Drugs

Sangamo BioSciences IND Approved for Hunter Syndrome Agent

FDA has approved a Sangamo BioSciences IND for SB-913, a zinc finger nuclease (ZFN)-mediated approach designed as a single treatment with the potential to provide a long-lasting therapy for Mucopolysaccharidosis Type II (MPS II, Hunter syndrome). The company is planning a Phase 1/2 clinical trial to assess the therapy’s safety, tolerability and efficacy in adults with MPS II. “Unlike conventional, non-integrating AAV [adeno-associated virus] gene therapy that has the potential to ‘wash out’ over time as the patient’s liver cells divide and turn over, SB-913 has the potential to provide a uniquely durable solution through genome editing,” the company says. “Ultimately, our target population will include pediatric patients who can benefit from a more durable solution.”

Sangamo says MPS II is an X-linked recessive lysosomal storage disorder that occurs almost exclusively in males. “It is caused by mutations in the gene encoding the iduronate 2-sulfatase (IDS) enzyme, resulting in a deficiency of IDS which normally degrades the glycosaminoglycans (GAGs), dermatan sulfate and heparan sulfate,” it says. “The inability to degrade GAGs leads to their accumulation within lysosomes throughout the body, and individuals with IDS mutations experience multi-organ dysfunction and damage. The rate of progression and degree of symptoms depend on the severity of the mutation.”

SB-913 was designed based on Sangamo’s In Vivo Protein Replacement Platform (IVPRP) approach, and is a single treatment strategy designed to produce continuous, durable therapeutic levels of IDS, the enzyme that is missing or defective in patients with MPS II. “The IVPRP approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic ‘safe-harbor site,’ that can be edited with zinc finger nucleases (ZFNs) to accept and express any therapeutic gene,” the company says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome