Cell and Gene Therapy World Congress 2024

2nd Cell & Gene Therapy World Congress 2024 Europe

Driving innovation, addressing significant unmet medical needs, and ultimately improving patient outcomes

00Days
00Hours
00Minutes
00Seconds

2nd Cell & Gene Therapy Global Congress 2024 Europe

The Fastest Growing Cell & Gene Therapy Networking Event.

Introduction
Insight
Who Should Attend

Cell and Gene therapies are forms of personalised, regenerative medicine. Described as the most innovative, promising and life-changing new categories of medicines whose full potential is only just beginning to emerge. They are cutting-edge and rapidly progressing fields of Biological Therapies. Also known as “Advanced Therapy Medicinal Products”, they involved the use of cells, tissues and genes to treat, prevent or potentially cure and eliminate complicated health conditions and difficult to treat diseases. These therapies promise life-changing treatments for a broad spectrum of complex diseases ranging from genetic conditions to cancers. Both approaches offer the potential to diminish the fundamental cause of genetic and acquired diseases by substituting the missing protein or cells causing the disease’s symptoms. Cell and gene therapies may offer longer lasting effects than traditional medicines. Convergence of advanced biotechnology, precision medicine, and expertise in manufacturing has facilitated the way for these therapies to become a reality, with remarkable successes observed in conditions such as cancer and genetic disorders.

Cell and Gene therapy has transformed the healthcare and biopharma industry by storm. Currently, after around 40 CGT launches across the EU and US, the market is worth around EUR 4.5 billion. A similar number are expected to be approved by 2028, when sales are projected to rise to EUR 19.2 billion. The development of biological therapies is rapidly expanding, however, commercialisation of this treatment poses different challenges to research and development, authorisation, manufacturing, accessibility, and ensuring safety throughout the lifecycle of the product.

The two-day Congress will provide an interactive, cutting edge, and comprehensive discussions in collaborative format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices on challenges, innovations, technologies, and concepts to help you gain access to the booming CGT market.

We are putting together an agenda that focuses on “driving innovation, addressing significant unmet medical needs, and ultimately improving patient outcomes”.

We are gathering high calibre expert speakers to participate and share their knowledge and expertise through Keynote Presentations and Panel discussions, addressing all aspects of the industry.

We look forward to welcoming you to the CGT World Congress 2024!

Sincerely yours,
Jocelyn Raguindin
Conference Director
Paradigm Global Events / Facilitate Live

GAIN LATEST INSIGHTS ON:

Gain the latest insights to successfully navigate the complex access landscape of CGT
Explore ways on how to strategically navigate the state of the art technologies in cell and gene therapies
Understand approaches in advancing research and drug development
Strategies in making HTAs more CGT focused
Tackling barriers in regulatory compliance: discussions to streamline the process
Know how to engage early with regulators and payers to enable swift roll-out of new therapies for the benefit of the patients
Learn the production process which is crucial for new players entering the field
Robust processes essential for ensuring performance, which helps prevent batch failures and associated costs
Scalable technologies to manufacturing and to meet increased patient demand
Have a meaningful conversation on how to improve access efficiently to innovative treatment to reach patient in a shorter period of time
Find out if CGTs offer long-term value for money to manufacturer, regulators and payers
Addressing the challenges associated with pricing and market access
Network and collaborate with key stakeholders, regulators, academia, HTAs and payers

WHO SHOULD ATTEND?

Network with Presidents, Heads/Chiefs, VPs, Directors, and Managers in the area of:

Clinical Research Organizations
Clinical Operations
Medical Affairs, Global Patient Advocacy & Alliances
Research and Development
Rare and Ultra-Rare Diseases
Cell and Gene Therapy
Regenerative Medicine
Government Policy and Advocacy
Digital Patient Experience Lead
Clinical Insights and Experience
Head of Strategy, Access Services
Vice President, Site Collaborations and Patient Centricity
Head of Neuroscience
Personalised Medicine
External R&D Innovation
Innovative Medicine
Translational Science
Molecular Geneticist
Program Management
Patient Advocacy Groups
Public Affairs
Medical Affairs
Regulatory Affairs
Market Access
Pricing and Reimbursement
Health Economics Outcomes Research
Commercial Development
Investments and Funding
Product Specialist
Global Strategic Services
Business Planning & Operations

