Lysosomal storage disorders are a group of rare inherited metabolic disease.
Transcription Factor EB (TFEB) is a master regulator of lysosome biogenesis. Increasing evidence has defined the crucial role of TFEB-dependent lysosome biogenesis on both lysosomal storage disorder (LSDs) and neurodegenerative disorders, including Alzheimer’s disease, Parkinson’s disease, and Huntington’s disease. An attractive approach to eliminate toxic aggregates in both LSDs and neurodegenerative disorders is to upregulate the cellular clearance of these wastes through induction of the lysosomal machinery.
Polaryx® Therapeutics utilizes either new drugs or repurposed drugs to tackle these diseases, and all of them are uniquely formulated. The company is currently preparing to perform a clinical trial to confirm the safety and efficacy of its drug candidates.
There is an urgent need for novel therapeutic interventions for lysosomal storage disorders, including neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases. That’s where Polaryx® comes in.Learn More
Discover our unique investigational methodology that Polaryx® is pursuing to treat lysosomal storage disorders, including neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases, non-invasively.Learn More
Learn about key mechanism of action of our drugs and about our development strategy.Learn More
Meet the team
With over 100 years of combined experience, we are ready to take on lysosomal storage disorders, including neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases.Learn More
Learn more about TFEB-dependent lysosome biogenesis, our key mechanism of action (MOA) to clear cellular wastes.Learn More
with novel and
Backed by an
Expanding to neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases.
Polaryx® Therapeutics Received Orphan Drug Designation From the European Medicines Agency (EMA) for the Treatment of All Neuronal Ceroid Lipofuscinosis With PLX-200
Click "read more" now to view the press release.
Renowned pharmaceutical industry publication, Biopharma Dealmakers, has featured Polaryx® Therapeturics in their June 2017 edition. Click "read more" now to view the publication.
To introduce Polaryx® and its technology, GHP magazine has interviewed the Polaryx® team. Click now to see the June 2017 GHP magazine edition. Polaryx® is featured on pages 26-27.
Polaryx® Therapeutics Receives Orphan Drug Designation From the U.S. FDA for PLX-200 for Rare Pediatric Disease
This new drug candidate will be used for the treatment of patients with Late Infantile Neuronal Ceroid Lipofuscinosis.
Polaryx® Therapeutics Receives Orphan Drug Designation From the US FDA for the Treatment of Neuronal Ceroid Lipofuscinoses With PLX-100
Polaryx® Therapeutics, Inc. is a biotech company developing patient-friendly oral small molecule therapeutics for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and other forms of NCL, commonly known as Batten disease. Neuronal Ceroid Lipofuscinoses qualify as rare pediatric diseases under Section 529 of the Food, Drug, and Cosmetic Act.