From Brain Shuttles to Oral Peptides: 5 Technologies Advancing Research in Difficult-to-Treat Diseases
Get an inside look at how AbbVie scientists are exploring new therapeutic approaches that could help shape the future of medicine.
For decades, scientists seeking to discover new therapies have been constrained by available approaches. While powerful, these approaches face limitations – like an inability to reach the right locations in the body and to selectively target drivers of disease.
Classic examples are chemotherapy, which can harm healthy cells in addition to diseased ones, or getting medicines past the blood-brain barrier, a key for treating brain disorders. For patients already facing complex diseases, existing modalities can be coupled with burdensome procedures and inconvenient dosing.
But a burgeoning landscape of emerging modalities is changing that narrative and transforming our understanding of what’s possible in medicine. At AbbVie, teams are embracing this explosion of technologies, using expertise in areas like biology, chemistry, engineering and artificial intelligence, alongside an expanded research and development (R&D) toolbox on a quest for better therapeutic outcomes.
Each modality is selected and pursued in service of matching the right tool to the real problem at hand. “We’re intentional about every step,” explained Clark Pan, Ph.D., vice president, Biotherapeutics and Genetic Medicine, AbbVie. “Once we identify what patients need and assess how best to address those needs, we’re bold in our pursuit of the right tool to make a difference.” Pan and other scientists see a paradigm shift where genetic medicine and other new technologies are leading the way.
Learn about five modalities that AbbVie is exploring on a mission to create the next generation of options for difficult-to-treat conditions.
5 technologies we're using to find new approaches in cancer, neuroscience and immune diseases
In Vivo CAR-T
In vivo CAR-T is an emerging therapeutic approach where a patient’s own T-cells are reprogrammed inside the body to express a chimeric antigen receptor (CAR) and become disease-fighting CAR-T cells, rather than these CAR-T cells being made outside in a lab and reinfused (like traditional CAR-T therapy). Once generated in the body, in vivo CAR-T cells seek out abnormal cells using their engineered receptor and eliminate cells expressing a specific target antigen. They then expand and persist, which may enable ongoing surveillance against the disease.
What is its potential?
Traditional CAR-T therapy involves a complex, multi-step process that happens ex vivo, outside the body. T-cells are collected from the patient or a donor, engineered and expanded in a lab and then re-infused into the body. This requires a multi-week manufacturing process and an intensive chemotherapy session to prepare the patient for infusion, posing significant burdens.
As an alternative to ex vivo therapy, in vivo CAR-T builds on the possibilities of traditional CAR-T and has the potential to help address challenging diseases in immunology and cancer.
What’s ahead?
AbbVie is investigating two in vivo CAR-T platforms in immune-mediated diseases and cancer.
Blood-Brain Barrier Shuttle
Blood-brain barrier (BBB) shuttles are engineered molecules that can help medicines cross the BBB. The BBB exists to protect the brain against infection and other threats by preventing most substances, including many medicines, from entering. Many BBB shuttles work by binding to naturally occurring receptors on the BBB, such as the transferrin receptor, which usually transports iron. When a BBB shuttle attaches to one of these receptors, it uses this pathway to help carry the medicine into the brain, enabling drugs that would otherwise be blocked to help reach their intended target within the brain.
What is its potential?
The BBB creates major obstacles when it comes to delivering medicines to the brain, limiting options for neurological diseases. Traditional approaches often require very high doses of medicine in the hopes that some will cross the BBB. However, this means that high levels of the drug will be in circulation around the body, which can cause unwanted side effects.
BBB shuttles help facilitate the delivery of medicines by using the BBB’s natural receptor pathways, allowing treatments to more effectively reach their intended targets. This delivery technology can open doors for developing novel therapies for difficult-to-treat brain diseases.
What’s ahead?
AbbVie is researching BBB shuttle technology to support the discovery and development of medicines across neuroscience.
Multispecific Antibodies
Multispecific antibodies are lab-made antibodies designed to recognize and bind to more than one target at the same time. This enables them to function as connectors, helping to bring a disease cell and an immune cell together so that immune cell-mediated targeting of the disease cell can occur more effectively.
What is their potential?
While traditional antibodies bind to a single target, multispecific antibodies can bind to multiple targets. This can support efforts to develop precise and effective therapies and can potentially lead to additional therapeutic options. These molecules have the potential to expand what’s possible in striving to improve outcomes in complex diseases across immunology and cancer.
What’s ahead?
AbbVie is investigating multispecific antibodies in pre-clinical research and clinical trials, including potential treatments in oncology and immune-mediated diseases.
Oral Peptides
Peptide-based medicines are treatments made from small, protein-like molecules that help the body target or fix specific disease processes. Traditionally, most peptides are delivered through injections.
Oral peptides are peptide-based medicines that can be taken by mouth.
What is their potential?
Many peptide-based drugs have properties that can make them challenging for patients to manage. For example, they have historically required injections because they break down easily in the digestive tract and aren’t absorbed well as pills. Oral peptides offer an alternative way to harness the efficacy and therapeutic advantages of peptides.
What’s ahead?
AbbVie is researching a suite of novel oral peptide candidates across several autoimmune diseases.
siRNA
Small interfering RNAs (siRNA) are a class of molecules that can “turn off” or block a specific disease-related gene in the body. By blocking that gene, siRNAs can prevent cells from making the protein that contributes to the disease. This makes siRNA a genetic medicine approach.
What is its potential?
siRNAs are designed to selectively silence genes linked to disease, which makes them a customizable tool that can go after targets that might be considered “undruggable” by traditional modalities like small molecules or antibodies. This opens new possibilities for tackling difficult-to-treat diseases. Beyond its versatility, siRNA also offers the potential for long-lasting effects that open the possibility for infrequent dosing.
What’s ahead?
Through strategic partnerships, AbbVie is exploring next-generation siRNA therapies across neuroscience, immunology and cancer.
“Once we identify what patients need and assess how best to address those needs, we’re bold in our pursuit of the right tool to make a difference.”
For AbbVie, pushing the boundaries of science means striving to deliver therapies that are more targeted and that can lead to better outcomes, or even cures. “There’s a tremendous amount of unmet need even in areas where there may be existing treatment options,” said Steven Elmore, Ph.D., vice president, Small Molecule Therapeutics and Platform Technologies, AbbVie. “That’s why we continue to look for new innovation in those spaces: to improve not just the number of patients we can treat but also to continue to improve standards of care.”
Media inquiries:
[email protected]
Related Content
Unless otherwise specified, all product names appearing in this internet site are trademarks owned by or licensed to AbbVie Inc., its subsidiaries or affiliates. No use of any AbbVie trademark, trade name, or trade dress in this site may be made without the prior written authorization of AbbVie Inc., except to identify the product or services of the company.
Copyright © 2026 AbbVie Inc. North Chicago, Illinois, U.S.A.
