In August 2021, the Alliance for Regenerative Medicine (ARM), in collaboration with GlobalData, published a new report highlighting that 2021 has already been a “year of firsts and records” for the regenerative medicine sector with significant clinical milestones, commercial progress and investment.
For example, CRISPR gene-editing technology was used for the first time in vivo, with Intellia Therapeutics announcing promising Phase I data from a clinical trial of NTLA-2001 in transthyretin (ATTR) amyloidosis patients. Using data from GlobalData’s Clinical Trial Intelligence database, the report also shows that there are over 2,600 trials for regenerative medicines ongoing worldwide, including over 1,300 industry-sponsored trials and with almost 250 in Phase III. In terms of investment, the sector raised a record $14bn in H1 2021, compared to $19.9bn for all of 2020.
In addition, 2021 is on track to have the highest number of regulatory approvals of gene therapy and gene-modified cell therapy products, with three approvals to date and four expected to get the green light by the end of the year. The report also highlights that Europe could be at risk of falling behind the US and Asia in terms of number of developers and new clinical trials.
Initiatives by ARM to educate policymakers and payers in the US and Europe on regenerative medicines are also addressed in the report. For example, ARM has assisted in shaping US policy by working with congressional sponsors on ‘Cures 2.0’ legislation and advocating for increased funding for the FDA’s Center for Biologics Evaluation and Research (CBER). In Europe, ARM was involved in removing a reimbursement hurdle for hospitals in Germany that provide regenerative medicines.