Agenda

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ACCELERATING BIOTHERAPEUTIC DEVELOPMENT AND PRODUCTION FOR INCREASED MARKET IMPACT OF LIFE SAVING THERAPEUTICS
Sponsored by Thermo Fisher Scientific
This panel of experts will share their insights and experience on some of today’s most pressing development challenges. The session will dive into key topics such as integrated vs. modular manufacturing approaches, ensuring security of supply, proactive approaches to regulatory compliance, and ultimately, the panel’s vision for the future.

ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPIES: LESSONS LEARNED AND FUTURE FOCUS
Sponsored by Evidera PPD
The first wave of transformative cell and gene therapies have launched into a healthcare landscape that arguably was not built with them in mind. Hurdles range from basic understanding to value demonstration, reimbursement and access. This session will debate lessons learned from the initial vanguard of cell and gene therapies, including building the right evidence, navigating the provider-side, achieving value-based payment, and ensuring patient uptake. Discussion will cover unanticipated challenges and critical success factors for future cell and gene therapy success.

ALTERNATIVE IPO STRATEGIES FOR CELL AND GENE THERAPY COMPANIES
Sponsored by Chardan
This session will center on special purpose acquisition companies (SPACs), a space heating up within the biotech world. As of August 2020, 10 biotech-focused SPACs have been successful at raising capital with many others coming down the pike. The purpose of the panel is to understand why all these biotech-focused firms are getting involved in the space, what cell and gene therapy companies need to know about this alternative to a traditional IPO if approached, and what seasoned executives of public companies should know about potentially sponsoring their own biotech focused SPAC.

mRNA PROCESS DEVELOPMENT AND MANUFACTURING ON THE ROAD TO THE CLINIC
Sponsored by Aldevron
Messenger RNA offers a promising new approach to address emerging pathogens and unmet medical needs. Shorter lead times and greater simplicity in manufacturing enable faster responses that will transform medicine. This panel will discuss the state of the art with an emphasis on CMC, manufacturing, and associated regulatory issues.

CURRENT HOT TOPICS AND EMERGING TRENDS IN GENE THERAPY
Sponsored by Pfizer
This panel will focus on emerging gene therapy scientific breakthroughs and challenges as well as notable clinical development advancements and results. Discussion will also touch upon gene therapy manufacturing challenges, evolving regulatory frameworks, and the gene therapy financing and investor landscape.

DOING BUSINESS IN JAPAN – AN EVOLVING MARKET: THE LATEST RISE OF COMMERCIALIZATION AND PROMOTING HARMONIZATION ON REGENERATIVE MEDICINE WITH ASIA
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

NOVEL CLINICAL TRIAL DESIGNS: MORE EFFICIENT AND EFFECTIVE TESTS OF REGENERATIVE MEDICINES DURING THE PANDEMIC AND BEYOND
Sponsored by IQVIA
This panel of industry, advocacy, and academic leaders will discuss challenges faced and innovative solutions explored, in addressing the unique requirements of clinical studies in cell and gene therapy. Participants will present their first-hand experience with various novel trial designs/elements including remote endpoints/telemedicine, virtual trials, hybrid/decentralized trials, platform trials, and external comparators. Cell and gene therapy trials already incur a unique set of challenges, and COVID-19 has dramatically transformed the clinical trial landscape, highlighting and accelerating the need for new ways of thinking that are more relevant now, but which will continue to be increasingly employed going forward.

SAFETY AND EFFICACY CONSIDERATIONS FOR CELL AND GENE THERAPIES: FROM MANUFACTURING THROUGH DISTRIBUTION
Sponsored by Thermo Fisher Scientific
This panel discussion will cover a number of safety and efficacy factors for advanced therapies including acceptable impurity levels, dosing considerations, the impact of platforms on the potency of products, and maintaining integrity of the material from the manufacturing floor to the clinic.

EUROPEAN COMMERCIALIZATION CHALLENGES AND OUTLOOK
With 10 advanced therapy medicinal products (ATMPs) currently marketed in Europe and four withdrawn, the sector’s commercial landscape is at turning point in Europe. So far, healthcare systems and developers have been successful in opening the way to the cell and gene therapy revolution. Treatment centers are managing patients, payers are exploring innovative ways to cover the cost for transformative therapies, and developers are inventing new ways of doing business. The next step will be to consolidate all of these successes and make them available for a significantly larger number of patients. On this panel, European business leaders will share their expectations and discuss how they are preparing to overcome these challenges.

