Fall Summit 2021

Fall Summit 2021

On October 21, 2021 the EFF Virtual Fall Summit will be held.

During the virtual webinar, we will be exploring the Science of RNA therapies. We are pleased to have Dr. Orla Galvin, Director of Research Policy at Retina International and Erwin van Wijk at Radboud University Medical Center join as special guests.

Together with Naveed Shams, Chief Scientific Officer of ProQR Therapeutics, they will be explaining to the audience in a comprehensive way what RNA science is, which has been the history of RNA therapies, how this technology has evolved and now being applied in clinics. We will also see the impact this science is having on the community, hopes for treatments and expectations for a future with genetic therapies. Finally, we will have a panel discussion where the experts will be answering questions from the audience.

To join the event register for EFF updates to receive the webinar details.

Program

Welcome to the webinar

Presenter: Daniel de Boer, Chief Executive Officer at ProQR
Time: 17:00 CET / 11:00 EST

What are RNA therapies?

Presenter: Naveed Shams, Chief Scientific Officer at ProQR
Time: 17:05 CET / 11:05 EST

A scientific perspective

Presenter: Erwin van Wijk, Radboud University Medical Center
Time: 17:15 CET / 11:15 EST

A community perspective

Presenter: Dr. Orla Galvin, Retina International
Time: 17:30 CET / 11:30 EST

Panel discussion

Moderator: Jennifer Pluim, VP Medical Affairs at ProQR
Time: 17:45 CET / 11:45 EST

Close out

Time: 18:30 CET / 12:30

 

Speakers & Moderator

Daniel de Boer

A profile image of Daniel de Boer, CEO of ProQR.

Daniel de Boer is the founding Chief Executive Officer of ProQR. When confronted with the diagnoses of his newborn son with cystic fibrosis, he decided to start ProQR to help patients like his son. He collected a group of experienced biotech entrepreneurs around him and in 2012 founded ProQR with co-founders Dinko ValerioGerard Platenburg and Henri A. Termeer. Daniel is an entrepreneur and passionate advocate for rare disease patients. Since founding ProQR he has assembled a group of successful biotech executives and built a team of a 150 experienced scientists and medicine developers, devoted to creating RNA therapies for patients in need. Under Daniel’s leadership ProQR has initiated clinical trials in multiple development programs for rare diseases. Before founding ProQR, Daniel was founder and Chief Executive Officer of RNA Systems, PC Basic and Running IT, companies he led through phases of growth, developing and launching several products in multiple European countries. In 2018 Daniel was named "Emerging Entrepreneur of the Year" by EY in the Netherlands.

 

Naveed Shams, MD, PhD

Naveed is the Chief Scientific Officer of ProQR. Prior to joining ProQR in 2020 Naveed was Senior Corporate Officer, Head of Global R&D and CSO at Santen, a global ophthalmology company. He has over 20 years of experience in global drug development across start-ups and large multinational companies including Novartis and Genentech/Roche. He has played a significant role in developing important ophthalmology products, including Zaditor®/Zaditen®, Rescula®, Lucentis®, and Omnidenapag. While at Genentech, Naveed helped to establish the Ophthalmology Development Group and led the development and approval of Lucentis®. Naveed received his MD degree from Dow Medical College in Karachi, Pakistan and his PhD in Microbiology and Immunology from the University of South Carolina. He completed fellowships in cornea and external diseases at Harvard Medical School and in histocompatibility and immunogenetics at Massachusetts General Hospital. Naveed was a faculty member at Schepens Eye Research Institute and Department of Ophthalmology, Harvard Medical School.

