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.

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