informed consent process
- Making clinical trials “patient friendly”
4:20 pm Q&A Panel Discussion
5:20 pm Biotech Quick Fire Round: Introducing the New Preclinical Players
Synopsis
• David Gutstein, Vice President, Strategic Program Direction – Hematology
Translational & Clinical Sciences, Regeneron Pharmaceuticals
• Oscar Segurado, Chief Medical Officer, ASC Therapeutics
• David Wilcox
6:00 pm Chair’s Closing Remarks
