Fulfilling the promise of gene therapy

About 40 years ago, our Co-Founder and Chief Scientific Officer Jude Samulski achieved an elusive scientific first. He discovered how to clone the adeno-associated virus (AAV), a breakthrough that helped set the course for gene therapy and allowed scientists around the world to begin thinking about new approaches that would one day be used to treat previously untreatable diseases.

At that time Jude knew he was on to something that could change lives, particularly for people with diseases for which there were few or no options and little or no hope. He was right. Now, thanks to the work of Jude and many other dedicated professionals, there are 32 gene and cell therapies approved in the United States and more worldwide.

Despite these gains, there is still much to be understood, and working to unlock the full potential of gene therapy is our passion at AskBio.

At their core, gene therapies can enable doctors to treat devastating diseases, often by introducing new genes into cells to help fight diseases or by making changes to the genes inside cells of the brain, heart, liver, muscles and elsewhere to treat a disorder or correct a genetic mutation. 

With six clinical stage therapies, AskBio is at the center of this effort. For example, we have scientists working to deliver a gene therapeutic to the brain to address a significant unmet need in the treatment of Parkinson’s disease, which affects roughly 10 million people around the world. Current treatments leave patients with inadequate control of symptoms as the disease progresses. Our Parkinson’s team, under the strategic oversight of Krystof Bankiewicz, MD, PhD, a world-renowned expert in neuro-restorative medicine, believes the exploration of gene therapies could alter disease progression and deliver robust and long-lasting functional benefits. Success here would be truly transformative. A recent blog post by Krystof, which was developed for the World Parkinson’s Congress, where he presented last month, provides an overview of gene therapy and growth factor approaches for Parkinson’s.

There are approximately 30 ongoing US studies testing neurological gene therapies for various disorders, according to the National Institutes of Health (NIH). Here is an inspiring look at the impact of some of the work in this area, including an NIH-funded effort by Krystof in collaboration with the National Institute of Neurological Disorders and Stroke (NINDS) and the University of California, San Francisco. 

Another area in which gene therapy is demonstrating promise is for the treatment of various types of heart disease. Roger J. Hajjar, MD, who provides strategic oversight for our team developing an AAV therapy targeting the heart, is encouraged by early data that hint at the therapeutic potential in certain patients with an advanced form of congestive heart failure, a highly prevalent disorder associated with high morbidity and mortality.  

As with all drug discovery and development, realizing the potential of gene therapy is difficult, complicated and expensive. Some diseases involve more than a single gene, clinical trials need to be ongoing and equitable and manufacturing processes need to be cost-efficient. Despite these challenges, we at AskBio are encouraged by the progress we are making with increased understanding and cutting-edge technologies. 

We are inspired every day by the opportunities we see to transform the lives of people living with diseases that need solutions.