Head of Patient Engagement
Patient Services
Engagement Strategy
R&D Patient Engagement
Medical Affairs
Regulatory Affairs and Policy
Patient Support and Patient Engagement
RWE, and Data Management
Quality and Compliance
Clinical Development
Programme Management
Supply Chain Management
Patient Access
Clinical Research
Global Head, Digital Accelerator
Patient Engagement & Portfolio Strategy
Patient Support Strategy & Insights
Patient Experience
Clinical Research Organizations
Research and Development
Personalised Medicine
Pharmacies
Academia
And much much more..

speakers

0 +

days Congress

Presentation

0 +

Attendees

0 +

Key Industry Expert Speakers

Comfirmed speakers Apply to speak

Schedule

Content Rich Program Agenda! Featuring Keynote Presentations, Stream Sessions, Panel, and Round Table Discussions.

CELL & GENE Therapy - Day 1
CELL & GENE Therapy - Day 2

2nd CELL & GENE THERAPY WORLD CONGRESS 2024 EUROPE

"Driving Innovation, Addressing Significant Unmet Medical Needs, and Ultimately Improving Patient Outcomes"

Day 1 - Thursday 3rd October 2024

08:30 - WELCOME COFFEE & REGISTRATION

9:00 - CHAIRPERSON’S OPENING REMARKS

COMMERCIALISATION & REGULATORY LANDSCAPE

9:15 am - OPENING KEYNOTE PANEL: EXAMINING THE EVOLVING LANDSCAPE OF CELL AND GENE THERAPY

Latest trends and valuable insights to drive innovation and remain competitive
Cutting-edge technologies revolutionizing treatment paradigms
Aligning with international regulatory standards in order to support the broadest possible patient access.
Ensuring affordability and accessibility of these therapies
Key drivers for attracting investment

Panellist:

Jeff Liter, CEO, OX2 Therapeutics

Dilip Patel, VP, Head of Global Market Access, Autolus

10:00 am - KEYNOTE ADDRESS: OVERVIEW OF UK CELL AND GENE THERAPY SECTOR

Introduction
Demonstrating the UK’s continuous strength in the cell and gene therapy sector.
Challenges facing companies in this space
Collaborative work and initiatives to guarantee continued growth of the sector
Lessons learned and strategies to unlock the full potential of this developing field

10:30 am - REGULATORY KEYNOTE: THE EU HEALTH TECHNOLOGY ASSESSMENT: HOW DOES A FIT FOR PURPOSE JCA IMPACT CELL AND GENE THERAPY DEVELOPMENT?

There are a number of changes and policy initiatives currently being developed which will start going into effect in 2025 and have a significant impact on the future of ATMPs in the EU.
At the EU level, we have the EU HTA Regulation, discussions on the General Pharmaceutical Legislation, the Biotechnology Strategy, and a renewed focus on the competitiveness agenda.
At national level, different Countries are reviewing their P&R and early access programmes.
For developers, it is important to take a step back and look at the landscape holistically when considering pipelines and forecast the direction of travel in the EU for the next 5 years, and beyond.

Dr. Alexander Natz, Secretary General, EUCOPE

11:00 - MORNING NETWORKING REFRESHMENTS

11:40 am - SPONSORED SPOTLIGHT: A HOLISTIC APPROACH IN CELL AND GENE THERAPY LOGISTICS MANAGEMENT

Understanding the key logistics requirements in operationalizing Cell and gene therapy trials
Comprehensive strategies to logistics management
Addressing a whole range of intricacies across all types of CAGT assets
Significance of stakeholder partnerships to optimize supply chain and enhance patient experience

12:10 pm - SPONSORED SPOTLIGHT: DEVELOPING AND LAUNCHING A SUCCESSFUL GENE THERAPY: HOW TO ADAPT TO NEW BUSINESS DESIGNS AND PRICING MODELS?

Ways to navigate and succeed in this promising yet competitive sector
Strategic portfolio assessments and management critical to success
Importance of robust clinical pipeline to attract interest from investors
Considering the complexity of the manufacturing process, supply chain requirements of each, along
with the required regulatory pathway to market.

