Category REGENERATIVE MEDICINE

CAR T Cell Toxicity

By late 2018, 2 chimeric antigen receptor T (CAR T) cell products have been approved by US and European regulatory authorities. Tisagenlecleucel (Kymriah, Novartis) is indicated in the treatment of patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse (ELIANA trial), or

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First gene therapy to treat rare blood disease nears European approval

The first gene therapy to treat a rare blood disorder is one step closer to approval Friday following a recommendation by European officials. Lentiglobin, the gene therapy for beta-thalassemia developed by Cambridge, Mass.-based Bluebird Bio, was recommended for approval by the Committee for Medicinal Products for Human Use (CHMP), the drug-reviewing arm of the European Medicines Agency.

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When it comes to cell, gene therapy, manufacturing is ‘Question one, two and three’

Panelists discussed the outlook for cell and gene therapy investing at the Alliance for Regenerative Medicine’s Cell & Gene Therapy Investor Day in New York Thursday. As cell and gene therapy become a focal point of investment in the public markets and private equity alike, one of the biggest barriers to them is still manufacturing.

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Exercise helps prevent cartilage damage caused by arthritis

Exercise helps to prevent the degradation of cartilage caused by osteoarthritis, according to a new study from Queen Mary University of London. The researchers show for the first time how mechanical forces experienced by cells in joints during exercise prevent cartilage degradation by suppressing the action of inflammatory molecules which cause osteoarthritis. The study, published

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Stem Cell Therapy for Heart Failure

With modern, guideline-directed therapy,1 which includes angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers (ARBs), β-receptor blockers, mineralocorticoid receptor blockers, and angiotensin receptor neprilysin inhibitors, up to 40% of patients with dilated cardiomyopathy (heart failure with reduced ejection fraction) may experience a return of left ventricular ejection fraction (LVEF) and ventricular geometry to normal.2 This encouraging result

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Study finds old brains still make neurons

https://www.nature.com/articles/s41591-019-0375-9 Reports of old brains’ decrepitude have been greatly exaggerated, scientists reported on Monday, unveiling results that contradict a much-discussed 2018 study and instead support the idea that human gray matter is capable of generating new neurons up to the ninth decade of life. The research, published in Nature Medicine, also found that old brains from

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A special edition of The American Statistician presents over 40 papers on “Statistical inference in the 21st century: a world beyond P < 0.05”

Statistical significance has become an infallible doctrine of scientific research. However, many scientists and statisticians argue that long-held beliefs about statistical significance have, in fact, harmed the scientific community. In hypothesis testing, the p-value gives the probability or likelihood that the null hypothesis is true and is frequently used as a measure of “statistical significance.”

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