Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2019-09-05

Magenta Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to MGTA-456 for the Treatment of Inherited Metabolic Disorders

 Magenta Therapeutics, a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advance Therapy (RMAT) designation for MGTA-456, a one-time cell therapy for the treatment of multiple inherited metabolic disorders. “This RMAT designation was based

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No Comments InCELL THERAPY
PIER MARIA FORNASARI
2019-09-04

Don’t Believe Everything You Hear about Stem Cells

The science is progressing rapidly,but bad actors have co-opted stem cells’ hope and promise by preying on unsuspecting patients and their families Source Scientific American By Deepak Srivastava on September 4, 2019 Stem cell science is moving forward rapidly, with potential therapies to treat intractable human diseases on the horizon.Clinical trials are now underway to test the safety

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No Comments InREGENERATIVE MEDICINE
PIER MARIA FORNASARI
2019-09-04

A Comparison of Phenotypic and Functional Properties of Mesenchymal Stromal Cells and Multipotent Adult Progenitor Cells

Both multipotent adult progenitor cells and mesenchymal stromal cells are bone-marrow derived, non-hematopoietic adherent cells, that are well-known for having immunomodulatory and pro-angiogenic properties, whilst being relatively non-immunogenic. The authors compare the phenotypic and functional properties of these two cell types, to help in determining which would be the superior cell type for different applications.

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No Comments InREGENERATIVE MEDICINE
PIER MARIA FORNASARI
2019-09-04

Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 × 10^12 GC/kg Dose as Optimal Dose for Phase 3 Study Ultragenyx Pharmaceutical, a

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No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2019-09-03

Four U.S. CRISPR Trials Editing Human DNA to Research New Treatments

Breaking down how the gene editing technology is being used, for the first time in the United States, to treat patients with severe medical conditions Source Smithsonian.com Last fall, the birth of genetically edited twin girls in China—the world’s first “designer babies”—prompted an immediate outcry in the medical science community. The change to the twins’ genomes,

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No Comments InCRISPR
PIER MARIA FORNASARI
2019-09-02

Gene Therapy Shows Promise for AADC Deficiency But Requires Early Diagnosis, Study Says

Early clinical trials of a gene therapy for aromatic amino acid decarboxylase (AADC) deficiency have yielded encouraging results for its safety and efficacy, a study highlights. But researchers call attention to the importance of a timely diagnosis for treatment success, as only early interventions could fully prevent long-term brain damage and other outcomes. Their study, “Aromatic amino

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No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2019-08-31

Pancreas on a chip

Harvard scientists have combined organ-on-a-chip and stem-cell technologies to make a powerful tool for diabetes research and beta-cell transplantation By combining two powerful technologies, scientists are taking diabetes research to a whole new level. In a study led by Harvard University’s Kevin Kit Parker and published in the journal Lab on a Chip on Aug. 29, microfluidics and human,

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No Comments InORGAN ON CHIP