Lysosomal Storage Disorders

Polaryx Therapeutics Receives FDA Fast Track Designation for PLX-200 for the Treatment of Patients with Juvenile Neuronal Ceroid Lipofuscinosis

PARAMUS, N.J., Aug. 20, 2020 /PRNewswire/ — Polaryx Therapeutics Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3) patients. JNCL/CLN3 is …

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New England Journal of Medicine Publishes Positive Phase 3 Evinacumab Results in Patients with Severe Inherited Form of High Cholesterol

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the New England Journal of Medicine (NEJM) published positive results from the Phase 3 trial of evinacumab in 65 patients with homozygous familial hypercholesterolemia (HoFH). Evinacumab is an investigational medicine that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), and is the first medicine of its …

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Penn Study Verifies Human Gene Therapy in Model of Rare Metabolic Disorder

PHILADELPHIA — Researchers are closer to finding a better way to treat children with a rare metabolic disorder called MPS I. It is caused by a deficiency of the key enzyme IDUA needed to break down complex sugars in cells. MPS I eventually leads to the abnormal accumulation of sugar …

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FDA Clears Sangamo Biosciences’ IND Application of SB-318 for Treatment of MPS I

RICHMOND, Calif., Feb. 8, 2016 /PRNewswire/ — Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type …

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FDA Grants Breakthrough Therapy Designation to Vtesse’s Drug for Treatment of a Rare Genetic Disease

GAITHERSBURG, Md., Jan. 6, 2016 /PRNewswire/ — Vtesse, Inc. announced today that the U.S. Food and Drug Administration (FDA) has granted its drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), Breakthrough Therapy designation status. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status …

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Breakthrough Status Granted to Genzyme’s Rare Enzyme Replacement Drug

Genzyme, a Sanofi company, recently announced that its investigational enzyme-replacement therapy was granted Breakthrough Therapy designation from US health regulators. The company said that the agency granted the status to its olipudase alfa for the treatment of patients with nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick …

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FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …

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