Our Approach

Jnana is moving beyond the limitations of conventional drug discovery to develop transformational medicines for diseases with high unmet patient need.
RAPID, our proprietary drug discovery platform, enables us to efficiently identify small molecule modulators for previously undruggable targets.
We have built an internal pipeline of programs addressing highly validated, hard-to-drug targets for rare genetic diseases, immune-mediated diseases and oncology. We are also developing therapeutics with biopharma partners for immune-mediated and neurological diseases.
We are building a company where all employees are empowered to contribute and work together to achieve therapeutic breakthroughs. We are bolstered by our renowned academic founders and the backing of leading biotech investors.

Therapeutic Areas of Focus

Rare Genetic Diseases 

Rare genetic diseases, where there is a clear and compelling connection between target modulation and human disease biology, provide ideal opportunities for applying our RAPID platform.

Jnana’s lead program is focused on phenylketonuria (PKU), a genetic disease where loss of function mutations in the liver enzyme phenylalanine hydroxylase (PAH) result in a high systemic excess of phenylalanine (Phe) which drives neurocognitive and neuropsychiatric pathologies in patients. Systemic levels of Phe are regulated by SLC6A19 (B0AT1), a transporter responsible for Phe reabsorption in the kidney proximal tubule. Analogous to existing SGLT2 (SLC5A2) inhibitors that work by blocking glucose reabsorption in the kidney proximal tubule and have had substantial clinical impact, blocking reabsorption of Phe via inhibition of SLC6A19 is anticipated to significantly lower levels of plasma Phe.1 This differentiated approach has the potential to provide a first-in-class oral therapy that can be used to treat any PKU patient, regardless of age or PAH mutation.

JNT-517, Jnana’s Development Candidate for the PKU program, was derived from an allosteric inhibitor series identified by the company’s RAPID platform. A proprietary cryo-EM structure of SLC6A19 has recently revealed that Jnana’s allosteric inhibitors bind in an unprecedented cryptic allosteric site on the SLC transporter.


1 Inhibiting Neutral Amino Acid Transport for the Treatment of Phenylketonuria, Belanger et al., JCI Insight. 2018, 3(14).


A dysregulated immune system can pre-dispose an individual to infection, increase cancer risk and can give rise to inflammation and progressive organ damage when poorly controlled. Despite progress developing novel therapies, the burden of autoimmune and chronic inflammatory conditions remains high. We are taking a two-pronged approach to developing novel treatments for immune-mediated diseases.

Our RAPID platform is ideally suited to efficiently drug targets with strong validation from human genetics and other patient data, but that have proven recalcitrant to other discovery technologies. We are building a pipeline of programs addressing high value targets from diverse target classes, e.g. SLC transporters, transcription factors and signaling scaffold proteins, to create transformational new therapies.

In parallel, we are leveraging internal capabilities to conduct unbiased genetic screens in primary human immune cell subsets to discover new targets for the treatment of autoimmune and inflammatory conditions. We then deploy our RAPID platform to quickly advance programs against these promising, novel immune targets.


Despite meaningful advances in cancer therapy, there are still many tumor types that are not adequately addressed and large patient populations with significant remaining unmet need. Jnana is focused on applying our RAPID platform to previously undruggable, highly validated oncology targets across multiple historically challenging target classes.

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