Nowadays, for as much as 95% of the 7 000 to 10 000 rare diseases that exist, there are no approved therapies. Of the estimated 400 M people worldwide afflicted with a rare disease, 50% are children. Cell and gene therapies have emerged as a new treatment paradigm for many rare diseases. These novel medicines have the potential to provide long-term therapeutic benefits for patients, however their development is both risky and cost intensive.

As of the end of 2020, there were 1220 ongoing regenerative medicine and advanced therapy clinical trials and 1085 gene- and cell-based therapeutics companies working worldwide to
develop therapies. These companies raised a cumulative amount of $ B in 2020, a twotimes increase from $ B in 2019. The number of investigational new cell and gene therapy drug applications has tripled in the last three years.

Obtaining better insights into the specific technological, clinical and commercial challenges is crucial to ensure advances of this emerging field. Implementation of innovative clinical research
methodologies, current and future focused pricing models and ‘up-to-date’ regulatory approval paths constitute interlocked parts towards long term value creation of these novel, curative
therapies. Assessing innovative business models and partnerships will enable acceleration of commercialization processes and guarantee timely patients’ access.