Jnana Therapeutics Inc. has synthesized sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of amino acid metabolism disorders, amino acid transport disorders and metabolic and nutritional genetic disorders, among others.
Jnana Therapeutics Inc. has identified sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatohepatitis, phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism spectrum disorders, among others.
Work at Jnana Therapeutics Inc. has led to the identification of sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, metabolic dysfunction-associated steatohepatitis (MASH), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental and autism spectrum disorders, among others.
Phenylketonuria (PKU) is an inborn error of metabolism caused by heritable phenylalanine hydroxylase gene mutations that result in decreased metabolism of phenylalanine (Phe) causing brain damage. The most severe phenotype termed PKU occurs when untreated individuals achieve plasma Phe concentrations of >1200 microM/L, which are neurotoxic.
Phenylketonuria (PKU) is an autosomal recessive disorder where the primary catabolic pathway for phenylalanine (Phe) is disrupted due to mutations in the gene encoding PAH. Elevated Phe levels lie behind several neuropathologic anomalies that can lead to severe and irreversible mental retardation, if untreated.
Belharra Therapeutics Inc., an early stage firm which is pioneering a new approach to chemoproteomics, has already found an influential backer for the distinctive way it systematically probes the interactions between small-molecule ligands and the proteins to which they bind.
Momentum is building at Jnana Therapeutics Inc. The firm has raised $107 million in a series C round and banked another $50 million up front from a new drug discovery and development pact with Roche Holding AG, which could potentially deliver significant near-term milestones and more than $2 billion in future milestone payments. It also commenced recruitment onto a first-in-human study of its lead drug candidate, JNT-517, an inhibitor of the phenylalanine transporter SLC6A19, which is in development for phenylketonuria.
Jnana Therapeutics Inc. closed a $50 million series B round and took the covers off its lead program, a small-molecule inhibitor of the solute carrier transporter SLC6A19, which it is prepping for clinical trials in phenylketonuria (PKU).
DUBLIN – Roche Holding AG has joined Jnana Therapeutics Inc. in its quest to drug the human repertoire of solute carrier (SLC) metabolite transporters as a means of tackling a swath of immune-mediated and neurological diseases. The Basel, Switzerland-based pharma is paying Boston-based Jnana $40 million up front and could pay over $1 billion more in research funding, preclinical, development and commercial milestones, as well as sales royalties, in a multitarget deal.