KMM reports consultancy fees from Novartis, Bristol Myers Squibb (Celgene Corp), Agios Pharmaceuticals, CRISPR Therapeutics, Vifor Pharma, Novo Nordisk and Pharmacosmos; and research funding from Agios Pharmaceuticals and Pharmacosmos. SS reports consultancy fees from Agios Pharmaceuticals, Bristol Myers Squibb and Novo Nordisk; being a member of a clinical trial steering committee for Vertex Pharmaceuticals; and research funding (for clinical trials) from Agios, Bristol Myers Squibb, Novo Nordisk and Regeneron. MDC reports consultancy fees from Novartis, Bristol Myers Squibb (Celgene Corp), Vifor Pharma and Vertex Pharmaceuticals; and research funding from Novartis, Bristol Myers Squibb (Celgene Corp), La Jolla Pharmaceutical Company, Roche, Protagonist Therapeutics and CRISPR Therapeutics. KHMK reports consultancy fees from Agios Pharmaceuticals, Alexion Pharmaceuticals, Bristol Myers Squibb, Forma Therapeutics, Pfizer, Novo Nordisk and Vertex Pharmaceuticals; honoraria from Bristol Myers Squibb; membership on an advisory committee for Agios Pharmaceuticals and Bioverativ/Sanofi/Sangamo; and research funding from Agios Pharmaceuticals and Pfizer. SR reports being a member of the scientific advisory board for Ionis Pharmaceuticals; has acted as a consultant for Disc Medicine, Protagonist Therapeutics, La Jolla Pharmaceutical Company, Meira GTx, GSK, Bristol Myers Squibb, Incyte, Cambridge Healthcare Res, Celgene Corporation, Catenion, First Manhattan Co., FORMA Therapeutics, Ghost Tree Capital, Keros Therapeutics, Noble insight, Sanofi Aventis U.S., Slingshot Insight, Spexis AG, Techspert.io, BVF Partners L.P., Rallybio LLC, venBio Select LLC, ExpertConnect LLC and LifeSci Capital; and is a co-inventor for patents US8058061 B2 C12N 20111115 and US7541179 B2 C12N 20090602. VV reports consultancy fees from Bristol Myers Squibb (Celgene Corp). AM reports being a member of advisory boards for Novartis, Bristol Meyers Squibb, Bluebird Bio and Vertex Pharmaceuticals; and research funding and travel support from Agios Pharmaceuticals. VV reports consultancy fees from Bristol Myers Squibb (Celgene Corp). ATT reports consultancy fees from Novo Nordisk, Bristol Myers Squibb (Celgene Corp), Agios Pharmaceuticals, Pharmacosmos and Roche; and research funding from Novo Nordisk, Bristol Myers Squibb (Celgene Corp), Agios Pharmaceuticals, Pharmacosmos and Roche. All competing interests are outside the present work.