Regenerative medicine couldn’t stop making news in 2021. The year was marked by Intellia’s first results in humans for gene editing, massive Big Pharma partnerships and record investment of $22.7 billion. This past year saw six new cell, gene and tissue-based therapies approved, according to an annual report from the Alliance for Regenerative Medicine (ARM). Meanwhile, CAR-T therapies put up the data to justify moving up a line in cancer treatment, and venture capital went all-in on the field.
Gene and cell-based therapeutics attracted the bulk of regenerative medicine investment last year, with gene-editing approaches garnering confidence following key clinical milestones, new research shows.
Regenerative medicine is taking aim at cancer and a wide range of other prevalent and rare diseases, according to the 2021 annual report of the Alliance for Regenerative Medicine (ARM). “The exciting advancements of 2021 demonstrate that regenerative medicine is here — and it’s not slowing down,” said Janet Lambert, CEO of ARM.
Despite a turbulent investment year, the cell and gene therapy space continued to innovate and develop at an extraordinary speed, according to the “Cell & Gene State of the Industry Briefing,” which was delivered on January 11 by the Alliance for Regenerative Medicine (ARM) in partnership with Biotech Showcase.
In 2022, the outlook for the field remains bright, but companies face critical questions that could shape whether, and how soon, new genetic medicines reach patients.
In terms of approvals of new products, 2021 was the second-best year for cell and gene therapies and tissue products.
Cell and gene therapy makers need fit-for-purpose chemistry, manufacturing, and controls (CMC) standards, according to the Alliance for Regenerative Medicine (ARM), which says the current guidelines lack clarity. Therapeutic candidates only become products if their developers can prove they are made in consistent manner, in line with quality requirements. Such issues are covered in the CMC section of dossiers filed for review.
Several trade associations, including the Pharmaceutical Research and Manufacturers of America (PhRMA), the Real-World Evidence (RWE) Alliance, the Biotechnology Innovation Organization (BIO), and the Alliance for Regenerative Medicine have all asked FDA to extend the comment period so that they can continue to review the EHR guidance and see how it aligns with other RWD guidance documents.
