Translating science into therapies for one or for many.
We develop a liberated set of tools and resources to expedite
The bottleneck for rare disease therapeutics is resource aggregation. Where gaps exist, we create new research assets - at cost and with extreme efficiency - and distribute them broadly to ensure that they are easily accessible to an organization’s entire scientific network.
We create a modality-agnostic research roadmap to get treatments to the clinic.
Leveraging our network of expert scientists, specialist laboratories, and leading biotechnology companies, we evaluate all feasible therapeutic approaches in parallel. We prioritize those that can be rapidly translated to the clinic regardless of future commercial upside.
The technology or drug to solve a disease may already be out there.
Our platform identifies intra-disease relationships that can benefit from similar or equal approaches or technologies. We continuously expand our knowledge base and know-how in every disease we are working on.
This is the decade of regenerative medicine and the democratization of drug discovery.
We run multiple therapeutic discovery pipelines in parallel through our network of expert collaborators and specialist laboratories.
Gene therapy replaces a mutated gene with a healthy copy, making it potentially curative rather than treating symptoms. We pursue a gene therapy approach for monogenic disorders where there is a feasible path forward and are partnering to access emerging technologies to overcome existing hurdles with delivery, toxicity and manufacturability.
Antisense oligonucleotides (ASOs)
ASOs are particularly promising for silencing the expression of harmful genes, for example in heterozygous mutation carriers where the mutated allele is a toxic gain of function or dominant negative. In these cases, an ASO can enable selective depletion of the mutated allele and deliver therapeutic benefit. We use a combination of genomics, computation, and wet lab science to design, synthesize, test, and validate oligonucleotide-based therapies.
Drug repurposing isn’t new, but it provides one of the most exciting outcomes for patients - a quick turnaround to treatment and a well known safety profile. We apply the latest advances in technology for functional assay development, including high-throughput screening and tools for extracting key data points and scientific knowledge through literature reviews. This helps us find new applications for novel therapeutics still in development at pharmaceutical companies and also find drugs that may be readily available at the pharmacy next door.
Decades of research and development has led to a solid infrastructure to screen, validate, and test small molecules. We combine advanced computational and high-throughput wet lab methods along with medicinal chemistry and pharmacology best practices to identify promising compounds. We perform screening in disease-specific cell lines or in model organisms and analyze the data with automated or AI-enabled systems.
Biologics such as recombinant proteins and antibodies have been successfully used to treat several rare disorders such as Gaucher disease, Fabry disease, and Pompe disease, as well as a number of inflammatory conditions. We develop therapeutic antibodies or enzyme replacement therapies when appropriate, and benefit from extensive existing infrastructure for biologics development and manufacturing.
For one or for many.
We fight to find therapies for a diverse group of disorders. These are some of the programs we are currently working on.
For us, it’s personal.
We're a passionate team focused on pushing the boundaries of translational medicine to help families and patient communities identify new therapeutic options with the highest probability of success.
Co-founder and CEO
Co-founder and COO
Chief of Staff
Elizabeth Iorns, PhD
Nicole Perfito, PhD
Director of Research Operations
Head of Partnerships
Amina Ann Qutub, PhD
We have a plan for everyone, let’s talk.
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Photography by: Ceridwen Hughes