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

Notable sector achievements in 2021 include:

• CAR T therapies are progressing to become viable first-line treatments
• Efficacy of Allogeneic Modified CAR T Cells in Autologous Cell Pairing
• Positive clinical evidence for in vivo curative CRISPR editing in the liver
• Advancing the “memory” editing capabilities of the genome with RNA edits or chromatin edits
• Evidence supporting the ability to reverse DNA damage with regenerative drugs for the treatment of complex and prevalent degenerative diseases

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

Looking forward to 2022, the main challenges that the industry will continue to face are:

• Dosing and administration of new therapies
• Chemistry, Manufacturing and Controls (CMC), including clarification of regulatory requirements
• Policy and Reimbursement (Navigating in a Legacy Health System)

These challenges were addressed in 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 cancer treatment. Experts also discussed industry growth, including updated regulatory pathways and landmark regulatory approvals that pave the way for future “living drugs.”

Panelists noted that, within the terms of their companies and the diseases they target, they are partnering with regulators to learn and evolve together. Regulators strive to develop well-characterized drugs that are both safe and effective. They seek clarification on product purity and the ability to scale products, which many experts have called “the product is the process”.

Although this is the gold standard, it is also extremely difficult to achieve when the technologies and modalities used are still emerging. Panelists reiterated that regulators and companies are learning on the fly.

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

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

In terms of commercialization, the panelists represented organizations at different stages of the pipeline, but each of the companies is at the forefront of unique and transformational technologies that are driving the field forward. While each indication and approach is unique, leveraging the benefits that individual therapies bring to each disease can be an effective route to commercialization and appropriate reimbursement.

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