A year of advancement

I have the privilege to work alongside individuals at AskBio who are firmly committed to pursuing life-changing science. Each day, I see the optimism and curiosity of our colleagues being applied to the work of potentially changing lives through gene therapy and delivering profound benefits for patients.

We have seen pioneering moments throughout 2023 that we believe can one day send waves of change across medicine. Among our highlights were several key patient firsts, including the first patient dosed in our Phase 1/Phase 2 trial for limb-girdle muscular dystrophy and the first participant randomized in our Phase 1 trial for multiple system atrophy. Looking ahead to 2024, we have more to be excited about, as we anticipate advancing two key programs to Phase 2.

Our becoming a Phase 2 gene therapy company will be an important achievement and the result of the work of our Research & Development, Technology Development & CMC, Therapeutics, and many other teams. This will also be a moment that we believe can bring tremendous hope to patients. Our advancing our clinical programs demonstrates our ability to work with urgency to deliver our therapies to the people who need them. These highly visible accomplishments matter a great deal, and they are a significant source of pride for all of us at AskBio.

Along with the advancement of our clinical programs, we also saw progress in our manufacturing capabilities, with our subsidiary Viralgen being licensed for the commercial manufacturing of recombinant adeno-associated virus (rAAV) gene therapies.

As an end-to-end gene therapy company, active from discovery through to commercial manufacturing, another important aspect of our work in 2023 was to align our leadership structure with the role AskBio anticipates playing as we bring our therapies closer to potentially helping those living with some of the world’s most debilitating diseases. This involved the creation of new roles and will ensure that we will be prepared to build on AskBio’s years of groundbreaking discovery experience and improve the company’s ability to bring its clinical-stage gene therapies to people around the world.

Our industry is realizing the promise of gene therapy, and AskBio is fully focused on doing its part. In building on our significant scientific contributions to the advancement of AAV, we will continue to pursue our late-stage clinical aspirations. Ours is an exciting future, and we look forward to a memorable 2024 and beyond. My sincere thanks go to everyone inside and outside of our company working to help make this possible.