CRISPR: The End of Genetic Disease?

Another revolutionary development in the field of genetics, CRISPR allows the DNA of living organisms to be edited with unparalleled accuracy and ease. With this breakthrough in-hand, humanity can realistically aspire to cure genetic diseases, create more resilient crops, and even design new organisms with custom properties. In the words of Trevor Martin, the CEO of Mammoth Biosciences, “what makes CRISPR really exciting is that it allows us to move from treating diseases to curing them. Rather than receiving treatment for the rest of your life you can access this one-time, potentially curative therapy that goes to the root cause of the disease.” CRISPR works by utilizing a molecule called RNA - which may sound familiar from the Covid-19 pandemic - that acts like a guiding system to direct a special enzyme to specific locations within the DNA, which allows one to remove, add or replace genetic material with almost absolute precision. “The rise of genomic and especially CRISPR technology completely shifted the paradigm in pharma; barriers were crossed and a new pool of opportunity developed,” says John Leonard, President and CEO of Intellia. Martin even claims that CRISPR’S presence implies a philosophical transformation: “We can make changes to our DNA rather than shooting in the dark and hoping that the molecule will affect the gene. If you cure one disease it makes it easier to tackle others, because you have already figured out delivery, dosage, etc., and all you are doing is switching up the guide RNA, a very reproducible and effective method. It simply changes the game of how we do drug development. There are 4,000 genetic diseases that we could target through our work.” CRISPR might also open new avenues of treatment for autoimmune diseases. These maladies are one of the biggest challenges in medicine due to their complex and multifaceted nature, and it is precisely by editing the genes that trigger the immune system to attack its own cells that CRISPR can revolutionize the way we understand and treat autoimmune conditions. The technology and infrastructure needed to make this a reality is still in its early days, however. Audrey Greenberg from the Center for Breakthrough Medicines enthusiastically tells us that “we are only in the early days of CRISPR technology, gene editing and allogeneic cell therapy - and they are already producing incredible results,” but Greenberg also highlights that “these innovations still remain extremely expensive.” Amongst other challenges, science has limited expertise in genomic analysis - we do not know what every specific gene ‘does’ yet - and difficulties in integrating these vast amounts of data into clinical decision-making. Additionally, some have voiced reasonable concerns apropos of how genetic data will be handled in terms of privacy and security. Yet, the exponential rate at which these technologies are advancing, make them the potential basis for a revolution that could save countless lives.

GIOVANNI CAFORIO | CEO, BRISTOL MYERS SQUIBB Precision medicine is definitely one of the highlights of today's biotech market. If in the past cardiovascular treatments were addressed to a large number of patients, today our drug Camzyos can address genetic cardiomyopathy in a deeply individualized way.

MORITZ HARTMANN | GLOBAL HEAD INFORMATION SOLUTIONS, ROCHE Since the early 2000's we have been focusing on precision medicine as we have seen how the rise of genomics drove the emergence of therapies that pre-identify if patients can benefit from a certain treatment.