Note: The list of speakers is subject to continuous updates and additions.

Moderator

Johan Rockberg
Professor in Antibody Technology and Directed Evolution
KTH Royal Institute of Technology

As Professor in Directed Evolution and Antibody Technology, Johan Rockberg heads a research group of ten at KTH Royal Institute of Technology, Stockholm.

His research team has a strong method development focus, directed at both discovery and bioprocess development of next generation biologics, including gene therapy and immunomodulating bi-specific antibodies. To this end, directed evolution and systems biology are tools used for improvement of both compound and producer cell lines. A particular research interest is human cell homeostasis and secretional machinery during stress such as upon viral propagation or during bioreactor cultivation. He is the director of AAVNova, for the development of next generation bioproduction platforms for virus-based gene therapy.

Since 2019, Prof. Rockberg is an elected member of the Swedish Young Academy. He has been holding industry positions as Director Biosynergy at Alligator Bioscience AB, Lund Sweden, as CSO of Atlas Therapeutics AB, Stockholm Sweden and is a co-founder of Abclon Inc, Seoul South-Korea, all focused on cancer immunotherapy.

Veronique Chotteau

Enabling and Intensifying Manufacturing of Biologics

Véronique Chotteau
Professor in Mammalian Cell-Based Bioprocess Technology
KTH Royal Institute of Technology

More info coming soon!

Ulf Ståhl

In-House Development of a Protein-Based Biological Drug: A Next-Generation Botulinum Neuromodulator Product for the Aesthetic Market

Ulf Ståhl
PhD, Product Research Leader
Galderma

For more than 15 years, Galderma Uppsala has developed a fully in-house, next-generation botulinum neurotoxin product for the aesthetic market, which has since been launched and approved in several European countries and in Australia. Botulinum neurotoxin is a highly active and delicate protein molecule produced by the bacterium Clostridium botulinum. It has been used for many years in both therapeutic and aesthetic indications. Galderma’s new product is a complex-free neurotoxin, presented as a ready-to-use liquid formulation. The manufacturing process, developed by Galderma, is termed PEARL™ Technology. This process employs modern protein separation techniques in combination with single-use systems. The toxin is preserved in its natural conformation in liquid state throughout processing, thereby ensuring the high specific potency of the product.

Ulf has a PhD in plant lipid biochemistry focusing on protein purification from the Swedish University of Agriculture Sciences (SLU) followed by 10 years of research in the field of lipid biosynthesis in eucaryotic cells with about ten peer reviewed scientific publications. From 2008 Ulf has worked with process development for an aesthetic biological product within Q-Med AB / Galderma. Initially working for two years as protein purification specialist in the early discovery phase of the project and then as team leader for the Bioprocess Development team, following the project development through all project phases such as toxicology, clinical, process validation and launch of the new innovative liquid toxin product.

Knut Steffensen

Karolinska ATMP Center: Accelerating Translational Research into Clinical Application

Knut Steffensen
Director, Karolinska ATMP Center
Karolinska Institutet

Karolinska ATMP Center builds on the collective strength of both Karolinska Institutet and Karolinska University Hospital to drive academic research into clinical application and patient care. The center further aims to collaborate across the Swedish ATMP ecosystem both serving as a national infrastructure for ATMP manufacturing and a partner for both industry and academic institutions.

Dr. Knut Steffensen is a molecular medicine researcher and experienced pharmaceutical industry professional, currently serving as the Director of the Karolinska ATMP Centre in Stockholm, Sweden. The centre is a collaboration between Karolinska Institutet and Karolinska University Hospital, focusing on advanced therapy medicinal products (ATMPs). Dr. Steffensen earned his PhD in molecular medicine at University of Oslo and has a robust background in both academic research and the pharmaceutical industry. Prior to his current role, he was the Nordic Medical Lead in Cell Therapy at Kite-Gilead, where he gained extensive experience in the development and implementation of cell and gene therapies. In addition to his industry experience, Dr. Steffensen is an Associate Professor at Karolinska Institutet, contributing to the academic community through his research and teaching. His combination of academic and industry expertise effectively positions him to lead the Karolinska ATMP Centre, aiming to advance the development and implementation of innovative therapies for patients. The centre continues to strengthen its collaborations with public sector entities and pharmaceutical companies, striving to bring cutting-edge therapies from research to clinical application.

Åsa Derolf

Introducing Cell and Gene Therapies in Sweden – the Role of the New Therapies Council

Åsa Derolf
Chair
the New Therapies Council

In order to achieve an equal, cost-effective and appropriate use of new pharmaceutical drugs for all patients in Sweden, all regions, several governmental agencies and the pharmaceutical industry collaborate in a joint process for the introduction of new therapies. The New Therapies Council has the regions’ mandate to issue recommendations on the use of new therapies that are subject to national joint introduction. In recent years there has been a great development in the field of cell- and gene therapies and several of theses therapies have been introduced in Sweden through this joint national process.