GMP MANUFACTURING OF CELL AND GENE THERAPIES – A 2020 PERSPECTIVE
This session will include a discussion about issues related to moving from development through to GMP manufacturing of cell and gene therapies including challenges, scalability, standards, and issues that have been raised in light of COVID-19.

STATE OF PLAY IN THE VECTOR ARMS RACE: PERSPECTIVES ON THE FUTURE OF GENE DELIVERY
Join the CEOs of three leading gene therapy companies as they discuss the relative merits of AAV, lentiviral, and non-viral delivery technology – and share their views on how these platforms will help shape the future of gene therapy. Expect a lively, insightful, and thought-provoking discussion. Both Prevail Therapeutics and AVROBIO are in the clinic and have publicly shared patient data while Generation Bio is preclinical with a novel non-viral platform.

VITALITY OF THE RANGE OF INVESTING: EARLY-STAGE SEED INVESTING TO PUBLIC INVESTING
This discussion between three leaders in biotech/pharma investing will delve into the vitality of early stage seed funding to mid stage to public investing in established companies, and how these stages of the investment spectrum interact. The panel will also discuss the importance of alternative investment structures such as venture philanthropy to promote therapeutic pipelines.

ACHIEVING DIVERSITY IN CLINICAL TRIALS
While obtaining racial diversity in clinical trials is critical to ensuring that the patients enrolled are representative of the population that will receive treatment, many clinical trials fail to achieve sufficient representation. The ARM Action for Equality (AFE) Taskforce has convened this panel to discuss the barriers, lessons learned, and opportunities to increase representation in clinical trials evaluating products for cell and gene therapy.

BESPOKE THERAPIES FOR ULTRA-RARE DISEASE
This session will explore the National Institutes of Health (NIH)’s public-private partnership to help individualized gene therapies reach patients despite their lack of commercial viability. Panelists will provide an overview of this initiative including how it will maximize benefits to patients, as well as explore the specific opportunities and challenges related to its success.

CURES 2.0 AND BEYOND: WHAT’S NEXT IN U.S. POLICY AFFECTING THE CELL AND GENE SECTOR

THE EUROPEAN REGULATORY ENVIRONMENT FOR ATMPS – SHOULD WE EXPECT MORE OR LESS REGULATION?
This session will discuss the recent initiatives and plans that will affect the regulatory environment for advanced therapy medicinal products (ATMPs) in Europe, such as the EC Pharmaceutical Strategy, the forthcoming implementation of the new Clinical Trial Regulation, GMO requirements for gene therapies, and the European Regulatory Network Strategy to 2025. The impact of these developments on patient access to ATMPs, on the ATMP sector in Europe, and on regulatory bodies will be addressed.

ARM ACTION FOR EQUALITY TASK FORCE INITIATIVES
The Alliance for Regenerative Medicine (ARM) Action for Equality (AFE) Task Force is exploring what actions ARM and ARM members can take to eliminate racial inequality and address underrepresentation of Black Americans and minorities in the regenerative medicine sector. Our goal is to create a meaningful impact through various initiatives that will provide minorities with more opportunities, exposure, and mentorship in regenerative medicine. The Task Force aims to build a successful program that ARM and ARM Members are energized to pursue together.

CELL AND GENE THERAPY PORTFOLIO STRATEGIES
This session will focus on how mid- to large-sized biotech and pharma companies develop a portfolio strategy designed to effectively progress cell and gene therapies. The panelists will discuss their respective approaches to building a robust pipeline.

FDA WORKING WITH PATIENT GROUPS
This panel will discuss innovative ways patient groups can work with the FDA throughout the drug development and evaluation process. In this session, panelists will explore the use of real-world evidence, patient experience, and registries as replacements for more traditional methods of data collection.

OVERCOMING CHALLENGES WITH THE DEVELOPMENT AND TRANSLATION OF A THERAPEUTIC FROM ACADEMIA TO INDUSTRY
Some of the most cutting-edge and innovative approaches to cell and gene therapy have originated from the academic setting. However, getting these treatments into the clinic can be especially challenging when the institution does not have the experience and infrastructure necessary to conduct preclinical studies, manufacture to GMP standards, and file regulatory submissions with the FDA. This panel of experts will discuss their lessons learned throughout the process and best approaches to moving a cell and gene therapeutic candidate from academia to commercialization.