Pfizer joined Novartis as the market leader in sickle cell disease when it acquired Global Blood Therapeutics last year. The US giant's Oxbryta may now move ahead following a clinical and regulatory setback for the Swiss major's Adakveo.

Investigational drugs from Outlook Therapeutics and Vertex/CRISPR could become first-of-their-kind products if they win marketing approval.

Editas is seen as a slow developer in the CRISPR-based therapeutics field compared with its rivals but its new CEO has unveiled a new strategy to speed progress.

From a scientific perspective, the outlook for patient with rare disease has never been brighter. This year, 13 new cell or gene therapies could be approved in the US, Europe or both by the end of 2023. However, the challenges that stop patients accessing new therapies remain. Could this year be the turning point when our health care systems start to catch up with our science?