Written in the CARs

For thousands of years, the night sky has served as both a scientific tool and a source of universal wonder.

In ancient times, stars helped sailors navigate the open seas and farmers determine when to plant and harvest crops. Several civilizations used the moon to track the passing of time, creating the first calendars. Simultaneously, the sky overhead offered a sense of cosmic connection. Ancient Egyptians believed that human souls ascended to the sky after death and the Greeks revered the stars as gods, watching over them from above.

Cell and gene therapies exist in a similar duality. What started with a theory that DNA sequences could be introduced into patients’ cells to cure genetic disorders has evolved into an entirely new paradigm of medicine. Functionally, they are incredible feats of science and engineering, offering the ability to repair or replace faulty or missing genes or transfer cells into a person to target disease. On a more metaphysical level, CGTs represent a beacon of possibility; the potential to shine a light of hope into previously dark prognoses.

So far, this light has been limited to a small subset of patients, most of which are clustered in the wealthiest regions of the world. Low- and middle-income countries bear approximately 90% of the global disease burden. Yet only four countries designated as LMICs by World Bank — China, Brazil, India and Philippines — have approved cell or gene therapies. And given the high price tag on treatments and complex manufacturing logistics, regulatory nods haven’t ensured that treatments reach patients.

But many in the industry believe that treatments — and hope — should be globally accessible. 

As you will read in this issue’s cover story, the idea of broader access has ignited the industry’s attention. Various stakeholders, including governments, industry associations, nonprofits and pharma companies are linking forces and chipping away at barriers. 

While stakeholders in the sector may have divergent or even competing interests, the end goal for all is getting more treatments to more patients. With multiple stakeholders coming at the problem from multiple angles, sustainable global access avenues are beginning to take shape. From process efficiencies aimed at reining in costs to nontraditional manufacturing and go-to-market models, the cell and gene ecosystem has now put forth multiple models that, when used in harmony, could put global accessibility within reach.

To quote Astronomers Without Borders, “Boundaries vanish when we look skyward.” No single nation owns the constellations. In a perfect world, this same philosophy would guide global access to cell and gene therapies. And I believe it was Jiminy Cricket who eloquently explained that no matter where we are or what our circumstances may be, no ambition is too extreme when casting our requests upwards to the stars. 

No topic could be more fitting to mark the launch of Cell & Gene Therapy Review. It is our wish to create a dedicated platform under which we can unite the ideas of the entire cell and gene therapy development and manufacturing universe.

Just as the vastness of a starry night sky never fails to fill me with awe, the cell and gene therapy industry continues to astonish me by redefining the limits of what we once believed possible. It is my absolute honor to serve as your inaugural editor-in-chief, and I look forward to watching you shine.