 

Erwin van Wijk, PhD

Portrait of Erwin van Wijk

Dr. Erwin van Wijk is an Assistant Professor at the Department of Otorhinolaryngology and the Donders Institute for Brain, Cognition and Behaviour (Radboud University Medical Center, Nijmegen, The Netherlands). He is head of the Usher Syndrome & Hereditary Hearing Loss Therapeutics Research Unit. From 2012 onwards he is exploring different therapeutic strategies to develop a genetic therapy for USH2A-associated retinal degeneration thereby employing the zebrafish model for evaluating therapeutic efficacy. Using the method of antisense oligonucleotide-based splice modulation he obtained pre-clinical proof of concept for the redirection of aberrant splicing caused by a common deep-intronic mutation in the USH2A gene. Furthermore he explored the use of antisense oligonucleotides for the purpose of skipping of the most frequently mutated exon in the USH2A gene, for which a phase 1/2  clinical trial was just successfully completed. The current work of his research team is dedicated to better understand the molecular mechanisms underlying different clinical types of Usher syndrome, non-syndromic inherited retinal dystrophies and inherited hearing impairment, and to directly apply that knowledge for the development of new treatment strategies for the many visually and hearing impaired individuals worldwide.

 

Dr. Orla Gavin

Portrait of Orla Gavin

Dr. Orla Galvin is the Director of Research Policy at Retina International. Orla has led a first-in-kind pilot study in Ireland and the UK, to better understand the impact of Inherited Retinal Disease on patients and families in these countries through literature review, and patient and carer survey. The learnings from this work have proven invaluable and have guided Orla in directing additional projects assessing the patient/carer and clinical perspective in other retinal disease areas in other countries.

Orla serves on the organisation committee of the Retina Youth Programme for the Retina International World Congress 2022, and acts as an advisor to the Retina International Youth Council.

Orla believes in continuous education and raising awareness of retinal conditions. In 2017 Orla co-ordinated the University College Dublin Inherited Blindness Summer School; and in 2018 she designed and developed the content for the Diabetes-related Eye Disease toolkit www.retina-ded.org which launched in January 2019. This educational site aims to provide timely, relevant and accurate information to patients and their families and professionals (Eye Care Professionals, Advocates, etc.) on Diabetes-related Eye Diseases (DEDs).

Orla is co-ordinator and moderator for the CME accredited Retina International ARVO 2020 education course on patient data “Patient Data: Recognition, Evaluation, Incorporation and Practice”. Orla took part in the first ever ARVO online event “Artificial Intelligence in ocular medicine: Seeing into the future” and is developing an education tool to create awareness of the advances and benefits AI can bring to the patient and to health care systems across the globe.

Orla came to Retina International with a background in academic and industry vision research. Orla is a Pharmacology graduate from University College Dublin with a PhD in Medicine from Queen’s University Belfast. Orla has won the Geraldine Duggan Young Investigator award and the SFI/TIDA venture training programme award.

During her postdoctoral career, including Wellcome Trust, and Marie Curie Fellowship positions, Orla’s research interests included drug discovery, design and delivery in academic and industry settings in Ireland and the UK. Orla continues to build upon her background in ocular therapy development, education, and inter-sector communication through Retina International, and believes optimal care and progress can be achieved through a multi-stakeholder approach.

 

Jennifer Pluim

Jennifer Pluim

Jennifer Pluim is the VP and head of Medical Affairs at ProQR Therapeutics. She is responsible for building strong Medical Affairs capabilities to help prepare ProQR for the successful launch of Sepofarsen, and all subsequent launches, with an emphasis on how to best serve patients and the medical community.  Jennifer has over 25 years of experience in the pharmaceutical industry, with extensive knowledge in clinical operations, project management and medical affairs.  Her experience spans several therapeutic areas, where she has been involved in developing the medical affairs strategy of therapeutic targets in infectious diseases, respiratory critical care, cardiovascular disease, oncology and ophthalmology. For 8 years prior to joining ProQR, she had a leadership role at Bayer in developing the medical strategy for the launch and lifecycle management of Eylea®- an anti-VEGF therapy for the treatment of retinal diseases.  During her career, she has had the opportunity to work for both big pharma as well as start-up biotechnology companies, where she has built and led medical affairs operations and infrastructure, focusing on providing data to meet the needs of patients and healthcare practitioners.

 

Contact & questions

If you wish to ask a question to the panelists, you can email the EFF team (EFF@proqr.com). You can also submit your questions anonymously using the form below.

The EFF team will collect all questions so they can be discussed with the experts during the panel discussion.