according to Selecta said it intends to combine Anc80 with recently discovered transgenes and Selecta’s SVP-Rapamycin to create a novel gene Therapy candidate for MMA.
To advance the MMA program, Selecta last year entered into a Collaborative Research and Development Agreement (CRADA) with MEE and the NIH’s National Human Genome Research Institute.
Lonza’s viral-based therapeutics unit Lonza Houston has agreed to manufacture an Anc80-AAV–based gene Therapy product for Selecta Bioscience’s proprietary program for the treatment of methylmalonic acidemia (MMA) and may produce other Anc80-based products for which Selecta holds exclusive options, the companies said today.
Anc80-AAV, an in silico-designed synthetic gene Therapy vector, has generated preclinical data suggesting its potential to provide what the companies termed superior gene expression levels in the retina, liver, muscle, cochlea’s outer hair cells, and other tissue targets.
The vector was developed by the laboratory of Luk H. Vandenberghe, Ph.D., director of MEE’s Grousbeck Gene Therapy Center and an assistant professor at Harvard Medical School.
according to “We view Lonza – one of the industry’s largest contract manufacturers of biologics and a leading supplier in gene therapy – as an ideal partner.
These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved.
Selecta intends to combine Anc80 with recently discovered transgenes and Selecta’s SVP-Rapamycin to create a novel gene therapy candidate for MMA.
MMA patients are unable to process certain proteins and fats, leading to the accumulation of toxic metabolites.
“This agreement with Selecta Biosciences continues to demonstrate Lonza’s leadership position in the cell and gene therapy space,” said Andreas Weiler, Ph.D., Head of Emerging Technologies Business Unit for Lonza’s Pharma&Biotech segment.
Rare Muscle Disease Treated Successfully with Gene Therapy
according to Their most recent findings (“Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs”) were published online in Molecular Therapy.
The data supports the development of gene Therapy clinical trials for myotubular myopathy, the researchers concluded.
“We report here a gene Therapy dose-finding study in a large animal model of a severe muscle disease where a single treatment resulted in dramatic rescue,” said Dr. Childers.
Gene Therapy holds the promise to treat many inherited diseases.
The rare disorder, called X-linked myotubular myopathy, or XLMTM, affects only males.
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