Expert Insights: The Promise of Genetic Medicine and AbbVie’s Role in Research

For the past 30 years, Clark Pan, vice president, biotherapeutics & genetic medicine technologies, AbbVie, has had a front row seat to the intriguing evolution of research in genetic medicines. 

The field, which focuses on using genetic information to treat or cure disease, first emerged in the 1970s. But as a young scientist in the early 2000s, Pan saw it enter a so-called dark age following a series of setbacks.

However, recent approvals for rare genetic diseases and pivotal discoveries — like the gene editing technology CRISPR and mRNA-based vaccines — have brought about a renaissance.

“People became true believers in the past 10 years when you started to see really dramatic impacts for patients emerging, like the blind being able to see again and babies who initially were not expected to live past age 2, now able to live much longer,” he explains.

Thanks to a wave of breakthroughs and ongoing investments, genetic medicines today are now more poised than ever to transform patient lives. We sat down for a Q&A with Pan to learn why and to explore how AbbVie is using genetic medicine technologies and platforms to find treatments, and potential cures, for patients. 

It refers to genetic materials, meaning DNA or RNA, used to treat or cure disease. These materials work by modifying, silencing, or replacing faulty genes that drive disease or by instructing cells to produce proteins that can help fight disease.

To create these therapies, you not only need genetic material but also a vector, or a vehicle for delivering genetic information. The most popular vectors today are viral-like vectors.

Viral-like vectors are proteins derived from viruses, such as Adeno-Associated Virus (AAV) and lentiviruses, that encapsulate and deliver genetic materials into patients’ cells. While vectors can be derived from a virus, they can also be made from a polymer of chemicals. One example is a lipid nanoparticle, a type of vector also being studied in this field.

The reason scientists like ours are excited about genetic medicine is because they have the potential to generate long-lasting effects in patients, and even potentially cure them, in a single treatment. This gives them an advantage over existing types of therapies, which often require multiple doses to maintain their effects.

Compared to small molecules and biologics, genetic medicines also have the potential to reach a broader range of targets. While small molecules can reach inside or outside our cells to get to a target, because they’re fairly small, they’re not as effective at impacting targets with a large surface area. Meanwhile, biotherapeutics like antibodies are sizable enough to bind to larger targets, but too large to penetrate cells.

In contrast, genetic medicines can address targets inside and outside of cells, both large and small surfaces, and have the potential to do so with a higher degree of control or specificity.

Scientists can use genetic medicine technologies to more precisely control biological effects by taking advantage of the different regulators to control the expression of genetic information. If, for example, we want to limit the expression of our genetic medicine to the brain, we can include, in our genetic medicine, a brain-specific promoter. That gives us another layer of specificity that would not be possible with other types of therapies. 

We’ve made substantial investments in this area initially through collaborations. One is with REGENXBIO, where we’re working to develop an AAV gene therapy for eye diseases. We have also collaborated with Capsida to utilize their AAV engineering platform and manufacturing capability to identify and advance eye and neurodegenerative disease programs.

Another collaboration we’ve invested in, with Umoja Biopharma, focuses on developing novel CAR-T cell therapies in oncology.

Through our collaborations, we’re seeking to address current challenges using genetic medicine concepts.

What’s also unique about AbbVie is that, not only do we look for partners, but we also invest in the ecosystem. Through our venture arm, we are investing in many novel technologies for genetic medicines while also looking out for strategic partnerships that can really move these technologies forward.

Our strategic investments, partnerships, and strong foundation in biologics, precision medicine, and complex diseases position us to make significant advances in genetic medicines. We have led therapeutic innovations in fields like ADCs (antibody drug conjugates) in oncology.

Our deep expertise in biotherapeutics and chemistry, and a talent pool of experts in the field, has propelled us to become a leading ADC company. Similarly, our strong foundation in biotherapeutics and small molecules, willingness to invest in cutting-edge technologies, and our ability to attract and nurture world-class scientific talent will help us advance the field of genetic medicines as a leader.

While it's very transformational, most of the clinical successes we’ve seen with genetic medicine have been limited to rare diseases. While that has a great impact, our vision for the future is to unleash the full potential of genetic medicine. It’s our vision within the next 10 years to see, through our investment and hard work that genetic medicine will have broader applications.

With genetic medicine, we also see a future where we can potentially cure patients, rather than manage their symptoms. That’s our vision for the future and we really believe genetic medicine has the potential to get there.

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