Advancing the Frontiers of Gene Therapy and Rare Diseases. Day 1- Lessons learnt in Gene Therapy & Rare Disease Development

Dr Zoya Panahloo a physician by background, is currently Senior global Medical Director in rare neurological diseases at UCB and Chair of the FPM Rare Disease and Gene Therapy Expert Group of FPM. She has over 15 years’ experience in both large Pharma and small biotech, specifically in Global Medical Affairs roles and in rare neurological Diseases area.

Professor Alan Boyd is CEO of Boyd Consultants Ltd and ex-President of FPM. He is a Pharmaceutical Physician who has made leading contributions to developing medicines in industry, facilitated numerous drug development programmes with academics, and driven the recognition for the new specialty of Pharmaceutical Medicine. His industry work developed multiple products that are now approved medicines for a wide range of indications. He is globally recognised as a pioneer in gene-based therapies, having led the development of the first gene therapy to be submitted for regulatory approval and being involved in five of the eight advanced therapies that are now approved medicines.

Innovative trial designs and regulatory approaches in rare diseases – how to maximise chances of success

During this presentation I will talk about the Gene Therapy based products that have received regulatory approval to date as prescription medicines, together with the issues that were encountered and overcome. I will also describe some points to consider in the clinical trial designs for such products and the regulatory issues that we are currently facing.

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.

This talk will cover some of the challenges of using basket trials (insufficient power for the individual diseases; validity of extrapolating results of the “basket” to the individual diseases within it; lack of sufficient safety data on individual diseases; practical regulatory issues such as orphan designation & how to apply the incentives; lack of a regulatory paradigm for approval), biomarkers (why they might be required and challenges related to regulation), and the use of complex clinical endpoints (why standard clinical outcome measures may not be suitable for (some) RDs). Real-life examples (and solutions) will be given.

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.

Considerations on Gene Therapy development: successes and challenges

The session will provide an overview of development of gene therapies including key factors to be taken into account for various aspects of development strategies. The presentation will also focus on successes and challenges illustrated by experience from development program of an approved gene therapy.

Dr Suyash Prasad is a Paediatrician, Clinical Development Physician, Translational Scientist and Executive Leader with 20 years in the biopharmaceutical industry. He currently serves as the Chief Medical Officer and Head of Research and Development at Taysha Gene Therapies, an organisation dedicated to developing AAV gene therapy approaches for treating children and adults with severe and rare neurological disease

Suyash graduated in medicine at the University of Newcastle upon Tyne, UK where he received commendations for Paediatrics, Obstetrics and Gynaecology, and Medical Ethics. His paediatric training was completed at recognised centres of excellence in the UK and Australia before he moved to industry. His industry career progressed at Eli Lilly, Genzyme, and BioMarin, and more recently he was Chief Medical Officer at the gene therapy company Audentes Therapeutics (now Astellas Gene Therapies), before moving to Taysha.

Suyash has dedicated his career to the well-being and advocacy of children who are afflicted by rare and severe disease.

What are the lessons we have learnt so far in this newly developing field?

This presentation will touch on some of the lessons that we have learnt in recent years in the rapidly evolving field of gene therapy approaches to treating rare and severe disease. Some of the topics that will be considered include the importance of high quality manufacturing, the immunology of gene therapy and how to manage the occurrence of immunological consequences, and understanding how best to translate pre-clinical data into planning for a clinical trial. It will also touch on the current gene therapy financing and market environment