A focus on personalized therapeutics
Genomic medicine field has progressed in leaps and bounds these last few years, and at the 3rd Annual CRISPR 2.0 conference, it was wonderful to again connect in person with colleagues to discuss those advances. It was a great opportunity to listen and learn from a prestigious lineup of gene editing experts from across the industry.
It is an exciting time to be involved in the CRISPR space and I was honored to have the opportunity to share the stage for a few minutes to talk about some of the latest technical innovations I work with that support cell and gene therapy manufacturing.
Presentations at CRISPR 2.0 were very focused on the development of personalized therapeutics for a variety of conditions, from cancer to pain disorders to congenital blindness. With so many high-quality talks, it can be challenging to just pick one to highlight, so instead I will touch on two themes that came up many times throughout the conference.
Base Editing and Prime Editing are Here to Stay
Base editing and prime editing are poised to take over the CRISPR field less than five short years after the initial publications from David Liu Lab at Broad Institute. Researchers immediately saw the therapeutic promise of a direct gene-modification approach that does not create double-stranded breaks in DNA, but low editing rates and challenges in manufacturability presented some early hurdles to therapeutic development.
Those days are now behind us. Several talks highlighted extremely exciting advances in base and prime editing. Dr. Kiran Musunuru from the University of Pennsylvania presented on a promising non-human primate study that applied adenine base editing to correct familial hypercholesterolemia. We also saw safety data from teams at St. Jude Children’s Research Hospital and the University of California – Irvine, who plan to use base editing to correct Sickle Cell Disease and Inherited Retinal Disorders, respectively.
Perhaps most exciting of all was the presentation from Dr. Jennifer Gori, Vice President of Research at Prime Medicine. Prime has made excellent progress in improving the efficacy of prime editors and now can correct all 12 possible point mutations in DNA in patient cells. Its dual-flap prime editing approach enables precision insertion and deletion of large, gene-scale DNA regions. Prime is also exploring the use prime editing to insert an integrase at precise loci, enabling insertion, deletion, or inversion of even larger DNA sequences!
Novel Nucleases Bring New Features and Functionality
Several presentations focused on the application of novel CRISPR nucleases. Speakers from Arbor Biotechnologies and Emendo Biotherapeutics highlighted novel proteins that can be applied to treat hypercholesterolemia and liver disease. For Aldevron’s part, I was excited to present on Eureca-VTM Nuclease, Aldevron’s newest Type-V CRISPR protein based on Incripta’s MAD7TM technology.
Scribe Therapeutics has really pushed the boundaries when it comes to improving new nucleases through engineering. As we heard from Scribe co-founder Dr. Brett Staahl, Scribe has engineered CasX into a novel “X-Editor” with limited homology to SpCas9 that is compact and broadly targetable. This protein is already being explored in cancer cell therapy and ALS therapeutic programs, with the prospect of epigenetic editing for tuneable gene repression on the horizon.
These are only a few of the excellent topics from the Precision CRISPR event. It was a great learning and networking experience for all who attended, and we’re looking forward to seeing everyone again in 2023.