Our Motivation
Gene therapy offers a transformative treatment strategy for patients with rare and ultra-rare genetic disorders. Advances in platform technologies have created unprecedented opportunities to develop treatments for hundreds of genetic disorders.
Despite the initial clinical successes and continued innovation largely driven by academic research funded by the National Institutes of Health (NIH), there remains a significant unmet need in getting these transformational therapeutics into the clinic for patients with rare disease. With more than 10,000 identified rare diseases, the need for innovative therapies has never been more urgent.
One of the immediate and key challenges in the rare disease gene therapy field is the limited access to innovative platforms. Cutting-edge gene therapy platforms, such as next-generation AAV vectors, optimized delivery systems, and validated preclinical models are often locked behind exclusive licensing agreements or siloed within proprietary industry pipelines. This creates a significant barrier for academic researchers, small foundations, and N-of-1 patient families, who are eager to develop treatments, but lack the means to access or apply next-generation technologies. As a result, promising therapeutic ideas for rare and ultra-rare diseases can be hindered not due to lack of scientific merit, but because the tools required to advance them are out of reach.
Addressing the access gap is critical to democratizing gene therapy for rare disease and cutting a path forward for under-served patient populations. This is our mission.
Our Approach
As novel gene therapy (GT) platforms continue to accumulate at a rapid pace, we must build a sustainable framework for translating these innovations in the clinic with the goal of replacing outdated modalities with safer and more potent medicines that truly deliver on the promise of transformative medicines for rare disease. But, without improved dissemination and adoption of innovative technologies starting at the early preclinical stages of drug development, the field risks reinforcing a cycle of stagnation and clinical disappointment.
There remains a significant unmet need in mechanisms that increase the influx of cutting-edge technology platforms into preclinical and clinical trial pipelines for treating rare and ultra-rare diseases.
To combat this “innovation gap”, the ATG Project will focus on enabling access to gene therapy innovation and services to accelerate the development of cutting edge and transformative medicines for rare disease. The ATG project is a 501(c)(3) non-profit initiative based on a unique model that pairs a nonprofit entity (ATG) with academic innovation and services, providing open access to a large and versatile portfolio of AAV GT intellectual property, along with preclinical gene therapy design and manufacturing services (Gene Therapy Resource Core, GTRC). ATG is also assembling a deep advisory network to provide scientific, clinical, and regulatory guidance. Platforms and services will be bundled at subsidized costs to accelerate rare disease gene therapy development. While academic research leading to innovative GT platforms and services will continue to grow within the academic environment supported by federal funding, the ATG Project will leverage partnerships with service industry (CROs and CDMOs) and philanthropic funding to provide access to IP and services to rare disease foundations, individual clients and families affected by rare disease as well as academic researchers working with these entities to develop new rare disease GT approaches.
Our Team
We are a group of motivated citizens and scientists, committed to enabling access to innovation.
