Improving Red Blood Cell Production and Iron Balance After Gene Therapy in Transfusion-Dependent Beta-Thalassemia

Sujit Sheth received grants or contracts from BMS/Celgene, Forma/Novo Nordisk, Agios, Regeneron, and Pharmacocosmos; consulting fees from Agios, Novo Nordisk, Beam Therapeutics, Chiesi, BMS/Celgene, and Vertex Pharmaceuticals Incorporated; honoraria from Plexus, CCO, and PER; travel support from Agios, BMS/Celgene, and Chiesi; and participated in an advisory board for Vertex Pharmaceuticals Incorporated. Selim Corbacioglu is an advisory board member for Vertex Pharmaceuticals Incorporated. Josu de la Fuente is an advisory board member for Vertex Pharmaceuticals Incorporated, Sanofi, MAAT Pharma, and Sangamo; received honoraria from Vertex Pharmaceuticals Incorporated. Mattia Algeri is an advisory board member for Vertex Pharmaceuticals Incorporated. Kevin H.M. Kuo received consulting fees from Agios Pharmaceuticals, Alexion Pharmaceuticals, Biossil, Bristol Myers Squibb, Novo Nordisk, Vertex Pharmaceuticals Incorporated, Pfizer, and Forma Therapeutics; honoraria from Agios Pharmaceuticals, Alexion Pharmaceuticals, Bristol Myers Squibb, and Vertex Pharmaceuticals Incorporated; research funding from Agios Pharmaceuticals and Pfizer; and is an advisory board member for Agios Pharmaceuticals and Sangamo. Ami J. Shah received consulting fees from Vertex Pharmaceuticals Incorporated, Sangamo Therapeutics, Editas Medicine, and Medexus Inc.; research funding from CRISPR Therapeutics. Hayley Markeley received consulting fees from Novartis, Vertex Pharmaceuticals Incorporated, Novo Nordisk, Takeda, Bristol Meyers Squibb, Amgen, Sobi, Medison, Chiesi, and Recordati. Ben Carpenter serves on a scientific advisory board for Vertex Pharmaceuticals Incorporated; received honoraria from Bluebird Bio. Markus Y. Mapara received consulting fees from Bluebird Bio, CRISPR Therapeutics, Incyte Corporation, and Ossium Health; advisory board member for Bluebird Bio, CRISPR Therapeutics, and Incyte Corporation. Stephan Grupp received consultancy fees from Novartis, Jazz, Adaptimmune, Vertex Pharmaceuticals Incorporated, Allogene, and Cabaletta; honoraria from Novartis; research funding from Novartis, Kite, Servier, Jazz, Cellectis, and Vertex Pharmaceuticals Incorporated; advisory board member for Allogene. Amanda M. Li is an advisory board member for Novartis Canada. Janet L. Kwiatkowski is on advisory boards for Agios Pharmaceuticals, NovoNordisk, Chiesi USA, and Silence Therapeutics; participated in clinical trials for Bioverativ Therapeutics, Bluebird Bio, Sangamo, and Vertex Pharmaceuticals Incorporated; consultant to Agios Pharmaceuticals, BioMarin Pharmaceuticals, Celgene Corporation, Chiesi USA, Forma Therapeutics, Regeneron Pharmaceuticals, Silence Therapeutics, and Vertex Pharmaceuticals Incorporated. Sakellarios Zairis, Tina Liu, and William Hobbs are employees of Vertex Pharmaceuticals Incorporated and may own stock or stock options in the company. Franco Locatelli serves on the scientific advisory board of Amgen, Novartis, Sanofi, Vertex Pharmaceuticals Incorporated; speaker's bureau for Miltenyi Biomedicine, Amgen, Novartis, Bristol Myers Squibb, Gilead, MEDAC, SOBI. Roland Meisel received research funding from Jazz Pharmaceuticals, Miltenyi Biotec, Neovii Biotech, Novartis Pharma, Celgene BMS, Gilead/Kite Pharma, CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated, all provided to institution; consulting fees from Novartis Pharma, Medac, Celgene BMS, Bluebird Bio, Beam Therapeutics, CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated. Haydar Frangoul received consulting fees from Vertex Pharmaceuticals Incorporated, Editas Medicine, BioLineRx and Rocket Pharma; honoraria from Jazz Pharmaceuticals; advisory fees from Rocket Pharmaceuticals; served in a leadership or fiduciary position at Vertex Pharmaceuticals Incorporated. Antonis Kattamis received consulting fees from Agios Pharmaceuticals, Bristol Meyers Squibb, Novo Nordisk, and Vertex Pharmaceuticals Incorporated. Peter Lang, Joachim Rupprecht, Robert I. Liem, Yogi Chopra, Melanie Kirby‐Allen, and Maria Domenica Cappellini have nothing to disclose.