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Home  >  Conferences  >  4th Annual Gene Therapy for Rare Disorders

4th Annual Gene Therapy for Rare Disorders

Feb 22 09:00 AM - Feb 25 04:00 PM, 2021   |   Boston, Massachusetts  United States Of America
Organized by : Hanson Wade
Specialties : Genetics   |   Biotechnology
Conference Brochure

4th Annual Gene Therapy for Rare Disorders is organized by Hanson Wade and will be held from Feb 22 - 25, 2021 at Boston Park Plaza, Boston, Massachusetts, United States of America.

Gene therapies are redefining the treatment of rare diseases. However, a set of complicated and different challenges must overcome these therapeutics to achieve their commercial potential.

With an abundance of population, higher doses required, production issues are more noticeable than ever. That’s why the 2020 agenda will devote two tracks wholly to overcoming manufacturing issues, by enabling you to,
• Satisfy the supervisory expectations for CMC and analytical packages
• Execute strong and scalable manufacturing processes
• Check the swiftly evolving logistical and infrastructure challenges facing the space

More gene therapies are in the clinic than ever before. In the context of this unprecedented progress, the event will give significant attention to clinical issues including:
• Maximizing significant output from trials through creative clinical trial design
• Including the patient voice into clinical development
• Operating the realities of the global regulatory landscape in light of recent approvals

How do you price gene therapies? This question and issues around reimbursement, payment models and the commercial realities of the space will form the basis of the commercial track, with insights that will enable you to:
• Understand the payer perspective on balancing access and affordability
• Contrast a variety of payment models including outcome- and performance-based payments
• Evaluating the commercial realities of rare and ultra-rare disease business models

Key Benefits of Attending:
Implement Effective Analytical & CMC Approaches to Improve Safety, Potency & Purity 

Explore technical insights into the analytical approaches supporting Spark Therapeutics’ gene therapy development and understand how bluebird bio has aligned CMC with regulatory expectations to support an approval

Establish Robust & Scalable Manufacturing Processes & Infrastructure to Support Increasing Demand
Equip yourself for commercial manufacturing, however, you utilize capacity – from making initial ‘make vs buy’ decisions, to CMO management and facility design, validation and maintenance, with insights from Pfizer, BioMarin, and Sanofi

Navigate the Complex Global Gene Therapy Regulatory Environment
Learn directly from international regulatory bodies including the FDA and China’s CFDA, to understand the realities of global regulatory expectations and how regulatory oversight is adapting to technical innovations in the field

Examine Innovative Financing & Reimbursement Strategies
Understand the payer perspective on the reimbursement strategies that are available, which approaches are working, which are not and how future government action will impact pricing and value-based agreements, with insights from Harvard Pilgrim Health, Blue Cross Blue Shield of Massachusetts and ICER

Investigate Novel Vectors to Enhance Selectivity & Efficacy While Reducing Immunogenicity Look beyond the current crop of gene therapy vectors to gain an insight into the next generation of delivery technologies with the promise to dramatically enhance the specificity of targeting, reduce costs of manufacturing and overcome immunogenicity challenges, with insights from 4D Molecular Therapeutics and Duke University School of Medicine

Additional details will be posted as soon as information is available.

View All Speaking

Luis Maranga
Luis Maranga


David M. Markusic
David M. Markusic


Emily Milligan
Emily Milligan

Public Health

Palani Palaniappan
Palani Palaniappan

Biochemistry and Molecular Genetics




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Venue Address

Boston Park Plaza
50 Park Plaza
Boston, Massachusetts, United States Of America

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