Targeting difficult-to-drug targets US$50 million to help develop next-generation small molecule discovery platforms

On August 18, 2021, Jnana Therapeutics announced the completion of a US$50 million Series B financing
.

The funds obtained will be used to advance the main oral small molecule project for the treatment of phenylketonuria (PKU) into clinical development, while advancing the development of other projects in the pipeline

PKU is an inherited metabolic disease caused by a deficiency of phenylalanine hydroxylase (PAH)
.

Phenylalanine (Phe) is an amino acid present in all protein-containing foods, and PAH is an enzyme necessary for the decomposition of Phe

Jnana's pilot project aims to reduce the level of Phe in the blood that drives the pathology of PKU disease by inhibiting SLC6A19, so as to provide a new oral small molecule therapy for the treatment of PKU
.

The data currently obtained from human biology and in vivo experiments in animal models show that this method has the potential to treat PKU and can bring benefits to patients

▲RAPID platform operation mechanism (picture source: Jnana company official website)

The structure and intracellular location of SLC transporters are highly diversified, usually difficult to express and purify, not easy to analyze using biophysical methods, and there may be no known ligands
.

The RAPID technology platform only discovers candidate compounds based on the ability of small molecules to bind proteins, which provides a promising method for processing challenging SLC targets

Dr.
Joanne Kotz, Co-founder, CEO and President of Jnana Therapeutics said: "The support of investors allows us to advance a wide range of pipeline projects into the clinic and continue to expand the application of our platform.

Jnana's pilot project provides an oral The differentiated method of small molecule treatment of PKU can be used to treat any patient with PKU, regardless of their age or the presence of PAH gene mutations
.

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Reference materials:

Reference materials:

[1] Jnana Therapeutics Closes $50 Million Series B Financing To Advance Lead PKU Program And Small Molecule Pipeline Based On Next Gen Chemoproteomic Platform.

Retrieved August 18, 2021, from https:// therapeutics-closes-50-million-series-b-financing-to-advance-lead-pku-program-and-small-molecule-pipeline-based-on-next-gen-chemoproteomic-platform/

[1] Jnana Therapeutics Closes $50 Million Series B Financing To Advance Lead PKU Program And Small Molecule Pipeline Based On Next Gen Chemoproteomic Platform.
Retrieved August 18, 2021, from https:// therapeutics-closes-50-million-series-b-financing-to-advance-lead-pku-program-and-small-molecule-pipeline-based-on-next-gen-chemoproteomic-platform/