ROCKVILLE, Md.—REGENXBIO Inc., a clinical-stage biotechnology company seeking to improve lives through the potential of gene therapy based on its proprietary NAV Technology Platform, recently announced the appointment of Alexandra Glucksmann, Ph.D., to its board of directors, effective as of May 25, 2018.
“Dr. Glucksmann has been instrumental to the success of several start-up biotechnology companies over her 25-year career,” said Don Hayden, Chairman of REGENXBIO’s board of directors. “She has consistently brought deep scientific understanding, strategic insight and operational expertise to her many roles, and we are thrilled to welcome Dr. Glucksmann to our board.”
Previously, Dr. Glucksmann holds the position of President and Chief Executive Officer at Cedilla Therapeutics. She was formerly an Entrepreneur-in-Residence at Third Rock Ventures where she focused on company formation. Before that, she was a founding employee and the Chief Operating Officer at Editas Medicine. Glucksmann was also a founding employee and Senior Vice President of research and business operations at Cerulean Pharma.
Glucksmann began her career at Millennium Pharmaceuticals as a research scientist and held roles of increasing responsibility, culminating in the role of Vice President of platform technology groups, prior to transitioning into a senior position in strategic program management and operations. Dr. Glucksmann earned her Ph.D. in Molecular Genetics and Cell Biology from the University of Chicago and completed her postdoctoral fellowship at the Massachusetts Institute of Technology.
“REGENXBIO is an engine for discovery across the entire gene therapy field, with its own robust clinical development pipeline and multiple licensing agreements with other leading companies,” noted Glucksmann. “The potential for the company’s NAV Technology Platform is remarkable. It is an exciting time to be joining this dynamic team.”
In early May, REGENXBIO announced it had received Fast Track designation from the FDA for RGX-121, the company’s novel, one-time direct-to-CNS investigational treatment for Mucopolysaccharidosis (MPS) II, also known as Hunter syndrome, and is also developing RGX-111, a novel, one-time, direct-to-CNS investigational treatment for MPS I. The company expects to initiate dosing in clinical trials for RGX-111 and RGX-121 in mid-2018.
RGX-121 is being developed as a novel, one-time, direct-to-CNS treatment for MPS II that includes the NAV AAV9 vector encoding for human I2S. Delivery of the enzyme that is deficient within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. This strategy could also provide rapid I2S delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS II patients.
Treatment with RGX-121 has been shown to restore I2S enzyme activity in animal models of MPS II to levels equivalent to or greater than those in non-affected animals. The extent of CNS correction in animal studies suggests that RGX-121 has the potential to be an important and suitable therapeutic option for MPS II patients.
RGX-111 is being developed as a novel, one-time, direct-to-CNS treatment for MPS I that includes the NAV AAV9 vector encoding a gene for human IDUA. Delivery of the enzyme that is deficient within cells in the CNS could provide a permanent source of secreted IDUA beyond the blood-brain barrier, allowing for long-term cross-correction of cells throughout the CNS. This strategy could also provide rapid IDUA delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS I patients.
“At REGENXBIO, we are committed to collaborating with the entire MPS community, including patients, advocates and healthcare professionals, in search of treatments,” said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. “We are dedicated to creating a brighter future for children with MPS I and II and their families by developing potentially life-changing gene therapies for these two rare and debilitating diseases with significant unmet needs.”