Future trends in Rare Disease drug development

Laura Iliescu has 20 years of experience in development and commercialisation of therapies, and patient-centred healthcare services. She has held strategic development roles in leading pharmaceutical, biotechnology, CRO and clinical homecare organisations throughout every phase of the product lifecycle from preclinical to beyond patent expiry. She has deep experience in rare metabolic, neuromuscular and respiratory indications among others. 

Within ICON’s Center for Rare Diseases, Ms. Iliescu advises clinical-stage rare disease focused companies on patient-centered clinical strategy to optimise both study efficiency and the experience of patients and caregivers. Ms. Iliescu holds a Master of Human Factors and Ergonomics from the University of Nottingham.

Dr. Tai joined Avidity in September of 2020 as an Executive Director in Clinical Development and has led the clinical development of del-desiran through it phase 1/2 trials. She has worked in the life sciences industry for over a decade as a medical monitor and clinical developer on phase 1 to phase 3 interventional trials in the areas of cardiovascular outcome, metabolic, and neuromuscular diseases. This include supporting the development and approval of Tegsedi® for the treatment for a rare genetic disease transthyretin polyneuropathy as well as the development of its 2nd generation compound eplontersen. 

Prior to that, Li focused on basic science research in the field of atherosclerosis and metabolism and took care of patients as an attending physician at the San Diego VA hospital. Li attended Northwestern University in the medical scientist training program for her MD. PhD degrees.

Daniel Leonard is the Executive Director of Global Patient Advocacy at uniQure, a pioneering biotechnology company developing gene therapies for rare diseases. Dan launched the Patient Advocacy function at uniQure and led Patient Advocacy activities for the Prix Galien winning product Hemgenix, the first ever gene therapy for hemophilia B. uniQure has an active clinical trial in Huntington’s disease, and Dan works closely with HD patient organisations to help educate the community on gene therapy and uniQure’s program. He serves as an ambassador to the patient community and works to ensure that everyone at uniQure understands the unique challenges and needs of the HD community. 

Dan has spent over 24 years working in the biotech industry. He recently joined the Termeer Foundation as an Advisor. Prior to joining uniQure, he spent 16 years at Genzyme, a company known as a leader in patient centricity. He is now a frequent speaker at conferences on the topic of patient advocacy with a focus on the gene therapy space. Outside of work, Dan like to spend time with his wife, their two daughters, and their pesky rescue mutt named Blue. He is a published short story writer, filmmaker, and avid triathlete.

Michelle joined the International Fibrodysplasia Ossificans Progressiva Association (IFOPA) as Executive Director in February 2016. The majority of her career has been spent working in the non-profit sector; seven years of which were spent leading a non-profit chamber of commerce training and consulting with local non-profit staff, boards of directors and volunteers. In that role, Michelle was exposed to many deserving causes, but her passion has always been in working with patient advocacy organisations, including tenures at the National Kidney Foundation and Polycystic Kidney Disease (PKD) Foundation. While at the PKD Foundation, Michelle became acquainted with the rare disease community which is part of what drew her to the IFOPA. 

At the PKD Foundation, Michelle served in the role of Chief Development Officer where she worked in all aspects of fundraising and maintained the organisation’s relationships with industry. Michelle also led the organisation’s education and advocacy initiatives, including organising patients for an FDA Advisory Committee meeting for the review of the first-ever treatment for PKD, Jynarque. At the IFOPA, Michelle leads an eight-member staff team and partners with the Board of Directors to execute the organisation’s strategic plan. She leads industry partnerships and heavily supports real-world evidence and patient insights programs. Every other year she leads the FOP Drug Development Forum, the only scientific conference dedicated to drug discovery and foundational research in FOP. Michelle also manages clinical studies and trials education programs for the IFOPA.

Mr. Nathan Peck is the co-founder and CEO of Cure VCP Disease, Inc., a patient 
advocacy organization dedicated to driving a cure for valosincontaining protein (VCP) associated multisystem proteinopathy (MSP), also known as IBMPFD (Inclusion Body Myopathy, Early Onset Paget’s Disease of Bone and Frontotemporal Dementia). The disease is an adult onset, hereditary, autosomal dominant disease caused by a mutation of the Valosin Containing Protein gene on chromosome 9. It can affect any combination of a patients’ muscles, bones and brain.

Mr. Peck is a patient with the disease and was officially diagnosed in 2015. His mother, aunt and one uncle are deceased from the disease and he has one uncle still living with the disease. Mr. Peck is retired, on full long-term disability and has quickly grown the awareness and influence of Cure VCP Disease among the research and patient community since 2018. Cure VCP Disease was selected to the Chan Zuckerberg Initiative (CZI) Rare as One (RAO) project in November 2021.