About Affinia Therapeutics

Our story

We are the future of gene therapy

At Affinia Therapeutics, we’re singularly focused on using innovations in gene therapy to lead the fight against devastating rare and prevalent diseases. We're working to find solutions to the limitations of conventional AAV capsids, promoters, and manufacturing approaches to improve tissue targeting, cell expression, packaging efficiency, and manufacturing quality, yields, and scalability. We stand determined to expand what gene therapy can and will do.

It all started with breakthrough science led by our scientific founder, Luk Vandenberghe, Ph.D., a pioneer in the field and inventor of AAV9, his student, Eric Zinn, Ph.D., and team, who brought their passion to the field of AAV gene therapy to advance it toward its full potential. Using computational design and high-throughput screening methods, a deep understanding of the underlying structure of the AAV was achieved and a new class of AAV capsids was enabled - one with novel properties and therapeutic possibilities vs. traditional capsids. Together with fellow pioneers Botond Roska, M.D., Ph.D., an expert in the design of next-generation promoters, and Aaron Tward, M.D., Ph.D., renowned for his expertise in high-throughput sequencing technologies, they founded Affinia Therapeutics. We’ve assembled a team of experienced industry leaders who want to see gene therapy's full potential realized. Together, we’re pioneering a new class of gene therapies.

At Affinia Therapeutics, we call it ART: Affinia Rationally-designed Therapies. Our platform for rational design of capsids, promoters, and manufacturing approaches is designed to benefit any genetic modality employed, such as gene replacement, vectorized antibody, gene editing, or RNA knockdown. Similarly, our ART platform is designed to be applicable to any tissue of interest including the CNS, muscle, lung, heart, kidney, liver, or eye.

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Propelling the promise of gene therapy, with purpose

Whether through small steps or giant leaps, we strive each day to advance the gene therapy field with our expertise, experience, and collaborative thinking. Using our proprietary platform, we design and develop capsids and promoters to methodically alter how gene therapies work in the body, and novel manufacturing approaches to improve quality, yields, and scalability. We’re expanding the utility of gene therapies as a transformative delivery mechanism for therapeutics to reach new tissues and address new diseases. We’re purposeful in what we do, driven to bring the hope of gene therapy to people living with a wider range of devastating diseases.

What we’re working on

Affinia Therapeutics is committed to transforming gene therapy with our platform and pipeline — to push science forward, to impact human medicine, and to give patients the options they deserve.

Our pipeline
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Revolutionizing AAV capsids

Capsids are the outer protein shells of AAVs. We're using analytics and machine learning algorithms on the evolutionary patterns of naturally occurring AAVs and proprietary structure-function datasets we've generated to rationally design novel capsids. Our capsid platform, cART, leverages ancestral sequence reconstruction to design novel capsid libraries with improved tissue targeting, immunologic profile, and manufacturability.

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Reimagining promoter design

Promoters are a kind of regulatory element in a gene therapy that controls where the therapeutic gene inside a gene therapy is expressed. Our promoter platform, pART, leverages computationally designed libraries of novel promoters to precisely modulate gene expression in specific cell types. Together with the targeting capacity of our proprietary capsids, our disease-optimized promoters will further control where the vector is active, which may increase therapeutic efficacy and decrease off-target effects.

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Streamlining manufacturing

We're innovating manufacturing approaches using our mART platform, to improve quality, yields, and manufacturing scalability to support rare and prevalent diseases.

Our vision

Affinia Therapeutics is setting a new standard in gene therapy, using our deep expertise to develop tomorrow’s restorative treatments.