Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2019-12-23

European Medicines Agency Validates BioMarin’s Marketing Authorization Application for Valoctocogene Roxaparvovec to Treat Severe Hemophilia A

Potential 1st Gene Therapy in Europe Directed at Any Type of Hemophilia Application to be Reviewed Under Accelerated Assessment BioMarin Pharmaceutical announced today that the European Medicines Agency (EMA) validated the Company’s Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.  The MAA review will commence in January

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No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2019-12-23

NTU Singapore scientists find easier way to harvest healing factors from adult stem cells in the lab

A research team at Nanyang Technological University, Singapore (NTU Singapore) has found an easier way to harvest healing factors – molecules that promote tissue growth and regeneration – from adult stem cells Presently, scientists ‘pre-condition’ adult stem cells to secrete healing factors by putting them in a low-oxygen chamber or by using biochemicals or genetic engineering.However in

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No Comments InREGENERATIVE MEDICINE
PIER MARIA FORNASARI
2019-12-19

Electric Phenomenon: A Disregarded Tool in Tissue Engineering and Regenerative Medicine

Tissue engineering and regenerative medicine (TERM) are paving the way to the generation of functional and mature biological tissues that closely emulate cellular, biochemical, and mechanical cues. Electrical fields in the human body modulate myriad biological processes, such as synapses, muscle contraction, hearing, and wound healing, which were disregarded in TERM until recently. The human

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No Comments InTISSUE ENGINEERING
PIER MARIA FORNASARI
2019-12-19

Production and Quality Requirements of Human Platelet Lysate: A Position Statement from the Working Party on Cellular Therapies of the International Society of Blood Transfusion

Human platelet lysate (HPL), rich in growth factors, is an efficient alternative supplement to fetal bovine serum (FBS) for ex vivo propagation of stromal cell-based medicinal products. Since 2014, HPL has been a focus of the Working Party for Cellular Therapies of the International Society of Blood Transfusion (ISBT). Currently, as several Good Manufacturing Practice

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No Comments InREGENERATIVE MEDICINE
PIER MARIA FORNASARI
2019-12-18

Gene Therapy Method Transforms Human Cells into Mass Producers of Potential Therapeutics

Researchers at Ohio State University and the University of Texas Southwestern Medical Center say they have developed a new gene therapy technique by transforming human cells into mass producers of tiny nano-sized particles full of genetic material that has the potential to reverse disease processes. Though the work was intended as a proof of concept,

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No Comments InEXOSOMES
PIER MARIA FORNASARI
2019-12-18

AbbVie Receives European Commission Approval of RINVOQ™ (upadacitinib) for the Treatment of Adults with Moderate to Severe Active Rheumatoid Arthritis

– Approval supported by data from the pivotal Phase 3 SELECT rheumatoid arthritis program evaluating nearly 4,400 patients– In five pivotal Phase 3 studies, RINVOQ™(upadacitinib) met all primary and ranked secondary endpoints across a variety of adult patient populations with moderate to severe active rheumatoid arthritis– RINVOQ, given alone or with csDMARDs, demonstrated improved rates

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2 comments InAUTOIMMUNE DISEASES
PIER MARIA FORNASARI
2019-12-15

University of Georgia researchers use exosome IV to treat traumatic brain injury

A team of researchers from the University of Georgia’s Regenerative Bioscience Center has found that neural exosomes—“cargo” molecules within the nervous system that carry messages to the brain—can minimize or even avert progression of traumatic brain injury when used as part of a new cell-to-cell messaging technology. The finding could result in the first delivery platform and

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No Comments InEXOSOMES
PIER MARIA FORNASARI
2019-12-13

FDA approves Sarepta’s 2nd Duchenne drug

The FDA unexpectedly reversed course Thursday, approving the second treatment developed by Sarepta Therapeutics for the rare disease Duchenne muscular dystrophy just a few months after rejecting the drug.  Sarepta announced Thursday evening that the agency had approved its second drug, called Vyondys 53. After the drug was originally rejected by the FDA in August due to concerns over the

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No Comments InGENE THERAPY RARE DISEASES