GrowthTransforming Rare Disease Care: Orchard Therapeutics and the Future of Gene Therapy

The genes we inherit build our bodies. When rare anomalies occur, they can cause serious developmental or immune disorders that deeply affect patients and their families.

Technologies known as gene therapies have been developed to supply the body with functioning copies of genes to treat or prevent certain genetic diseases. Founded in 2015 and a Kyowa Kirin company since 2024, Orchard Therapeutics is a leader in hematopoietic stem cell (HSC) gene therapy, a revolutionary method that can potentially transform severe, often fatal inherited genetic diseases with a single treatment.

We spoke with Bobby Gaspar, M.D., Ph.D., Orchard Therapeutics CEO and a pioneer in gene therapy, to learn more about the journey of bringing this life-changing treatment to patients around the world.

Bobby is a world-renowned scientist and physician with more than 25 years of experience in medicine and biotechnology. He is one of the principal scientific founders and the current chief executive officer of Orchard Therapeutics, a global gene therapy leader acquired by Kyowa Kirin in 2024. A pioneer in gene therapy, Bobby was named to the TIME100 Health list in 2024. He is also an honorary professor of pediatrics and immunology at the University College London (UCL) Great Ormond Street Institute of Child Health.

I'm a pediatric physician by training, and was working at one of the world’s leading children’s hospitals in London. I was looking after a child with a severe genetic disease, and asked the professors how the child would be treated. They said, “She's going to have gene therapy,” and I genuinely thought that was a trick they play on the most junior member of the team. At the time, it sounded like science fiction medicine. But it was real: She was going to be the first individual in the UK to have any form of gene therapy. I thought it was amazing.

I volunteered to oversee the child's care. Since this was the first-ever treatment, we had to put together protocols, make ethics submissions, and work out ways of doing things, which I found fascinating. Knowing that there was a form of medicine that could have this kind of impact—I was hooked. So I went into the lab and did a Ph.D., and built up a gene therapy program at the hospital. I was meant to do six months, but ended up staying 25 years.

As a faculty member at University College London, I worked on developing and progressing gene therapy. Together with another physician scientist, we built up one of the largest programs worldwide. We showed for the first time that gene therapy can correct genetic diseases. The first patient we treated successfully was in 2001. That boy—now a man—has a functional immune system. He's finished his university studies. He posts on social media about his life. And that was due to a single administration of gene-modified cells. It was very powerful to see the impact this could have on him and his family.

Over the years, we had children coming from different parts of the world to access therapy in London. And for the vast majority, it was very successful. It was incredibly rewarding and satisfying to clinically treat patients and see the effect that it had on them and their families. It became clear that we needed to expand this from the academic setting into an entity that could make these academic endeavors into standard medicines and provide access for patients worldwide. We had interest from venture capitalists to set up our own company, which would give us a lot of say in how that happened. And that's the path that we chose to take.

HSC gene therapy is a form of genetic medicine where we remove blood stem cells, called hematopoietic stem cells, from an individual and modify them by introducing working copies of a faulty or missing gene. This is done using viral vectors to modify the hematopoietic stem cells. These genetically modified cells are transplanted back into the patient, where they migrate, multiply, and proliferate throughout the body.

One of the most important is the fact that you can get a potentially durable therapeutic effect with a single administration. Unlike other forms of genetic medicine, in HSC gene therapy, the viral vectors integrate the new genetic material into the chromosome of the cells. As each cell divides, the corrected genetic material is passed on. With every cell division, you create another cell that is also gene modified as well. If you modify HSCs in the laboratory and then introduce them back into the patient, you have this expanding pool of gene-modified cells that remains for the lifetime of the patient, resulting in a potentially durable effect.

The other really innovative aspect is that HSCs can naturally become all the other different parts of the blood system. They become white cells, immune cells, or red blood cells. They can become platelets. Or they can become what are called monocyte-macrophage lineage cells, which can migrate into other tissues, creating the opportunity to address many different diseases. For example, they can migrate into the brain, allowing you to deliver new genes into the brain. That's how we've been able to correct a neurodegenerative condition. We have patients who were treated nearly 15 years ago, and they continue to show normal development—something extraordinary in this disease. That's how powerful the HSC approach is.

