Advancing Innovative Science

At Asklepios BioPharmaceutical, Inc. (AskBio) , we value our pioneering spirit. It is embedded in all we do and drives our commitment to developing innovative gene therapies. We aren’t alone in this pursuit and aren’t surprised to see the impact of the first of these to be approved for use. In fact, we’ve been working alongside our industry peers and look forward to delivering our own approved therapies as soon as possible. Our culture, company, structure, and our decision-making are all built around dreaming big for those with debilitating diseases, and we celebrate every gene therapy success, including our own milestones.

In 2023 alone, durable cell and gene therapies achieved over a dozen approvals from the FDA with hundreds in development.¹ In November 2023, we saw the first regulatory authorization of CRISPR-based therapy in the world – CRISPR is unique gene editing technology for precise, directed changes to genomic DNA. Further, it’s predicted that by 2030, there’ll be more than 60 US approvals of cell and gene therapy products and more than 500,000 patients treated with gene therapies.² While those of us working in gene therapy have long seen the promise of these treatments, now we have some numbers to prove this is happening. Gene therapies for orphan diseases that enter Phase 1 trials have a two to three-and-a-half times higher likelihood of approval versus other modalities and continue to have a higher likelihood of approval in each following development phase. They are also two times as likely to be approved when entering Phase 1 as the average drug in similar therapeutic areas, such as hematology, autoimmune, metabolic, neurology, and ophthalmology, and they continue to outperform in the following development phases.¹

In the first three months of 2024, AskBio celebrated three milestones for our Phase 2 congestive heart failure and Phase 1b Parkinson’s disease trials. As we expect to focus on Phase 2 trials in these therapeutic areas in 2024, we’re excited about what this means and how this brings us another step closer to changing the lives of those living with these diseases.

It's important to appreciate the value of early-stage and small trials for gene therapy – gene therapies are often, but not always, developed for rare diseases and large trials are not possible since large patient populations aren’t available to take part. This means there is a need for health care providers to develop infrastructure to dose gene therapy patients, especially since gene therapies also often show early signs of clinical efficacy during these trials, resulting in accelerated development programs. Peter Marks and the FDA have done a remarkable job of working with patient advocates and drug developers through accelerated approvals and expanded treatment options. Clinical trial requirements are changing and regulatory bodies, like the FDA, are changing the way they place value on this, considering innovative clinical trial designs. Combining the Phase 1, 2, and 3 processes and post-approval trials for gene therapies are becoming more common, and regulatory bodies have issued guidance documents that address new clinical trial designs.

However, understanding and celebrating innovative trials and moving into Phase 2 doesn’t mean AskBio is focused on research alone. We know that delivering any successful treatment in a commercial landscape is as important as development. Bringing complex new treatments to patients requires close collaboration with regulators and globally aligned regulatory pathways, which are critical for the advancement of gene therapies and commercialization. Preparing for delivery to patients is a large part of our investment.

As we continue to watch the scientific, regulatory, and commercial success of gene therapies, we’re excited to be one of the organizations breaking barriers at the forefront of the effort to bring hope to those who often have none. We expect more milestones this year, next year, in 2030, and beyond. Join us for the journey.

¹ Tufts Medical Center. Available at: https://newdigs.tuftsmedicalcenter.org/are-cell-and-gene-therapy-programs-a-better-bet/. Accessed March 2024.

² Drago D, Foss-Campbell B, Wonnacott K, Barrett D, Ndu A. Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs. Mol Ther Methods Clin Dev. 2021;21:524-5292.