Exploring the future: Opportunities and challenges in cell and gene therapy for rare diseases
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Rare disease treatment poses distinct challenges for physicians, patients, caregivers, and pharma companies. With many of these conditions lacking approved treatments, the standard care often revolves around symptom management or substitution therapies. Even when treatments are available, they are frequently costly and deliver only modest improvements.
For pharma companies, advancing new treatments is no easy task. The small population sizes and limited natural history data make clinical trial development particularly difficult, often resulting in smaller evidence bases.
Cell and gene therapy presents a potential solution to these challenges, offering the possibility to treat diseases at a cellular or genetic level. However, as promising as these therapies are, they come with their own unique set of hurdles. Misconceptions about their effectiveness and concerns about their complexity can add additional layers of uncertainty – particularly in the rare disease space, where conditions are already poorly understood.
In this webinar, sponsored by IPG Health, we will explore the role that cell and gene therapies can play in treating rare diseases, the unique challenges faced by pharmaceutical companies, healthcare professionals, and patients, and the opportunities these therapies offer.
Join us on Wednesday 5th March at 3pm GMT - 4pm CET - 10am EST for an in-depth discussion on how the future of rare disease treatment is evolving and gain valuable insights into the future of rare disease treatments and the expanding field of CGT.
Key Topics:
- The current treatment landscape for rare diseases
- Barriers to developing therapies for rare diseases
- The potential of cell and gene therapies
- Support strategies for HCPs and patients
- Looking ahead at the opportunities and challenges in CGT for rare diseases
Date and Time: Wednesday 5th March 2025 at 3pm GMT - 4pm CET - 10am EST
Don’t miss out on this opportunity to deepen your understanding of the evolving rare disease treatment landscape and the promising role of CGT. Register now to reserve your spot.
About the speakers
Dr Kristian Johnson serves as the Global Medical Lead, Cell & Gene Therapies for Ophthalmology and Rare Diseases, with Bayer Pharmaceuticals in Massachusetts, US. With almost 20 years of experience spanning clinical practice, research, and pharmaceutical industry leadership, he brings a unique combination of hands-on expertise and industry experience to his role. Dr Johnson earned his medical degree and board certification in Germany, complemented by the European Board of Ophthalmology Diploma.
Imre Varju is a medical scientist turned health communication specialist with 15+ years of healthcare experience, with the last six years focused exclusively on patient support programmes. As SVP, Director, he leads the Learning Strategy team at YuzuYello, an IPG Health Company. His team is comprised of Certified Health Education Specialists credentialled to use health behaviour models, health literacy best practices and adult learning principles to design interventions that foster patient adherence across therapeutic areas, including a range of rare disease brands and portfolios.
Lynda Chang is a Medical Strategy Director at Complete HealthVizion, an IPG Health company, with 15+ years’ experience in the medical communications space covering numerous therapy areas, including rare diseases. She oversees strategic and tactical planning, as well as delivery, across all the medical communication tactics driving delivery of publications and medical education programmes and is passionate about the “so what” of data dissemination.
Jane Blyth is a Senior Medical Strategy Director at Complete HealthVizion, an IPG Health company, with over 25 years’ experience in the pharmaceutical industry, including pharma company roles in medical affairs and publications. She also has clinical nursing experience within the UK and USA. As part of IPG Health, Blyth provides scientific and strategic leadership consultancy for pharmaceutical industry partners working to develop impactful solutions, including award-winning medical education programmes in rare diseases.
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