Advancing the Frontiers of Gene Therapy and Rare Diseases. Day 2- Lessons Learnt in Gene Therapy & Rare Disease Patient Engagement and Access

Dr Sheuli Porkess is Vice-President of FPM, the Director of Actaros Consultancy, an experienced pharmaceutical physician and is the former Director of Research, Medical and Innovation at the Association of the British Pharmaceutical Industry. Her career began in clinical medicine in the NHS and includes a number of medical leadership roles in companies.

Sheuli has expertise in policy development within the UK and internationally, is a Fellow of the Faculty of Pharmaceutical Medicine in the UK and a Global Fellow in Medicine Development with IFAPP.

Dr Tony Hall has over 25 years of drug development experience with ten years working specifically on rare diseases. Prior to joining Healx, he worked as CMO at Aparito and as Therapeutic Area Head of Orphan Drugs at Mereo BioPharma. He also co-designed the development programme for DevelopAKUre, an FP7-funded project to repurpose nitisinone for the treatment of the rare metabolic disease, alkaptonuria (AKU). Tony is currently Trustee of Findacure and a member of the Scientific Advisory Board at the patient group charity Duchenne UK. He is also co-editor of The Patient Group Handbook for rare diseases. Tony graduated from King’s College London with first class joint honours in physiology and pharmacology before going on to study medicine at the Royal Free Hospital.

Fleur is a health economist with over 30 years of experience in the pharma industry in clinical trials, health technology assessment and evidence generation largely at Sanofi and GSK.  Fleur is Head of Market Access UK and Ireland at Sanofi, is on the parent advisory board at Duchenne UK, and chair of Project HERCULES.

Sue Farrington is the Chief Executive of Scleroderma & Raynaud’s UK (SRUK) and Chair of the Patient Information Forum (PiF) and sits on the rare diseases forum at the NHS in this capacity. She has a broad knowledge of rare diseases and patient contact.

Due to a family bereavement Sue will not be able to make this event. Our thoughts are with her and her family during this time.

Dr Arseniy Lavrov, PhD, FFPM is a neurologist and pharmaceutical physician with 9 years of clinical practice and academia experience. He completed a PhD research project in vascular dementia and vascular cognitive impairment. Dr Lavrov has a further 16 years of pharmaceutical industry experience with a focus on drug development in Neurosciences, across all phases of development, from discovery to post approval. His experience involves translational, clinical development, marketing authorization of gene therapies for neurological diseases. His main areas of interest also include rare neurological diseases; movement disorders; dementias and cognitive impairment. Dr Lavrov has also an interest and experience in digital biomarkers and endpoints. Previously Head Unit Physician and Clinical Development Head at Neurosciences Therapy Area Unit at GSK, Dr Lavrov is leading EMEA Clinical Development at Novartis Gene Therapies since 2018 where he was extensively involved in development, regulatory approvals and access to gene therapy for spinal muscular atrophy in the region and globally.

Dr. Prasad has served as Senior Vice President and Chief Medical Officer at Audentes Therapeutics since February 2014. He is a Paediatrician with a wide range of experience and achievements in international drug development across Phase I to IV, with a specific focus on the clinical development of innovative therapies to treat rare, paediatric disorders.

Dr. Prasad has worked in medicines development in positions of increasing responsibility at BioMarin Pharmaceutical, Genzyme Corporation, and Eli Lilly and Company. He has broad therapeutic expertise in neuromuscular disease, metabolic medicine, bone metabolism, neuroscience, endocrinology, immunology, and genetics. Dr. Prasad sits on the board of the international patient organization, the National PKU Association, and is an Honorary Fellow of FPM.