8:00 am
Registration & Breakfast

8:50 am Chair’s Opening Remarks

INSIGHTS FROM REGULATORY EXPERTS & THE REGULATORS THEMSELVES

9:00 am Securing & Maintaining an Orphan Drug Designation

  • Gloria Palomo Carrasco Non-Clinical Assessor, AEMPS; Member of the Committee for Orphan Medicinal Products, EMA, AEMPS, EMA

Synopsis

  • Outlining the most important principles in applying for an EMA orphan designation
  • Understanding how to use data to fulfil the designation criteria, and the importance of maintaining designations by the marketing authorisation phase
  • Highlighting the importance of ‘significant benefit’

9:30 am Clarifying the Differences in Orphan Drug Designation Between the EMA & FDA

Synopsis

  • Highlight the key strategic differences between the two bodies for success in gaining Orphan drug designations
  • Explore the main incentives related to Orphan designations
  • Understand the concept of similarity for Orphan drug exclusivity

10:00 am Roundtable: Insight on Case-by-Case Reviews of Various Regulatory Submissions

Synopsis

There are currently over 209 companies in Europe developing regenerative medicines and ATMPs. In lieu of this however, there are only 11 EMA-approved gene therapies in the market. To gain an insight into best practices navigating regulatory hurdles during various stages of the gene therapy development process, we will dive deeper into some gene therapy success stories, facilitated by the following regulatory affairs specialists:

10:30 am
Morning Break & Networking

INSIGHTS INTO THE NEW CTIS PLATFORM & ALL THINGS GMO

11:00 am GMO Legislation Implementation Across the EU & Where These Frameworks Fit into the CTIS

  • Nathalie Lambot Public Health Advisor: Clinical Trials – Regulatory Affairs, pharma.be

Synopsis

  • The national adaptation approaches to gaining GMO approval for ATMP clinical trials
  • The importance of each EU Member State facilitating pragmatic and simple approaches to GMO assessments
  • What submission structures exist for GMO applications via the new CTIS platform?

11:30 am Advocacy for Simplification of GMO Procedures & Requirements for Investigational ATMPs in the EU

Synopsis

  • Outline of current GMO regulations for ATMPs within the EU
  • In what ways do these regulations hinder gene therapy development?
  • Latest developments to ongoing advocacy towards simplification of EU GMO legislation, as applied to investigational ATMPs

12:00 pm
Lunch & Networking

1:00 pm Case Study: Navigating Temporary Halts Through a Collaborative Approach Between Sponsors

Synopsis

  • Discuss the challenges presented with temporary halts on gene therapy clinical trials
  • How to successfully co-operate with regulatory agencies to renew proceedings
  • What impacts do these halts have on the patient rare disease community?
  • A collaborative approach: A mechanism to mitigate risks in gene therapy development programmes

A FOCUSED LOOK AT KEY REGULATORY CHALLENGES

1:30 pm Insights Gained From the Successful Launch of a Gene Therapy Product into the EU

Synopsis

  • From preclinical research to FDA then EMA approval – an overview of the milestones in the approval process for Zolgensma
  • Explore how patient safety was demonstrated to the regulators and the key challenges faced in doing so
  • The specific challenges in developing a gene therapy product for a small biotech company and how these were overcome

2:00 pm The Bespoke Gene Therapy Consortium: Exploring the Initiative

  • P.J. Brooks Acting Director, Office of Rare Diseases Research, NCATS, NIH

Synopsis

  • Outlining the structure of the Consortium and its goals
  • Understanding how to define minimal critical quality attributes through the use of animal toxicology studies
  • Exploring the long-term implications for rare disease in having an internationally harmonised approach

2:30 pm
Afternoon Break

VECTOR DELIVERY & ANALYTICAL METHODS CONDUCIVE TO SUCCESS

3:15 pm Developing a Strategy for Re-Administration of AAV Gene Therapies

  • Anne Douar Chief Development Officer, Vivet Therapeutics

Synopsis

  • Discuss the concerns surrounding immune responses to AAV vectors
  • Outline the importance of shifting away from a ‘one-and-done’ approach
  • How to develop a successful strategy to enable re-administration of gene therapies

3:45 pm Moving From Simple Capsid Ratios to Drug Product Integrity Evaluation

  • David Dobnik Senior Research Associate, NIB & Chief Scientific Officer, Niba Labs, Slovenian National Institute of Biology

Synopsis

  • Exploring the need for an industry standard analytical method for full-empty capsid ratio analysis
  • The importance of vector genome integrity and characterising AAV samples
  • Outlining early regulatory involvement and necessary conversations moving forward

4:15 pm Exploring Alternative Viral Vector Methods for Delivering Gene Therapies

  • Thijs Gerritzen Principal Scientist Process Development, Amarna Therapeutics B.V.

Synopsis

  • An overview of the immunogenicity issues surrounding AAV vectors and some different approaches to overcome this
  • How to overcome immune barriers to gene therapy delivery via a novel viral vector method
  • Discussing some of the regulatory challenges unique to developing novel viral vectors

4:45 pm Chair’s Closing Remarks