A special time for gene therapy

AskBio has a strong connection with the European scientific community. One of the many occasions when this comes to life is when scientists from our company, our industry, and academia gather each year at the European Society of Gene and Cell Therapy (ESGCT) congress to share the work being done to advance the field. Our community is a powerful one, and the sense of excitement at presentations and in hallway conversations at this meeting is always palpable.

At this week’s ESGCT, our team—including manufacturing subsidiaries Viralgen and TAAV Biomanufacturing Solutions —will deliver 20 presentations focused on everything from assays, capsids, and cassettes to vectors, promoters, and immune modulation, with this last topic being the subject of an oral presentation. We will also provide insights related to enzymatic DNA, tissue- and muscle-specific AAV, and the use of gene therapy in Huntington’s disease and congestive heart failure.

AskBio’s contribution to our community’s current feeling of progress also reaches beyond Europe and ESGCT. For example, in November, at the American Heart Association Scientific Sessions 2023, we will present interim data from our Phase 1 gene therapy study of AB-1002, an investigational gene therapy for the treatment of congestive heart failure, which has completed enrollment in patients with Class III heart failure.

When I look at what we and others are doing, it is easy to see that gene therapy has entered a new phase—what AskBio Co-Founder and Chief Scientific Officer Jude Samulski described in a recent podcast as a renaissance. He attributes this shift to a “retooling” that has allowed our field to be more creative in approaches to developing therapies for currently untreated diseases of the eye, brain, heart, and lungs. As a result of the work of many in our community, we can anticipate seeing more gene therapies becoming available in the coming years, which means many patients won’t need to wait another decade or more. 

I see this commitment to boldness and ingenuity every day at AskBio, and it is thrilling. With the support of Bayer, we are breaking new ground as a molecular medicine discovery engine capable of developing, manufacturing, and delivering solutions to patients and their dedicated doctors for rare and more common diseases. We also have what it takes to address the next challenges of discovery and development and are exploring ways to potentially treat complex diseases involving more than one gene. And, we are doing this while remaining focused on and committed to increasing the efficiency of our production capabilities, to tackle challenges related to commercialization.

When I think about what success looks like, it is a vision in which the impact is driven by the talent and leadership of absolutely everyone in this field. It is a journey of unleashing potential, and this is well underway.