Cell and Gene Therapy Landscape: Challenges and Opportunities for Biotech and Investors

Published October 19, 2021

Executive Summary

The American Society of Cell & Gene Therapy (ASCGT) wrapped up their 2021 conference earlier this year, which covered a range of topics, from new scientific advances to the evolving pricing and market access dynamics surrounding cell and gene therapies (CGT). Trinity had the opportunity to attend the conference, which brought together stakeholders from across the CGT landscape to discuss some of the challenges and opportunities within the space today.

In this post we will cover the most poignant insights from a variety of speakers who attended the conference and provide insights into forward-looking cell and gene therapy trends and new developments in the space.

Cell and Gene Therapy Focuses from a Biotech Perspective

One of the first speakers was founder and CEO of Taysha Gene Therapies, RA Session. Taysha Gene Therapies is a clinical stage biotechnology company that develops AAV-based CGTs to treat neurodegenerative diseases. Session focused on Taysha’s rapid growth, from a private pre-clinical company to a public, pivotal stage company in less than one year. This served as an example of the fast-paced development that can occur when starting from a validated technological platform with a well-thought-out development strategy.

The key takeaway from this presentation was that Taysha’s success is not only predicted from its innovative science, but also its experienced and talented people. Session emphasized the importance of working with established researchers that have robust experience in translational CGT. Moreover, he highlighted that having experienced team members in commercializing CGTs is equally as important as having a strong technological platform – Session’s views resonate with Trinity, as our experiences with commercialization of CGTs have similarly presented these unique technologies require the generation of tailored strategies developed by innovative and knowledgeable teams.

Cell and Gene Therapy Focuses from an Investor Perspective

To understand the fundraising side of CGTs, a panel of experts from biotechnology investment groups, including Atlas Venture, Cowen, Capital Group, and Barclays spoke about their perspectives on the space. Investors acknowledged the significant opportunity presented by gene therapies in their potential precision, durability, and modularity. However, panelists also raised key challenges for biotech’s looking to demonstrate their value to investors.

Manufacturing constraints were brought up as a major consideration for biotechnology companies when commercializing new products given the high upfront costs and strict regulatory requirements. Bluebird Bio’s ZYNTEGLO was highlighted as a recent example of manufacturing difficulties delaying drug approval, but some investors pointed out that the FDA’s recent Chemistry Manufacturing and Controls (CMC) guidance for CGTs could help to streamline manufacturing. Panelists suggested that either in-house or third-party manufacturing can work but emphasized that commercial-scale manufacturing should be invested in, and employed in clinical development, early to ensure any issues are resolved prior to regulatory filing.

Echoing the biotech perspective, investor panelists also noted the importance of credible and experienced management teams, which can instill confidence in investors. Strong business fundamentals were also a significant point of emphasis in helping bolster investor confidence in the potential for long-term commercial success. Demonstrating a well-funded balance sheet and feasible development plan backed by robust cost analyses and market models were considered essential to securing further investment.

Lastly, panelists discussed broader market trends in the CGT space and their expectations for its future evolution, including:

• The increasing number of companies deciding to quickly go public, even before pivotal trial data has been released:
  • Investors viewed IPOs as means of generating the large amounts of capital required to reach the regulatory stage of development, rather than the exit strategy it is typically used for
  •  Panelists expect small biotech’s to continue this strategy to secure the funding needed commercialize their assets
• The growing number of companies within the increasingly crowded cell and gene therapy space:
  • The idea of consolidation was welcomed as it would allow companies with strong fundamental value to de-risk their asset(s) by joining with others that could provide additional guidance and experienc
  • Crowding in the CGT space was also expected to increase the evidence thresholds required by regulatory and payer bodies, who may become more restrictive with reimbursement and access as more CGTs become available.

Themes From Biotech and Investors in Cell and Gene Therapy

Panelists from both investor and biotech perspectives highlighted the importance of having a team with strong commercial experience in the CGT space given the unique commercial development and reimbursement dynamics surrounding these types of therapies

Fundraising through IPOs or mergers can help the industry overcome barriers (i.e., manufacturing investment, trial costs, talent recruitment) to help more quickly bring CGTs to market

Biotech’s can make themselves more attractive to investors by lowering risk through some of the following strategies:

• Recruit experienced scientists and commercial teams that have proven track records within the CGT space
• Utilize established technological platforms that have already been clinically validated and that have shown to be commercially viable
• Invest early in manufacturing to ensure this is not a regulatory or distribution barrier later in the development process

By Cameron Lam and Ismail Ismailoglu, PhD