Åsa Rangert Derolf is a specialist in hematology and PhD and has been head of the Department of Hematology at the Karolinska University Hospital. As a hematologist and researcher she has had an interest in acute leukemias and precision medicine. She I currently the head of the Swedish New Therapies council.

Fredrik Sjöö_fix

The Price of Innovation: How Do We Ensure Access to Gene Therapy?

New payment models are needed for life-changing rare disease therapies

Fredrik Sjöö
Head of Medical Affairs, Nordic Region
CSL Behring

The New Therapies Council (NT-rådet) recently chose not to recommend a gene therapy for hemophilia B. The cost was deemed too high – and the council highlighted uncertainty regarding how long the treatment’s effects would last.

Sweden is below the EU average when it comes to patient access to treatments for rare conditions. According to this year’s WAIT survey, patients in 20 European countries have access to more orphan drugs than patients in Sweden.

This highlights the importance of developing new payment models for therapies targeting rare and severe diseases. Outcome-based agreements – where payment is linked to a treatment’s clinical outcome – can help reduce the financial risk for payers in terms of projected spending, potential cost savings, and improved health outcomes.

Several countries have already implemented such agreements. Denmark, for instance, considers this payment model to be cost saving. During his presentation, Fredrik Sjöö, Head of Medical Affairs Nordic Region at CSL Behring, discusses how other European countries have approached this, why it is successful there, and how we can work together to ensure that new, innovative treatments benefit patients – also in Sweden.

If no one is willing to pay for gene therapies today, there will be no gene therapies tomorrow.

Fredrik Sjöö is a seasoned medical affairs leader with a clinical background in hematology and extensive experience in the pharmaceutical industry. He currently serves as the Head of Medical Affairs for the Nordic Region at CSL Behring, based in Stockholm, Sweden, where he leads strategic medical initiatives and fosters collaboration across the Nordic markets. Fredrik earned both his MD and PhD from Karolinska Institutet. Before transitioning to industry, he spent many years as a clinical hematologist and section head at St Görans Hospital in Stockholm. Fredrik is part of the Nordic launch team for Hemgenix, the first approved gene therapy for hemophilia B, contributing to its strategic planning and medical engagement across the region. His combined expertise in clinical medicine and pharmaceutical strategy positions him as a trusted partner to healthcare professionals and stakeholders.

Anna-Lena Spetz

Immune Checkpoint Induction by a Topical Oligonucleotide Therapy for Atopic Dermatitis

Anna-Lena Spetz
Chief Scientific Officer
TIRmed Pharma

TIRmed Pharma AB develops topical immunomodulatory therapies based on TIR-01, a single-stranded oligonucleotide functioning as a tolerogenic adjuvant. TIR-01 dampens TLR3-mediated inflammation, inhibits Th2/9 and Th17 responses, and induces immune checkpoints (ILT3, PDL1) on myeloid cells, restoring immune balance in inflamed skin. Delivered via the TIR-C formulation for optimized uptake, this mechanism yields rapid reduction of atopic dermatitis symptoms and durable remission with minimal treatments. Proof-of-concept animal studies confirm high efficacy and safety, supporting broad dermatological potential.

Anna-Lena Spetz, MD, PhD, Professor, has thirty years of experience from research in immunology from academia, currently at the Stockholm University and has previous experiences from Karolinska Institutet, Institut Pasteur, and Dana-Farber Cancer Institute, Harvard Medical School. She has previous experiences from a small biotech as project leader and co-founder of Avaris AB (2001-2012) and is now founder and CSO of TIRmed Pharma AB.

Gunilla Enblad

CAR-T Cells – Swedish Experiences

Gunilla Enblad
Professor in Oncology
Uppsala University

More info coming soon!

Per-Ola Carlsson_fotograf Johan Alp

Photo: Johan Alp

Transplantation of Allogeneic Beta Cells with No Immunosuppression

Per-Ola Carlsson
Professor of Medical Cell Biology
Uppsala University

We have clinically translated the concept of immune evasion with hypoimmune (HIP) gene modifications entailing the depletion of HLA class I and II and overexpression of CD47 to enable allogeneic transplantation without immunosuppression. A patient with long-standing type 1 diabetes received allogeneic, gene modified, primary donor islet cells. He did not mount any immune response against the HIP islet cells over a 26-week follow-up period. A PET-MRI at 12 weeks showed a GLP-1R signal at graft site. The patient developed stable C-peptide levels that increased in mixed meal tolerance tests. This case confirms the immune evasive character of HIP cells in a patient and provides an optimistic outlook that immune evasion might be generalizable for more cell types, tissues and organs for allotransplantation without immunosuppression.

Per-Ola Carlsson is a professor of Medical Cell Biology at Uppsala University, Sweden. He also serves as a senior consultant in endocrinology and diabetology at Uppsala University Hospital and as director of Uppsala Diabetes Centre.

His research focus over the years has been on islet physiology, islet vascular biology, stem cell research, beta-cell replacement and early phase clinical cell therapy trials in type 1 diabetes. He has authored more than 200 peer-reviewed publications and is the P.I. of the presented first-in-human study of hypoimmune islet cell transplantation in type 1 diabetes.

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