Accelerating Precision Oncology

Expanding Treatment and Improving Outcomes

Bringing Targeted Therapies
to Patients in Need

Leapfrog Bio is developing targeted therapies for cancers driven by genetic mutations that cannot be treated with conventional approaches. Our advanced pharmacogenomic approach yields novel insights into the biology of these cancers and simultaneously identifies small molecule compounds to effectively treat them. Our goal is to rapidly develop these compounds as genetically targeted therapies for cancer patients with high unmet medical needs.

Science

Two thirds of cancers are driven by genetic mutations that cause a critical gene not to function. These cancers can be treated by exploiting a biological phenomenon known as synthetic lethality. A synthetic lethality relationship exists when a cell will die from a loss of function of two genes, but not from the loss of function of just one or the other. If a cancer cell has a genetic mutation that turns off the functioning of one gene, there may be a paired gene that can be pharmacologically targeted to cause cancer cell death through synthetic lethality.

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Platform

Using CRISPR based gene editing and next generation sequencing, our Precision PGx Platform™ is able to discover synthetically lethal relationships between loss of function mutations and pharmacological drug effects. This creates an opportunity to directly identify drug treatments for cancers driven by loss of function mutations.

Using CRISPR based gene editing and next generation sequencing, our Precision PGx platform is able to discover synthetically lethal relationships between loss of function mutations and pharmacological drug effects. This creates an opportunity to directly identify drug treatments for cancers driven by loss of function mutations.

Through Leapfrog’s unique Precision PGx Platform we are able to evaluate a broad range therapeutic molecules against all significant loss of function mutations. This process allows us to quickly and efficiently identify known therapeutic agents that can be targeted against cancers with particular genetic mutations. Utilizing the existing data relating to these compounds, we can rapidly advance them through pre-clinical validation and into clinical development.

To test the insights generated from our platform, we retrospectively evaluated real world data to determine the performance of approved therapeutics against cancers with a range of genetic mutations. This evaluation consistently confirmed improved clinical efficacy of treatments predicted by our platform to be effective in specific genetically-defined patients.

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Pipeline

We are currently evaluating a number of highly promising therapeutic compounds identified by our Precision PGx Platform in standard pre-clinical models of cancers where there is a high unmet clinical need. By leveraging the existing data on these compounds, we anticipate that we will be able to initiate clinical trials in 2023.

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