The CRISPR Journal
Volume 5, Issue 1 / February 2022
https://www.liebertpub.com/toc/crispr/5/1
Editorial
CRISPR Rewrites the Future of Medicine
Rodolphe Barrangou
Published Online:22 February 2022
…As always, the CRISPR collaborative network relies on teamwork, encompassing leaders in academia, clinical settings, and regulatory agencies enabling industry pioneers to develop CRISPR drugs. Critical translational endeavors by genome-editing companies and their pharmaceutical partners are translating progress into actual therapies that patients need, barely 2 years after the initial dosing of SCD patient Victoria Gray.
Several ongoing studies are anticipated to corroborate the safety and efficacy of CRISPR medicines, with about 50 studies currently registered on clinicaltrials.gov. Active studies in the aforementioned disorders and immunotherapies will be joined by trials on lymphoma (CB010A), myeloma (CTX-120), leukemia (NTLA-5001), carcinoma (CTX-130), Leber congenital amaurosis (EDIT-101), angioedema (NTLA-2002) and others, with more than two dozen studies actively recruiting. Most activity is centered in the United States and China, with trials also taking place in the United Kingdom, Canada, France, Sweden, and New Zealand, illustrating the global enthusiasm for CRISPR-based therapies.
CRISPR’s path to the clinic is reaching a critical point where clinical-stage development requires translational efforts by biotech pioneers joining forces with pharmaceutical companies to scale up these programs. Publicly disclosed partnerships include Intellia Therapeutics with Regeneron and Novartis; Editas Medicine with Bristol Myers Squibb and AskBio; CRISPR Therapeutics with Vertex and Bayer; Beam Therapeutics with Pfizer; and Caribou Biosciences with Abbvie. This trend has inspired additional partnerships and investments in up-and-coming players such as Mammoth Biosciences and Metagenomi, as well as fueling next-generation CRISPR startups such as Prime Medicine, Graphite Bio, LifeEDIT, Scribe Therapeutics, Tessera Therapeutics, Tome Biosciences, and Tune Therapeutics.
We thank our special issue guest editors, Annarita Miccio (Institut Imagine, Paris) and Matthew Porteus (Stanford University), for helping to shepherd this issue to completion. The articles in this issue illustrate how quickly translational efforts are progressing, how far into the clinic we already are, and set the stage for the expanded and accelerated deployment of CRISPR therapeutic payloads. We look forward to following this path in future issues of The CRISPR Journal.