Thoughts Ahead of ASGCT

There are many reasons people dedicate their careers to advancing medical science. Mine have always been rooted in the goal of helping patients fight challenging diseases. Since joining AskBio two months ago, I have experienced first-hand the extraordinary dedication of scientists, researchers, and leaders to finding the breakthroughs that will deliver the much-needed medicines of the future.

As the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) approaches, I am looking forward to joining the gene therapy research community to share data and find new ways to collaborate. Delivering life-changing and life-saving treatments to patients—many of whom have exhausted all options or never had any at all—is a big job, a team effort.

I am very proud of AskBio’s pioneering work and robust scientific contributions that, as our Co-Founder and Chief Scientific Officer Jude Samulski says, “spans the subtlety of AAV vector design, quantifiable transgene expression, and preclinical efficacy, with the aim of furthering AAV therapy for additional unmet clinical need.” Our leaders will share our latest data and insights in four oral presentations and seven poster presentations on AskBio’s adeno-associated virus research, development, and pre-clinical product progress.

We believe viral-vector gene therapies are here to stay, and our cutting edge pre-clinical research is essential for the development of the successful gene therapies of the future. It is exciting to see our AAV-based therapeutic development expand from rare diseases like Huntington’s and Pompe to now include more common diseases like congestive heart failure and Parkinson’s disease. The results of our Phase 1b study investigating AB-1005 as a potential treatment for Parkinson’s disease were recently presented at the AD/PD 2023 Conference on Alzheimer’s and Parkinson’s Diseases.

Increasing demand requires gene therapy companies to overcome barriers, particularly those related to production. Manufacturing AAV-based therapeutics is a highly complex, science-intense process. With our subsidiaries Viralgen and TAAV, we have focused a great deal on the challenges associated with efficient production of gene therapies, which impact the entire industry. Manufacturing for clinical trials and at scale will enable AskBio and others to get new gene therapies to the people who need them the most. We understand that developing potentially transformative therapeutics is only half of our job. We at AskBio are equally committed to doing the work needed to get these therapies to patients. Development and delivery are paramount to us, and this is a commitment we share with our colleagues at Bayer, a critical partner in accelerating the future of gene therapy.

With each day at AskBio, I feel more confident we are getting closer to a time where we will be able to have gene therapies that will treat the untreatable and improve conditions not just incrementally, but profoundly. And we’ll achieve this through scientific advances we could not have imagined only a few years ago.  

I am energized by the commitment of our world-class team at AskBio and our potential to not only have the answers to the many questions patients have when faced with a diagnosis, but also the gene therapy solution. I look forward to being even more inspired by the strength and determination of the gene therapy community at #ASGCT23. See you there!