We are the future of gene therapy
We are a team with decades of proven success in translating science from bench to bedside across many companies and medicines, in gene therapy and beyond. We are driven by a purpose: to make a lasting positive impact in the lives of people affected by devastating rare and prevalent diseases where the genetic cause is understood.
Our thesis is simple and our vision, clear: gene therapies have an immense potential to cure many diseases with a one-time dose, but they are limited by current delivery methods for the genetic payload. We formed Affinia Therapeutics to singularly focus on creating innovations that can expand the reach of gene therapies to more prevalent diseases. We’re re-engineering adeno-associated virus serotype 9 (AAV9), a gene therapy delivery method that has shown promise but only in a small number of diseases.
Propelling the promise of gene therapy, with purpose
We use AI, structural modeling, and other cutting-edge scientific tools to methodically re-design AAV9, enabling it to better target tissues and cells that are the desired sites of efficacy while avoiding sites of toxicity. Our goal is genetic medicines that can be given as a one-time dose to effectively and safely correct defective genes in the body – the underlying root cause of many diseases. We dream of using the word “cure.”
At Affinia Therapeutics, we call it ART: Affinia Rationally-designed Therapies. ART can benefit any tissue of interest such as the eye, heart, kidney, liver, lung, muscle, nervous system, or pancreas. ART can benefit any type of gene defect such as one over-producing or under-producing a protein or one producing the wrong type of protein.
Most importantly, we know it starts with the patient in mind. That’s why our initial work is focused on diseases of the heart and of the nervous system – this is where the patient need is high, the limitation for gene therapy is one of targeted delivery, and our ART platform has solutions.
Whether through small steps or giant leaps, we’re expanding the reach of gene therapies and bringing hope to people living with a wider range of devastating diseases.
Revolutionizing capsids
We design novel capsids that better target tissues and cells that are the desired sites of efficacy while avoiding sites of toxicity. We do this by re-engineering AAV9 as it’s been shown to be safe in people and has worked in a small number of diseases. We incorporate short peptides into the AAV sequence that serve as homing beacons for specific tissues, while avoiding others. We also check that the capsids are safe and manufacturable and translate from animals to people. These novel AAV-peptide capsids are specific for the tissues relevant to the disease we are trying to treat.
Reimagining payloads
We construct innovative therapeutic payloads that serve as the genetic message to treat the disease. The message is written in a ‘code’ the cells in the body understand. It can ‘program’ the cells to start making a new protein that it didn’t make before (gene replacement), or to interfere with the cell’s ability to make a specific protein that’s causing disease (knockdown), or to change the genes of cells (gene editing). It can ‘program’ cells to manufacture their own medicines (e.g., vectorized antibody). To get the best therapeutic payload for a disease, we may form strategic partnerships with other companies or academic institutions.
Innovating manufacturing
We develop new manufacturing approaches to improve the quality, yield, and cost of gene therapies. We explore new cell lines, plasmid designs, and transfection agents to support development of our next-gen gene therapies for not only rare diseases but also prevalent ones. We believe that manufacturing approaches are as critical as efficacy and safety in gene therapy. At every step of our research and development and in our selection of capsid, payload, and manufacturing approach, we have the patient in mind. We can’t wait to create the next transformative medicine.
