Genomic Medicine Milestones: The Resurgence of CRISPR Gene Editing
The future of genomic medicine relies on technical considerations and ongoing industry collaboration
Rachel Diamant, MSc, co-founded Emendo Biotherapeutics and is responsible for its IP strategy. As the chief business officer, Damiant builds the pipeline of clinical programs and industrial collaborations for the preclinical development of selected indications and genomic targets. She holds an MSc in neurobiology from the Weizmann Institute of Science.
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A decade ago, research demonstrating the promise of CRISPR sparked a wealth of interest in genome editing. CRISPR technology was revolutionary in the realm of rare diseases, ushering in a new era of medicine that allowed scientists to engineer the genome in ways previously unimaginable.
Like any technology, CRISPR has its limitations, which have become more apparent in recent research. For all its potential to change the landscape of genomic medicine, key challenges have slowed progress toward an unspoken industry-wide goal of creating a world where incurable diseases become curable. Delivery constraints, limited genomic target accessibility, off-target effects, and limited editing efficiency are a few of the most notable challenges.
However, the last few years have seen a resurgence of interest and commitment to CRISPR-based gene editing, fostering new hope of reaching its full potential.
Industry advancements make CRISPR better, safer
The future of genomic medicine continues to be an exciting and emerging topic of interindustry conversations critical to advancing the field as a whole. The recent Cell & Gene Meeting on the Mediterranean conference is an excellent example of industry voices coming together to share findings, learn from one another, and propel an industry that has the potential to impact millions.
Discussions at the conference revolved around critical challenges in CRISPR gene editing, particularly the need for more precise editing. A limited range of genomic targets prevented early CRISPR-based technologies from being able to treat many patients. Solutions to this limitation are now beginning to take shape, as researchers focus on approaches that allow access to more of the genome: By pairing protein engineering with computational and machine learning platforms, gene editing can now be customized to improve genome accessibility and reduce off-target effects to provide safer options for broad patient populations suffering from various genetic disorders.
Delivery mechanisms are also an essential consideration, directly informing the development and eventual success of next-generation gene editing. The Meeting on the Med also included active discussion on how CRISPR-based technologies can accommodate a variety of delivery modalities, including in vivo and ex vivo approaches, and be scaled to allow for large-scale manufacturing.
The Next Generation of Genomic Medicine
The future of genomic medicine does not solely rely on novel technologies—it relies on technical considerations and ongoing industry collaboration to further the evolution of gene editing therapeutics. Through this integrated lens, we can envision the next generation of genomic medicines making many incurable diseases curable. With enhanced precision, accessibility, and indications, realizing the potential of genomic medicine is becoming more tangible.