Partner With Us
Fostering collaborations to break barriers to innovation
Delivery is the bedrock of gene therapy
Driven by our commitment to transform patient lives, we aim to make gene therapy safer, more effective, and more accessible by developing next-generation delivery technologies that are available for out-licensing, beginning with our proprietary capsid, AAV-SLB101. We are on a mission to create a suite of effective tools designed to be highly targeted and applicable to neuromuscular and cardiac indications, and that are supported by extensive preclinical data to help you accelerate development.
Together, we can revolutionize gene therapy.
Therapeutic Pipeline
Immunomodulatory Strategies
Innovative Delivery Technologies
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FOCUSED
Robust transduction, biodistribution and expression achieved in preclinical mouse and non-human primate (NHP) models, and in initial patients dosed in Phase 1/2 INSPIRE DUCHENNE clinical trial (MDA 2025)
PATIENT CENTRIC
Reduced liver exposure vs. AAV9 in mice and NHPs translated to promising early safety and tolerability seen in initial patients dosed in INSPIRE DUCHENNE trial (MDA 2025)
COLLABORATIVE
Comprehensive preclinical data available to support regulatory discussions and de-risk development
CENTER OF EXCELLENCE
Express MTAs and flexible licensing available to accelerate access for academic and corporate partners
AAV-SLB101: Next-Generation Cardiac & Muscle-Tropic Capsid
Robust transduction, biodistribution and expression achieved in multiple animal models and in initial participants dosed in the Phase 1/2 INSPIRE DUCHENNE clinical trial (NCT06138639)
Reduced liver biodistribution vs. AAV9 in mice and NHPs, and no biomarker or clinical evidence of liver injury in INSPIRE DUCHENNE study participants (data cutoff March 7, 2025; n=7)
Comprehensive preclinical data available to support regulatory discussions and de-risk development
Express MTAs and flexible licensing available to accelerate access for academic and corporate partners
Research Tools (Capsids)
The development and adoption of novel capsids remain hindered by siloed research, restricted access to proprietary vectors, and a lack of standardized comparisons across platforms. We aim to break these barriers by fostering open and collaborative partnerships that streamline capsid evaluation and accelerate innovation.
Our proprietary capsid is cardiac and muscle-tropic, liver-detargeted, and has demonstrated clinical safety and efficacy. Partners can leverage our extensive preclinical data to support regulatory discussions and de-risk development efforts. We recognize the inefficiencies in the current model—where each organization independently tests capsids with limited cross-comparability—and are committed to enabling more transparent, data-driven benchmarking.
To facilitate access, we offer an express MTA for academic researchers and a flexible licensing structure for industry partners. Whether you are looking to incorporate a clinically validated capsid into your program or compare its performance in your system, we welcome discussions on how our approach can support your work.
Partnership agreements with universities, biotechnology companies and other industry partners for use of AAV-SLB101 as of March 2, 2025
View recent scientific presentations:
MDA 2025
View corporate presentation:
Therapeutic
We are advancing programs in syndromic and non-syndromic cardiac conditions as well as muscular and neuromuscular skeletal diseases. While these are priority areas, we remain open to innovative and transformative opportunities beyond cardiac and skeletal. We are particularly interested in programs that go beyond single-gene corrections and offer scalable potential across multiple conditions. Lead or development-stage assets are preferred, though we are open to earlier-stage platform technologies.
Immunomodulatory Strategies
We are exploring novel approaches to overcome immune challenges that currently limit the reach of gene therapies. This includes strategies to enable redosing, address pre-existing immunity (e.g., AAV seropositivity), and improve safety and efficacy. We welcome partners developing tools or technologies that modulate immune responses to broaden patient access and enhance therapeutic durability.
Innovative Delivery Technologies
We are seeking advanced delivery platforms—both viral and non-viral—that improve tissue targeting, overcome biological barriers, and enhance therapeutic precision. Areas of interest include LNPs, engineered capsids, and other novel vehicles capable of delivering genetic medicines efficiently to muscle, heart, and beyond. We are also interested in manufacturing-enabling technologies that reduce costs and improve scalability.