Cell and gene therapies


Cell and gene therapies

cell DNA double helix

Ambitious vision or the real deal?

It is fair to say that cell and gene therapies (CGTs) have not walked an easy road, from reaching the peak of excitement in the 1990s to descent into a period of disillusionment following a spate of safety red flags in the technologies’ early clinical days. However, intensified clinical research and investment has, to some extent, helped certain CGTs weather the storm in bringing the first generation of these new drug modalities to market. Yet, with elevated pricing, manufacturing complexities and the limited disease scope that current CGT treatments can target, they are by no means, to date, therapies for the masses. These challenges can only be addressed through continued innovation, which we believe is housed in biotech companies. Ultimately, it will be the most robust science that unlocks the true potential and opportunity that CGTs possess.

Separating the hype from the science

CGTs have undoubtedly topped the list of clinical buzzwords over the last 20 years, but much hype coupled with very few wins left many questioning whether CGTs were nothing more than a therapeutic pipedream. With innovative advancements in gene-editing strategies, delivery technologies and an increasing number of biotechs focused on CGT development, the 2010s finally gave way to the first wave of CGT clinical successes.

Such approvals certainly indicate that the field is heading in the right direction; however, it will be important to not get caught up in a second CGT hype cycle, as concerns over toxicity and treatment durability continue to linger. For CGT development to make further strides, it must be rooted in companies with a solid scientific foundation aimed at making treatments more effective, safer and affordable. Indeed, some big pharma players have firmly backed CGT technologies with substantial financial investment in the form of biotech deals, stockpiling their CGT pipelines in anticipation of delivering the next blockbuster (see Exhibit 1).

Exhibit 1: Big pharma CGT pipelines

Source: Evaluate Pharma, Edison Investment Research

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