Category REGENERATIVE MEDICINE

The University of Arizona Center for Innovation in Brain Science has received a $37.5 million grant from the National Institutes of Health to research a potential regenerative therapy for Alzheimer’s disease.

A team led by Roberta Diaz Brinton, PhD, director of the Center for Innovation in Brain Science, received the multi-million dollar grant from the National Institute on Aging. The five-year grant will fund a national multi-site Phase 2 clinical trial to determine the effectiveness of allopregnanalone, or allo, as a treatment for individuals with early-stage Alzheimer’s who

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Pitt’s McGowan Institute aims to study stem cells, 3D printers in space

The University of Pittsburgh is teaming up with the International Space Station U.S. National Laboratory (ISSNL) to study how the low-gravity environment of space can improve treatments for kidney disease, traumatic brain injury and juvenile diabetes. Pitt’s McGowan Institute for Regenerative Medicine will collaborate with the ISSNL Industrial Biomedicine Program on research that will look

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Health Technology Assessment of Gene Therapies in Europe and the USA: Analysis and Future Considerations

Gene therapies constitute a new concept of transformative therapies, administered once in a lifetime. The value assessment of these innovative therapies constitutes a challenge for health technology assessment (HTA) bodies. The HTA reports for all seven gene therapies that have to date been granted a market authorization in the European Union (EU) and/or the United

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Heart regeneration: Scientists uncloak one of nature’s deepest secrets

Scientists at the University of Texas Southwestern have uncloaked the molecular mechanisms underlying heart regeneration in the only mammals currently known to regrow a significant portion of their hearts after injury: neonatal mice. These newborns have long held the secret to regenerating approximately 15 percent of their ventricular tissue, a capability that occurs within a fleeting window of about

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Brain disease drug delivery platform developed by researchers

So-called ‘nanodiamonds’ could serve as a platform for diagnostic and therapeutic approaches for neurodegenerative diseases. Scientists at the Max Planck Institute for Polymer Research (MPI-P) have produced tiny diamonds, so-called ‘nanodiamonds’, which could serve as a platform for both the therapy and diagnosis of brain diseases. Led by Dr Jana Hedrich, Professor Heiko Luhmann and

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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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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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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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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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