When building the company, at one stage we were reliant on the financial markets. Things had happened in the gene therapy field that made the markets very bearish, so we couldn't raise money like before. It became clear that we could no longer resource everything we wanted to do. We had to let go of a number of programs, and people as well. That was a really tough time.

We had around 250 staff, but could only support around 150 or 160, meaning we had to let around a third of our workforce go. But the fact was, either we do this, or we won't survive as a company and won't move forward at all. We have to be united on what we're going to do, even though it's tough. And we have to communicate that to the organization. That was a seminal moment for us as a company.

We narrowed our goals and focused our purpose, which was extremely galvanizing. Then I asked my leadership team, “It’s going to be tough, but do you want to be part of this?” And they all said, “Yes, we want to be part of this. Let's make this happen.”
I strongly believe that it’s the people that in our company that are most important. Once people commit, they put their all in. We had to be very streamlined; in some areas we couldn’t have teams of five—we just couldn’t afford it. Despite the challenges of working in a lean environment, our people showed incredible dedication to the program and rose to the challenge.

I think this saved the company, but only because we were very clear about what our purpose was, that we were all in it together, and that we were going to have to work extremely hard. We adopted what we called a STRIVE mindset: we were striving to thrive as a company. I think these tough decisions and commitments are also ultimately what led to our success. We were able to move forward and get approval for a medicine that was an extraordinary achievement for such a small company.

Many of our staff don’t have a scientific or medical background, but they need to know about what we do. The best way is to hear directly from the families, so our patient advocacy team invites patients and their families to talk about their experiences. I think initiatives like this bring our staff together with a shared sense of purpose, so everyone feels that that they are contributing to save patients’ lives.

At our last company-wide event, we invited a family where it was unfortunately too late to treat one child, and she’s now confined to a wheelchair. But we were able to treat her younger sister, who's now able to run around. Both children came to the meeting, and the whole company could see firsthand what a difference gene therapy can make. I think it's very important to bring these stories to the team, and they give us incredibly positive feedback about how motivating it is.

It's been incredibly rewarding being part of Kyowa Kirin, and a very productive relationship. We’re doing more than before, combining resources, and thinking about how we can combine research capabilities. I think the two companies are very similar in several ways.

First is our mindset: the STRIVE mindset aligns very well with the KABEGOE principles and associated behaviors. Everything we’ve been doing at Orchard has a KABEGOE element to it: ingenuity, doing the right thing, caring for patients, being brave, being agile, being bold. So, we want to formally adopt the KABEGOE principles as well.

Another similarity is that this type of therapy is very patient-centric, and Kyowa Kirin is a very patient-centric organization. Kyowa Kirin aims to create life-changing value, and I think we have a shared vision to build therapies for many different diseases—to treat rare diseases, but also more common diseases as well.

We're in rare diseases at the moment, and I think we can have a dramatic effect on a number of them. Ultimately, our vision is for gene therapy to be seen as a mainstream medicine, which means standardizing it and making it more accessible. We haven't reached that stage yet, but have programs in the pipeline.

While HSC gene therapy isn’t approved yet in Japan, we're working with Kyowa Kirin to go through the regulatory process. Whether scientific or regulatory, we've come across many hurdles and will continue to do so. But when you know there’s something that provides such an extraordinary outcome for patients, you find a way to overcome challenges.

The future of HSC gene therapy.
After this interview, in December 2025 it was announced that two genetic diseases, including metachromatic leukodystrophy, have been added to the U.S. Recommended Uniform Screening Panel (RUSP), the federal list of medical conditions recommended for newborn screening (NBS). Currently, 14 states representing more than half of U.S. births have mandated RUSP alignment. NBS enables identification of listed diseases, which is critical to achieving the earliest possible treatment. Globally, community-led initiatives continue to drive progress, marking a highly anticipated step toward expanding access to care and support for patients and families. Orchard teams played a huge part in making this happen and demonstrated how having a shared purpose and real commitment can make a significant impact.