TOPRA 2025

CASE STUDY Future therapies realised today

Regulatory scientists like Michela Gabaldo are at the forefront of delivering the potential of gene therapies. After graduating from Padua University, Italy, with a degree in pharmaceutical chemistry and technology she joined GlaxoSmithKline in 2001 and worked in clinical operations and CMC (chemistry manufacturing controls) regulatory affairs.

Ten years later, she was managing gene therapy projects at Alliance Management and Regulatory Affairs at Fondazione Telethon, Milan, Italy. She made a key contribution to the clinical development and EU marketing approval of Strimvelis (GSK), an autologous gene therapy for the treatment of an ultra-rare severe combined immunodeficiency (ADA-SCID), which was the first ex vivo gene therapy approved in the world. She is also a winner of the Futures category of the TOPRA Awards for Regulatory Excellence, in recognition of her contribution of ‘innovative ideas and solutions to overcome the regulatory hurdles that are typically associated with a highly innovative medicine as gene therapy for a rare disorder’. Dr Gabaldo is now Head Alliance Management & Regulatory Affairs Manager at SR-TIGET San Rafaele Telethon Institute for gene therapy, Milan, Italy and a member of the International Rare Diseases Research Consortium (IRDiRC) Therapies Scientific Committee.

Michela Gabaldo

Preparing regulatory professionals for expanded roles

There are nowmany different career routes and specialties open to the modern regulatory professional; expectations for skills, knowledge and competencies have changed radically in the 21st century. TOPRA offers relevant training and professional development to enable regulatory professionals at all levels to pursue these options and achieve their potential. Academic preparation For more than 30 years, TOPRA has offered a postgraduate specialist regulatory qualification. It continues to adapt to regulatory needs and expectations. In 2017, the TOPRA MSc Regulatory Affairs programme was radically updated and expanded with the University of Hertfordshire, offering two pathways – Medicines andMedical Devices. Students on either pathway can now choose up to two modules from the other – an innovation that reflects the increasing number of professionals working with combination products or needing in-depth knowledge of a particular area,

eg, data management and digitisation. Newmodules and Masterclasses are being considered and developed, eg, the regulatory aspects of developing advancedmedicinal therapy products (ATMPs). A number of regulatory professionals are studying for a PhD with TOPRA. Adding to the body of knowledge, their research will help to underpin future development of regulatory science. Soft skills Regulatory knowledge and expertise are not the only requirements to build a successful career. To be a truly effective regulatory professional, individuals need to draw on a toolbox of practical, soft skills to manage their expanded roles. Communications and project management are key areas for professional development. Such needs are being met by an increasing range of CRED and online courses and webinars, covering document writing, regulatory operations, digitisation, etc.

TOPRA 2025 – Aspire | Adapt | Achieve 16