12:40 pm - Strategies to mitigate the commercial/technical barriers to scaling stem cell derived regenerative medicines / cell therapies

To Follow

Adam Glen, Co-founder, Unicorn Bio

13:00 - NETWORKING LUNCH

DEVELOPMENT & MANUFACTURING

2:00 pm - New class of cancer vaccines based on an off-the-shelf Antigen Presenting Cell line (PDC*line)

Last clinical results in NSCLC cancer patients in association with Pembrolizumab

Eric HALIOUA, President & Chief Executive Officer, PDC*line Pharma

2:30 pm - Preclinical development of gene therapy applications for Duchenne muscular dystrophy

Muscular dystrophy as paradigm for orphan drug development
Most promising gene therapies strategies for the treatment of Duchenne muscular dystrophy
Future perspectives; orphan drugs in the pipeline

Dr Alberto Malerba, Lecturer in Gene Therapy, Gene Medicine Laboratory for Rare Diseases, Royal Holloway, University of London

3:00 pm - CAR-T CELLS MANUFACTURING LANDSCAPE: LESSONS LEARNED AND CONSIDERATIONS FOR EARLY CLINICAL DEVELOPMENT

Current landscape of CAR-T cells manufacturing
Challenges limiting production capacity
Latest technical developments in GMP production platform design
Challenges associated with translation of new scientific developments into clinical products for patients

Dr Theres Oakes, Head of Product Characterisation, Achilles Therapeutics

3:30 pm - AFTERNOON NETWORKING REFRESHMENT

4:10 pm - Advance therapies at NHS Blood Transplant*

Utilising NHSBT’s supply chain and infrastructure to deliver ATMPs into NHS
Manufacturing of red blood cells in vitro for RESTORE clinical trial
Manufacture of gene therapy products at NHSBT’s Clinical Biotechnology Centre

Dr Lilian Hook, Director Cell, Apheresis & Gene Therapies, NHS Blood and Transplant

4:40 pm - RNA, DNA And LNP Targeting To Brain And Other Organs

In contradistinction to other forms of therapy RNA activation activates gene expression via transcription to physiological levels
Transferrin RNA aptamer can deliver various RNA oligonucleotides to the central part of the brain.

Dr Nagy Habib, Professor of Surgery, Imperial College London

5:10 pm - PANEL DISCUSSION: INNOVATIVE PROCESS DEVELOPMENT AND MANUFACTURING TECHNIQUES FOR CELL AND GENE THERAPIES

State of continuous process implementation and some ongoing challenges.
Technological advancements that helps drive batch productivity and reduce the costs for patients
Improving the design and efficiency of facilities to focus on quality, getting into manufacturing quicker
Lowering the overall cost of goods and the amount of raw materials used
Lessons learned and best practices

Moderator:
Jeff Liter, CEO, OX2 Therapeutics

Panellist:
Alex Blyth, Founder & CEO, Lift Biosciences

5:55 pm - CHAIRPERSON’S CLOSING REMARKS

2nd CELL & GENE THERAPY WORLD CONGRESS 2024 EUROPE

"Driving Innovation, Addressing Significant Unmet Medical Needs, and Ultimately Improving Patient Outcomes"

Day 2 - Friday 4th October 2024

8:30 - WELCOME COFFEE & REGISTRATION

09:00 - Chairperson’s Opening Remarks

COLLABORATION AND PATIENT ACCESS

09:15 - OPENING PANEL DISCUSSION: ADDRESSING KEY CHALLENGES TO THE ATMP INNOVATION ECOSYSTEM THROUGH STAKEHOLDER COLLABORATION

The importance of stakeholder engagement: perspectives across different functions
Creating a CGT platform: stakeholder engagement as a tool to support not only the development/commercialization of specific assets, but to build a sustainable platform
How national level activities can impact on patient access to ATMPs
Further national level collaboration between academic, clinical and industrial players is necessary to overcome the existing hindrances and boost further development of ATMPs in Europe.

Panellist:
Dr. Alexander Natz, Secretary General, EUCOPE

10:00 am - The socio economic impact of the ultra rapid cell therapy evolution on the global health care systems with respect to low income countries

Cell based therapeutics are promising tools revolutionizing the treatment protocols for severe diseases. Euopean countries are showing a likely dispersed pattern in terms of the regulation of market access regulations as well the pricing structures and compensation policies of insurances.

Marco Traub, CEO, The Trans European Stem Cell Therapy Society

10:30 am - BRIDGING THE GAP: EQUITABLE AND TIMELY ACCESS TO INNOVATIVE TREATMENT

The significance of rationalizing clinical trials to reduce logistical and financial burdens for participants
Leveraging technology to enhance access to treatment and specialist care
Data-driven strategies to make informed decisions
Patient-centred approach for better experience and outcomes

11:00 am - MORNING NETWORKING REFRESHMENTS

11:30 am - Biofoundry-in-a-Box: By-Design Biologics drug product manufacture for rapid response

Focusing on ensuring the equitable access to biologics and specifically RNA based drug products by everyone, everywhere.

