Accelerating Development of Gene & Cell Therapy

From the Lab to Patient Access: A Deep Dive into Advanced Therapy Medicinal Products

The International Training Course will be organised through Symmetric s.r.o. ORPHA Strategy's Principal, David Schwicker, will be the Trainer


Online Training Course, 4-6 April 2022

13:00 - 17:00 CET, 7:00 - 11:00 EDT


Tailored In-house Trainings Upon Request

Download the Complete Course Agenda:

ATMPs Digital Final Agenda.pdf
Adobe Acrobat Document 477.2 KB

To Register & for Further Information, please contact: 

Lydia Makori | Business Development Specialist at Symmetric

T +421 222 200 543 | Email 

Symmetric s.r.o. | Mliekarenska 9 |82109 Bratislava | Slovak Republic | Office: +421 948 262 346

"There are actually over 280 programs in cell & gene therapy. Not all of those will work.
A lot of that comes down to the ability of companies to move the lab-based processes to commercial
and manufacturing processes. And that takes a level of investment that is not insignificant.
Who is able to actually master the technology and bring these processes to market?
So we'll have a lot of great clinical results, a lot of great clinical promise coming through and
readouts from these programs. But not every program will be ready to move to the commercial stage
because of the required investment in meeting the FDA requirements for manufacturing."
David Lennon, President AveXis (Novartis)"

The existing pipeline of cell & gene therapies is expected to lead to 40-60 launches, with 15-30 launching within the next five years, and around 500,000 patients in the United States treated with 40-60 products by 2030 (source: FoCUS, an MIT NEWDIGS Project, October 2018). These therapies represent an unprecedented opportunity for patients and caregivers, potentially offering cures in numerous complex conditions that previously had inadequate treatments, were extremely debilitating and life-threatening. In addition to manufacturing requirements, the intense debate on how to value, price and pay for cures challenges the successful commercialisation of ATMPs. Reasons enough to invest time to investigate the entire value chain of gene & cell therapies.


Learning Objectives


This highly interactive training has been designed to teach participants specifically about autologous and allogeneic gene therapies, tissue engineered products and somatic cell therapies.

  • Explore the opportunities of digital and big data to accelerate discovery, development, regulatory approval and market access
  • Gain insight into the most recent methodologies for faster, smaller, smarter clinical trials, including Phase I/II studies that are robust enough for marketing application
  • Discuss strategies to limit the number of patients and the placebo exposure of participants in pivotal clinical trials in rare and ultra-rare conditions
  • Discover the newest regulatory approaches for gene and cell therapies
  • Consider the crucial role of patient engagement throughout the ATMP lifecycle
  • Analyse the current market access challenges for ATMPs as well as innovative strategies that integrate patient-centric real-world evidence

Who should attend?


Heads of R&D, Global program leads, Regulatory executives, C-level executives, Senior business development strategists, Portfolio executives, Pricing and market access executives, Medical affairs executives, Marketing and Commercial executives, Finance executives, Patient advocates, Epidemiologists, Outcomes researchers, Health economists, Digital health specialists, Big data and RWE researchers.


This International Training is for professionals who want to take a deep dive into the opportunities and challenges of the expanding business of Advanced Therapy Medicinal Products (ATMPs).


It is also for those who want to learn more about the 6-7.000 rare and ultra-rare conditions, 80% of which have identified genetic origins, making them potential targets for gene therapies. The 50-60 million individuals living with rare conditions of genetic origin represent a huge unmet medical need, which is often life-threatening, highly debilitating and requires great urgency.


The cross-cutting theme of this masterclass is therefore “fast to patient”: the acceleration of discovery, development, authorisation, manufacturing and market access in order to ensure the timely access to potentially curative treatments, contributing to the survival and improved quality of life of individuals affected by severe and complex conditions.

"Successful commercialisation of Advanced Therapies requires the integration
of development, regulatory, manufacturing and market access strategies.
The intersection point of these strategies is proposed as the
joint vision of earliest patient access"


Real-Life Case Studies


Throughout the three days, delegates will discuss, develop and brainstorm strategies to address ATMP challenges based on the presentation of current case studies.

  • Somatic cell therapy orphan designation and orphan exclusivity at marketing authorisation
  • Single-arm open label clinical studies supported with external control data
  • The first randomised gene therapy trial - delayed start crossover design with real-world controls
  • CAR-T-cell success stories and caveats: regulatory strategy and post-authorisation commitments, manufacturing and commercialisation challenges, pricing controversy
  • Autologous vs allogeneic - strategies needed with donor-derived therapies
  • Gene therapy rare disease registry for the long-term follow-up of patients
  • Master protocols and platform trials - are collaborative approaches the future?
  • MAA/BLA and early approval with first-in-human phase 1/2 study data
  • Regulatory review based on the “totality of the evidence” and “on a per-subject basis”
  • Targeted immuno-oncology market access with accelerated oncology approvals (based on Phase 2 proof of concept data)
  • Differentiating gene therapy from RNA therapy: Zolgensma (in vivo gene therapy) vs Spinraza (RNA oligonucleotide therapy) in Spinal Muscular Atrophy (SMA)
  • Comparative effectiveness studies in a hyper-competitive marketplace

Deep-Dive Interactive Workshop


A deep-dive into Voretigene Neparvovec-rzyl (Luxturna) in RPE65 Mutation Associated Inherited Retinal Dystrophy (IRD) – adeno-associated viral vector (AAV) Gene Therapy: clinical development, regulatory strategy, market access and pricing in the US and Europe.*


* Delegates will receive selected pre-reading materials to familiarize themselves with these topics before the course. Note that all the information presented in the case examples and the deep dive workshop is based on publicly available scientific papers, articles and press releases.