The briefing is an annual update on the sector's progress in the areas of innovation, clinical and regulatory development, and funding. Janet Lambert, CEO of ARM, delivered the update, which was followed by two panel discussions with industry leaders.

Notable achievements for the sector in 2021 included the following:

• CAR T therapies progressing toward becoming viable first-line treatments
• Allogeneic-edited CAR T cells' efficiency in matching autologous cells
• Positive clinical evidence for curative in vivo CRISPR editing in the liver
• Advancing capabilities to change genome "memory" with RNA edits or chromatin modifications
• Evidence supporting the ability to reverse DNA damage with regenerative medicines for the treatment of prevalent, complex degenerative diseases

Interestingly, while the investment landscape in 2021 was volatile, the sector saw a 16% increase in the total dollars invested, totaling $23.1 billion. Lambert explained that the downturn was due to macro forces (such as inflation hikes) and fourth quarter clinical trial failures that resulted in short-term volatility. Despite the tough financial situation, there were six new U.S. regulatory approvals in the sector and 2,261 active global clinical trials.

Looking forward to 2022, the key challenges the industry will continue to face are around:

• Dosing and delivery of novel therapeutics
• Chemistry, manufacturing, and controls (CMC), including gaining clarity in regulatory requirements
• Policy and reimbursement (navigating a legacy healthcare system)

These challenges were discussed during the first panel discussion, which focused on cell and gene therapies for rare diseases, while the second panel discussed the role of these therapies in treating cancer. Experts also discussed the growth of the sector, including updated regulatory pathways and landmark regulatory approvals that are paving the way for future "living drugs."

The panelists noted that within their companies' modalities and the diseases they are targeting, they are partnering with regulators to learn and evolve together. Regulators are striving for well-characterized drugs that are both safe and efficacious. They are seeking clarity around product purity and the ability to scale products, which many experts referred to as "the product is the process."

While this is the gold standard, it is also tremendously difficult to achieve when the technologies and modalities used are still emerging. The panelists reiterated that regulators and companies are learning on the fly.

There was an overall tone of optimism in both panels in terms of the regulatory and CMC outlook. The pace of innovation is extremely fast right now.

In addition, the sector is trying to develop new standards without regulators having enough experience, bandwidth, knowledge, or time to create appropriate guidances. However, this is an opportunity to establish productive dialogue between regulatory agencies and drug developers to exchange lessons learned," Osvaldo Flores, PhD, CEO of Century Therapeutics, explained.

In terms of commercialization, the panelists represented organizations at various stages in the pipeline, but each of the companies are pioneering unique and transformational technologies that are advancing the field forward. While every indication and approach are unique, leveraging the benefits that individual therapies bring to each disease can be an effective path to commercialization and appropriate reimbursement.

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