Prof Harris Makatsoris, Professor of Manufacturing Systems, Department of Engineering, King’s College London

12:00 pm - SPONSORED SPOTLIGHT: TRANSLATING EARLY RESEARCH INTO PROMISING NEW ATMPS FOR PATIENTS THROUGH PUBLIC-PRIVATE PARTNERSHIPS

Unique public-private partnership that has increased the access to new treatment options for patients
Promote further innovation in clinical research using the strengths of the partnership
Align potentially conflicting organizational cultures to maximize efficiencies and share project leadership
Utilize a precision medicine master protocol to bring cutting-edge advances
Allow for broader patient access and clinical trials that are more inclusive than standard industry-run trials

12:30 pm - Systematic Serendipity - A Novel Approach for Developing New Therapies for Unaddressed Rare Diseases

How the Systematic Serendipity methodology de-risks rare disease therapeutics development
Leveraging human and machine intelligence to build collaborative teams around relevant therapeutic opportunities
Case studies and outcomes from our recent cohorts will be presented

Dr Ron Jortner, Founder/CEO, Aspire Biosciences

1:00 - NETWORKING LUNCH

MARKET ACCESS AND PRICING

2:00 pm - CELL AND GENE THERAPY ACCESS MODELS: OVERCOMING BARRIERS TO CGT ACCESS

Circumventing operational complexities and regulatory barriers
Consider near- and longer-term implications and opportunities to influence the design of the model
Ability of the model to reduce costs and improve access
Monitoring the development to better understand successful contracting arrangements
Assess how potential therapeutic area expansion may impact pricing, reimbursement, and access for other gene therapy products.

Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross

2:30 pm - Perspective on Cell and Gene Therapy in Japan

Introduction of GCT Market Potential and Trends in Japan (commercial, regulatory and reimbursement)
Opportunities in Japanese GCT market (political considerations and incentives)
Challenges and Recommendations

Mohamed Oubihi, Founder and CEO, YAKUMED LIMITED

3:00 - AFTERNOON NETWORKING REFRESHMENT

3:30 pm - PANEL DISCUSSION: INNOVATIVE PAYMENT SOLUTIONS: LESSONS LEARNED AND SUCCESS STORIES FROM GENE THERAPY LAUNCHES

Current landscape
Existing innovative contracting with gene therapies
How to overcome key challenges to innovative payment models.
Leveraging real-world evidence to define outcome measures
Implications to gene therapy manufacturers

4:45 - CLOSING PANEL DISCUSSION: LOOKING INTO THE FUTURE OF NEXT GENERATION ATMP’S IN EUROPE AND BEYOND

Breakthroughs and innovations in research and development
Implementing next-generation technologies
Novel pricing and market access strategies pharma will employ to increase patient access
Various CGTs in the pipeline
What will the next decade hold for these life-changing therapies?

5:00 - CHAIRPERSON’S CLOSING REMARKS

Event sponsors

Cell & Gene Therapy Global Congress 2024 Europe

Become a sponsor/exhibitor

Newsletter Sign Up

No Spam - You will only receive relevant information on Cell & Gene Therapy, and events updates.

Testimonials

what people say

Thank you for a good experience with the PARADIGM Global Events… very high level, very good Organization, we overpassed our expectatives, we will be pending for another congress related to pharmaceutical and biotech… we learned a lot, and i think that the content of agenda was state of the art, thank you again.

SALVADOR BERRIOS

ERP/ CRM / Product Configurator Manager

The consistently high quality of international faculty was most impressive. It was also obvious that they were carefully chosen not only for their particular expertise but also for their ability to communicate the subject matter to clinical and non-clinical participants alike. As market access consultant for pharma clients of various sizes, I now feel much better prepared to explain the nuances & dynamics involved in bringing their products to market. In addition, I am in a much better position to intelligently advice my clients & help them set realistic expectations

THOMAS MARTIN

Associate Vice President, VCGA - Quintiles

I was impressed with the organization of this event. I attend many conferences per year and find the organizers on site to be detached and not focus on good service to the delegates. This team was even providing weather forecast warning to ensure everyone was aware and made appropriate plans. Well Done.