1.30 pm
Registration and Coffee
2.00 pm
Gene Therapy of Rare Diseases: Current Status and Future Prospect
Professor Robert Wynn, Consultant Paediatric Haematologist / Bone Marrow Transplant; Honorary Professor of Clinical Paediatric Haematology and Cell Therapies, Royal Manchester Children's Hospital
2.35pm
The Manchester Rare Conditions Centre in the Era of Personalised Medicine
Professor Siddharth Banka, Consultant Geneticist, St. Mary's Hospital
Talk synopsis:
Diagnosing, understanding, managing, and treating Rare Conditions (RCs) remains challenging. People living with RCs often require a lifetime of complex care that can profoundly impact education, financial stability, and physical and mental health. To address these unmet needs and benefit from the extraordinary regional infrastructure and expertise across the life course, the Manchester University NHS Foundation Trust and partners established the Manchester Rare Conditions Centre (MRCC) in 2021 as a virtual platform with a mission to improve the lives of individuals and families living with RCs. Since its formation the MRCC has evolved into an effective platform to link and coordinate RCs-related work in the region, break silos, facilitate cross-institutional, cross-disciplinary, cross-theme connections, and to enable the sharing of best practices. It is positioned to improve the lives of those affected by RCs by anticipating shifts in policies, scoping new opportunities, and undertaking advanced planning to develop new large-scale, high-impact projects and programmes. The work of the MRCC is enhancing efficiency, preserving, and enhancing the legacy of partners, and developing institutional memory for specialist tasks. In this talk I will focus on the '4 Ps' of rare conditions related Personalised Medicine work happening in Manchester - Prediction and Prevention of disease; Precise diagnoses; Personalised and targeted interventions; and a Participatory role for patients.
Learning objectives:
- Understanding the unmet needs of patients with rare conditions
- Structure and function of the Manchester Rare Conditions Centre
- Role of precision medicine in rare conditions
3.10pm
Break
3.40 pm
Precision Medicine in Cancer
Dr Matthew Krebs, Clinical Senior Lecturer in Experimental Cancer Medicine and Honorary Consultant in Medical Oncology, The Christie NHS FT
Talk synopsis:
This talk will cover with a brief history of how precision medicine has evolved in cancer and the relevance of DNA mutations for treatment selection. Oncology has played a leading role in implementing precision medicine in routine practice, supported by the NHS Genomics Medicine Service. Innovation in genomic testing with liquid biopsy, specifically circulating tumour DNA (ctDNA), will be covered and how this is being implemented in the NHS for the benefit of our patients. Finally, the talk will cover current state-of-the-art Precision Medicine research studies and future direction.
Learning Objectives:
- Understand relevance of genomic mutations in treatment selection for cancer patients
- Understand structure of the NHS Genomics Medicine Service for cancer patients in England
- Gain insight into future direction of travel for precision medicine in cancer with liquid biopsy and state-of-the art clinical trials
4.15pm
Advances in cystic fibrosis management
Dr Anna Shawcross, Consultant in Paediatric Respiratory Medicine, Manchester
Learning objectives:
- An understanding of the state of current cystic fibrosis (CF) clinical care including diagnosis via newborn screening, medical management of CF and current prognosis into adulthood
- An introduction to CFTR modulator drugs, including mode of action and clinical effects
- The challenges of diagnosis CF and CFTR-related disorders.
4.50pm
Coffee
5.20 pm
Annual General Meeting followed by the Presidential Address of Dr Ruth Gottstein, Consultant Neonatologist, Manchester University NHS FT
"Individualised medicine leading to advances in neonatal care"
Talk synopsis:
How we have progressed in neonatal medicine to a more individualised treatment strategy for our small and vulnerable patients.
6.10 pm
Close
This meeting has been supported by Genedrive with
Subsistence and Exhibition space only and had no involvement with the agenda or content.
*Venue directions: https://www.mandec.co.uk/contact/
MANDEC is built on the roof of the Dental Hospital and has its OWN ENTRANCE in Bridgeford Street. PLEASE NOTE: Access is NOT available through the Dental Hospital Patient Entrance – the MANDEC entrance door is to the left of this. On entering the building take the lift or stairs to the 3rd floor.
PARKING
Please note: Booth Street West car park is chargeable. Access is from HIGHER CAMBRIDGE STREET. SATNAV postcode M15 6AR. The entrance road to the multi storey car park is opposite the Trinity High School and behind the Hyatt Regency Hotel.
For further information regarding Manchester University Campus parking see link https://www.manchester.ac.uk/study/undergraduate/open-days-visits/open-days/travel/parking/