News & Events

9 top CRISPR breakthroughs of the past year

The world of gene editing is one where the impossible becomes possible, and the future of healthcare is being rewritten before our eyes. CRISPR and other gene editing based therapeutics represent a transformative step in healthcare, moving us from an age of treatment and living with disease, to one of cure.

In the last year alone, advancements in CRISPR research have been nothing less than awe-inspiring, from promising results in clinical trials to the first gene therapies being approved by the FDA and EU. Below, we’ll explore nine of the most thrilling CRISPR breakthroughs from the past year, each of which is a testament to human ingenuity and a glimmer of hope for people around the globe.

These are not just milestones; they are the harbingers of a new dawn in healthcare, promising a brighter, healthier future for all.

1. A Historic Approval by the European Commission

Imagine a world where genetic diseases like severe sickle cell disease and beta-thalassemia are no longer life sentences. The FDA’sand now European Commission’s – approval of CASGEVY™, the first CRISPR/Cas9 gene-edited therapy, is a giant leap toward that reality. This breakthrough is a testament to the power of collaboration and innovation in transforming research into tangible treatments.

While we celebrate this groundbreaking achievement, it’s also a moment to reflect on the critical importance of precision and safety in gene editing. At SeQure Dx, our commitment to advancing genomics intelligence underscores the value of meticulous off-target analysis to ensure the safe application of such innovative therapies as CASGEVY™.

This approval not only opens new doors for patients but also highlights the collaborative effort in the scientific community to turn genetic research into life-changing treatments.

2. A New Horizon for Friedreich’s Ataxia Patients

Friedreich’s Ataxia has long been a challenging condition, but CRISPR-Cas9’s potential to address its root causes offers a ray of hope. By correcting mitochondrial defects in iPSC-derived neurons, this approach might just be the game-changer we need. What’s thrilling is the promise it holds for not only improving lives but potentially reversing the course of this debilitating disease.

If you are working on iPSC-based therapies, you’re probably already aware of the challenges of OTE with traditional methods such as guide-seq; that’s why our ONEseq assay has become the go-to technique for off-target analysis of iPSC derived therapies.

3. A Revolutionary Approach to Treating Angioedema

Gene therapy is a rapidly evolving field with the potential to revolutionize treatment for a wide number of diseases. In one of the latest breakthroughs, researchers may have found a single-dose treatment that provides a permanent cure for angioedema, a debilitating condition that causes swelling in the face, throat, and extremities.

The therapy uses CRISPR-Cas9 gene editing, and with just a single dose it targets the liver and reduce the production of a protein that causes swelling.

Early results have shown a 95% reduction in symptoms and significant improvement in quality of life for patients.

Why is this significant? As the article explains, CRISPR-Cas9 is a new modality that rather than treating symptoms, providing bandaid treatment, and relying on lifelong drug regimes, these diseases can be repaired at the root cause with a single treatment.

4. Breaking the Silence: A Cure for Genetic Deafness?

Could gene therapy soon provide a cure for a rare, genetic form of deafness? Early results from Eli Lilly’s Phase I/II trial of its experimental AK-OTOF treatment are looking promising.

The Children’s Hospital of Philadelphia last week shared results from the first patient in the US treated with the experimental gene therapy.

The 11-year-old patient, who was born deaf in both ears, has shown significant improvement in his hearing within a month of receiving the treatment.

At SeQure Dx, we’re committed to advancing gene editing and diagnostics, ensuring therapies like AK-OTOF are delivered safely and effectively.

5. Exploring New Frontiers with CRISPR-Cas3

Less researched than Cas9, CRISPR-Cas3 genome-editing technology is showing its capability in the early results of a collaboration between the Yan Zhang Laboratory at the University of Michigan and the Ailong Ke Laboratory at Cornell University.

Scientists are exploring CRISPR-Cas3 for its potential to precisely target and remove large sections of DNA, offering a new approach for studying genetic diseases by enabling the deletion of long genomic sequences. It’s early days, but scientists are encouraged by its potential clinical uses.

It’s an exciting step toward uncharted territories in genomic medicine, and with our nuclease agnostic ONEseq assay we’re already thinking about how we incorporate it into our services.

6. Targeting Viral Genomes with CRISPR

The use of CRISPR to combat herpetic stromal keratitis, a serious condition caused by the herpes simplex virus, is a brilliant example of how gene editing can be tailored to tackle viral diseases. By directly targeting the viral genome, this approach offers a new paradigm in antiviral therapies.

The significance of this breakthrough lies in its potential to provide durable solutions for viral-induced conditions, opening a new frontier in infectious disease treatment.

These developments also underscore the importance of precision in gene editing, a core focus of our technology here at SeQure Dx.By ensuring therapies are free from off-target effects early on, we not only enhance safety but also save time and money, accelerating the path to clinical success.

7. MEGA: A Reversible Gene Editing System

Researchers at Stanford University in California have developed a new CRISPR-based gene editing system called MEGA that targets messenger RNA (mRNA) instead of DNA. Unlike DNA editing which causes permanent changes, mRNA editing is temporary since mRNA is short-lived.

The excitement around MEGA stems from its potential to pave the way for innovative treatments that are both effective and reversible, offering a new level of control in gene therapy.

We look forward to seeing where this compelling new research leads next. And as new therapies emerge, SeQure Dx will be here to improve their safety and effectiveness, using our unrivalled diagnostic tools.

8. Fast Track for a CRISPR-Based HIV Cure

The Fast Track designation for EBT-101, a CRISPR-based therapy for HIV, could be a huge breakthrough in the quest to conquer this virus.

The excitement here is twofold: the potential to provide a cure for a disease that affects millions and the broader implications for the power of CRISPR to tackle other persistent viral infections. This development could mark a momentous step forward in our fight against HIV.

9. Cholesterol Management Revolutionized by VERVE-101

The level of cholesterol reduction achieved with VERVE-101 offers a glimpse into the future of cardiovascular health management. This gene-edited therapy’s potential to provide lasting solutions for familial hypercholesterolemia is yet another testament to the transformative power of CRISPR, and this potential breakthrough represents a major stride toward tackling heart disease at its genetic roots.

In all of these breakthroughs, we see more than just scientific achievements; we see hope, promise, and a future where the boundaries of medicine are being continually expanded. As we celebrate these milestones, we also look forward to the next chapter in the use of CRISPR, eager to witness the unfolding of this remarkable technology’s full potential.