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Find Drugs for Genetic Disease in Phase II/ Phase III Clinical Development
Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.
The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.
QRX003 is a once-daily topical lotion comprised of a broad-spectrum serine protease inhibitor, formulated with the proprietary invisicare® delivery technology. It is being investigated for treating netherton syndrome.
REN001 (mavodelpar) is a potent and selective peroxisome PPARδ agonist currently in clinical development for two rare genetic mitochondrial diseases that typically present with myopathy and PMM and LC-FAOD.
AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.
DNL126 (Recombinant Iduronate 2 Sulfatase) is a recombinant SGSH enzyme engineered to cross the blood-brain barrier, replace the SGSH enzyme and treat neuropathic and systemic forms of the Sanfilippo syndrome A.
Lead Product(s):
Recombinant Iduronate 2 Sulfatase
RGX-121 is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase gene which encodes the iduronate-2-sulfatase (I2S) enzyme to the CNS, allowing for long-term cross correction of cells throughout the CNS.
REGN3918 (pozelimab) is an investigational fully human monoclonal antibody designed to block the activity of complement factor C5, a protein involved in complement system activation, for the treatment for adults and children as young as 1 year of age with CHAPLE disease.
AT-007 (gavorestat) is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG.
Market Place
Company
Pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme. 
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