Name: CRISPR-based gene editing revolutionized medicine—what's next?
Uploaded: 2025-06-18T17:59:24.000Z
Duration: 5 min 10 s
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Recent technological advancements have paved the way for the development of medical treatments for life-threatening diseases.

At the forefront of this is CRISPR-Cas9, a gene editing technology elucidated over a decade ago and honored with the Nobel Prize in Chemistry in 2020.

I traveled to Berlin to meet Emmanuel Charpentier, one of CRISPR-Cas9's inventors.

CRISPR is a system that exists in bacteria and allows bacteria to defend themselves against infection by viruses.

So bacteria can also be infected by viruses and they have evolved different immune systems and one of them is the CRISPR-Cas9 system.

And so by our interest in the lab to find new molecules that could have a role in the bacterium Streptococcus pargenes, we came across a very neat mechanism that allows us to really recognize the virus that infects the bacterium in a very, very specific manner at the level of the genome of the virus.

And we exploited this natural mechanism to develop the CRISPR-Cas9 technology.

With a mission to cure disease, in 2013 Emmanuel co-founded CRISPR Therapeutics, a biotech company that uses CRISPR technology to develop gene therapies.

After 10 years in operation, CRISPR Therapeutics and its Vertex Pharmaceuticals saw their drug Casgivy become the first CRISPR gene therapy to be approved by the FDA.

It treats two serious inherited blood disorders, transfusion-dependent beta thalassemia and sickle cell disease.

We spoke with the CEO of CRISPR Therapeutics, Samath Kulkarni, at the company's headquarters in Boston.

In the U.S. there are about 100,000 patients that suffer from sickle cell disease.

Nearly a quarter of them are severe sickle cell patients that have pain every day, that have to be hospitalized every so often, and ultimately don't live as long as everyone else.

With Casgivy we're taking the bone marrow cells from the patient, making the edit for that particular patient, and we're putting it back into the patient.

And it reconstitutes the hematopoietic system of the patient.

CRISPR Therapeutics currently has seven clinical and 10 preclinical programs in the fields of oncology, autoimmune cardiovascular disease and diabetes.

By 2027 it hopes to have enough data to launch the next wave of therapies, most of which will be created in these labs.

Shaheen Kabir is CRISPR Therapeutics principal scientist.

The first part of developing a therapy is figuring out what gene or target sequence is causative of disease and what you want to basically target to correct or repair.

The second part is then designing components that will allow you to go to that gene sequence and perform the edit of interest.

Once you've developed these components you now have to figure out how to deliver them to the cells or the tissues that you're interested in.

And then finally these pieces all come together and you would assess the efficacy and the potency of the therapy that you've developed.

CRISPR gene therapies can be administered in two ways.

Ex vivo, the most common practice, is when cells are removed from the patient's body, edited and then reintroduced.

The alternative method is in vivo, when changes to a cell's genetic material are made inside the patient's body, something CRISPR Therapeutics is working to make more widely available.

The scientists here are exploring how CRISPR components can reach hard-to-target tissue such as those in the brain or lungs for in vivo editing.

To investigate and develop new therapies, the company set up another research team called CRISPR-X.

There are a lot of diseases that we're not currently able to target with CRISPR and that may be because of delivery, we're not able to get the right components to the right place, but it might also be that the kind of change that we need to make, we don't necessarily have the tools for yet.

And that's what the focus of CRISPR-X is.

CRISPR was developed to advance medicine but its groundbreaking capabilities have led to its use beyond healthcare, including biotechnology and agriculture.

I think it's always important to figure out, you know, what are the needs for the population and then whether CRISPR is the right technology to use and if this comes together then it's important to, you know, to consider new technologies.

So looking into your crystal ball, Emmanuelle, where do you see the future of this technology including with CRISPR-X?

So we will see the use of the technology in the future, directly or indirectly, being important for other diseases that will be cured.

So either using CRISPR or new therapeutics that would have come up on the market thanks to CRISPR because CRISPR was used in the process of research and development.