Gene editing medicines

07 - 08 November 2024 09:00 - 17:00 Online Free Watch online
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Abstract 3d DNA molecule helix on a blue background

This meeting will explore the innovations that are transforming the future of gene editing medicines.

Just 12 years after the discovery of CRISPR/Cas9, the first gene editing medicines have been approved and many more are in clinical development. This meeting will explore the innovations that are transforming gene editing medicines and their impact on patient health.

The potential applications of gene editing technologies for treating diseases are beginning to be realised. The science underpinning gene editing medicines continues to develop at a rapid pace, and clinical applications are growing. However, challenges in terms of manufacturing, scale-up, regulation, equitable access, health economics and safety make the widespread roll-out of these medicines difficult. 

This two-day conference will discuss recent advances, future priorities, and the wider social and economic context for the application of gene editing approaches in a healthcare setting.

Transforming our future conferences

This conference forms part of the Royal Society's industry-focused Transforming our future series. These unique meetings feature cutting-edge science and bring together experts from industry, academia, healthcare settings, funding bodies, the wider scientific community and government to explore and address key scientific and technical challenges of the coming decade.

Organisers

  • Steve Rees

    Steve Rees

    Steve Rees is Senior Vice-President of Discovery Sciences at AstraZeneca with responsibility for the discovery of novel drug candidates, using multiple therapeutic modalities, for projects for all AstraZeneca therapy areas. Prior to his current appointment Steve held positions of increasing responsibility at AstraZeneca including leadership of the Discovery Biology and Screening Sciences and Sample Management departments. He has served as Chair of the European Laboratory Research and Innovation Group, Chair of the European Council of the Society of Laboratory Automation and Screening and as Industry Trustee of the British Pharmacological Society and is a member of  the Royal Society Science, Industry and Translation Committee. Steve was awarded an OBE by Her Majesty the Queen in 2021 for services to science and the COVID19 response.

  • Laura Sepp-Lorenzino

    Laura Sepp-Lorenzino

    Laura Sepp-Lorenzino, Ph.D. joined Intellia Therapeutics in 2019 as Chief Scientific Officer and is responsible for Research and Early Development. Intellia is harnessing CRISPR-based technologies to revolutionize the future of medicine. Laura previously held leadership positions at Vertex, Alnylam and Merck. She serves on the Board of Directors of Taysha Gene Therapies and the Alliance for Regenerative Medicine, and on the Scientific Advisory Boards for Thermo Fisher Scientific, the U.K. Nucleic Acid Therapies and Arsenal Capital Partners. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina, and both her M.S. and Ph.D. in Biochemistry from New York University.

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    Professor Robin Ali

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    Professor Robin Lovell-Badge

Schedule

09:00-09:05 Welcome and opening remarks
Professor Dame Linda Partridge

Professor Dame Linda Partridge

Institute of Healthy Ageing, University College London

09:05-09:25 CRISPR/Cas9-based in vivo gene editing for transthyretin amyloidosis
Professor Julian Gillmore

Professor Julian Gillmore

UCL

09:25-09:30 Q&A

Chair

Steve Rees

Steve Rees

AstraZeneca

09:30-09:35 Introduction to Session 1
Steve Rees

Steve Rees

AstraZeneca

09:35-09:55 CRISPR
Marcello Maresca

Marcello Maresca

AstraZeneca

09:55-10:15 Epigenome editing
Angelo Lombardo

Angelo Lombardo

SR-TIGET

10:15-10:35 Novel technology
10:35-10:50 Q&A

Chair

Laura Sepp-Lorenzino

Laura Sepp-Lorenzino

Intellia Therapeutics

11:20-11:25 Introduction to Session 2
Laura Sepp-Lorenzino

Laura Sepp-Lorenzino

Intellia Therapeutics

11:25-11:40 Cell engineering
Birgit Schultes

Birgit Schultes

Intellia Therapeutics

11:40-11:55 Lentiviral systems
Luigi Naldini

Luigi Naldini

SR-TIGET

11:55-12:10 Virus-like particles
12:10-12:25 Adeno-associated virus vectors
Professor Robin Ali

Professor Robin Ali

King's College London

12:25-12:40 Lipid nanoparticle systems
12:40-12:55 Q&A

Chair

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Professor Robin Lovell-Badge

The Francis Crick Institute

14:00-14:05 Introduction to panel
Professor Robin Lovell-Badge

Professor Robin Lovell-Badge

The Francis Crick Institute

14:05-15:10 Panellists
Sharmila Nikapota

Sharmila Nikapota

Cure EB

Chair

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Professor Waseem Qasim

UCL

15:40-15:45 Introduction to session 3
Professor Waseem Qasim

Professor Waseem Qasim

UCL

15:45-16:05 Therapeutic gene editing for cardiovascular and metabolic diseases: from the leading cause of death to N-of-1 disorders
Professor Kiran Musunuru

Professor Kiran Musunuru

University of Pennsylvania

16:05-16:25 Talk 2
Benit Maru

Benit Maru

SSI

16:25-16:45 Talk 3
Dr Suk See De Ravin

Dr Suk See De Ravin

National Institutes of Health

16:45-17:05 Talk 3
Professor Waseem Qasim

Professor Waseem Qasim

UCL

17:05-17:20 Q&A
09:00-09:05 Welcome and opening remarks
Professor Robin Ali

Professor Robin Ali

King's College London

09:05-09:30 Opening keynote
Professor Fyodor Urnov

Professor Fyodor Urnov

University of California, Berkeley

Chair

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Professor Robin Ali

King's College London

09:30-09:35 Introduction to session 1
Professor Robin Ali

Professor Robin Ali

King's College London

09:35-09:55 Talk 1
09:55-10:15 Talk 2
Christopher Vann

Christopher Vann

Autolus Ltd

10:15-10:35 Talk 3
Vanessa Almendro

Vanessa Almendro

Danaher

10:35-10:50 Q&A

Chair

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Professor Robin Lovell-Badge

The Francis Crick Institute

11:20-11:25 Introduction to session 2
Professor Robin Lovell-Badge

Professor Robin Lovell-Badge

The Francis Crick Institute

11:25-11:45 Talk 1
Stephanie Krogmeier

Stephanie Krogmeier

Vertex

11:45-12:05 Talk 2
Dr Sam Roberts

Dr Sam Roberts

NICE

12:05-12:25 Precision in Progress: Assessing and Mitigating Genotoxic Risks in Genome Editing
Professor Toni Cathomen

Professor Toni Cathomen

University of Freiburg

12:25-12:40 Q&A

Chair

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Professor Robin Ali

King's College London

13:40-13:45 Introduction to session 3
Professor Robin Ali

Professor Robin Ali

King's College London

13:45-14:05 Talk 1
Professor Bobby Gaspar

Professor Bobby Gaspar

Orchard Therapeutics, UCL

14:05-14:25 Talk 2
Stefano Benvenuti

Stefano Benvenuti

Fondazione Telethon

14:25-14:45 Talk-3
14:45-15:00 Q&A

Chair

Steve Rees

Steve Rees

AstraZeneca

15:30-15:35 Introduction to panel
Steve Rees

Steve Rees

AstraZeneca

Chair

Dame Sue Ion GBE FREng FRS

Dame Sue Ion GBE FREng FRS

Chair of the Royal Society Science, Industry and Translation Committee