Category REGENERATIVE MEDICINE

Letter to the Duchenne Community: Gene Therapy IGNITE DMD Status and New Clinical Sites

Dear Duchenne Community, We want to provide you with an update on the progress of SolidBiosciences IGNITE DMD Phase I/II clinical trial for our investigational microdystrophin gene transfer, SGT-001. Since we communicated preliminary clinical data in February, we advanced the study to evaluate SGT-001 at a higher dose in the second cohort of patients. As

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German Ethics Council: germline interventions currently too risky, but not ethically out of the question

Last year, the birth of the first genetically modified babies shook the world. The German Ethics Council now presents a comprehensive ethical investigation into possible interventions in the genome of human embryos or germ cells. The Council does not deem the human germline to be inviolable. It does, however, consider germline interventions to be ethically

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Queen’s University Belfast researchers discover revolutionary stem cell treatment

**For the first time, researchers have developed a non-intrusive way to generate large quantities of stem cells using only a small amount of blood** **The stem cells can repair cells damaged as a result of vascular diseases, which has the potential to prevent blindness and reverse the need for amputations** A discovery by researchers at

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The Alliance for Regenerative Medicine Releases Q1 2019 Data Report, Highlighting Sector Trends and Metrics

The Alliance for Regenerative Medicine (ARM) today released its most recent quarterly data report, offering an in-depth look at cell therapy, gene therapy, tissue engineering, and broader global regenerative medicine sector trends and metrics in the first quarter of 2019. By further curating information provided by ARM’s data partner Informa, the quarterly data report details

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Researchers uncover mechanism blocking retina regeneration

A discovery opens the possibility of one day restoring loss of vision by activating the retina’s ability to regenerate. Researchers at Baylor College of Medicine, the Cardiovascular Research Institute and the Texas Heart Institutereveal in the journal Cell Reports that although the mammalian retina – a layer of specialized nerve cells that mediates vision and is located on the back

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Oversight of Regenerative Medicine: FDA’s new regulatory approach

Regenerative medicine—a field that includes stem cells, bioengineered tissues, and gene therapies—holds great potential. In November 2017, the Food and Drug Administration updated its regulatory framework for these products. Peter Marks, who leads the agency’s Center for Biologics Evaluation and Research (CBER), about the framework gave this interview. Q: What led FDA to update its regulatory framework

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