Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies

Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their

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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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DiscGenics Receives FDA Fast Track Designation for Cell Therapy for Disc Degeneration

DiscGenics, Inc., a clinical stage biotechnology company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its investigational cell therapy, IDCT, currently being evaluated in regulator-allowed clinical trials

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Adoptive cell therapy using engineered natural killer cells

The generation of autologous T cells expressing a chimeric antigen receptor (CAR) have revolutionized the field of adoptive cellular therapy. CAR-T cells directed against CD19 have resulted in remarkable clinical responses in patients affected by B-lymphoid malignancies. However, the production of allogeneic CAR-T cells products remains expensive and clinically challenging. Moreover, the toxicity profile of

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Moving Towards Automated Manufacturing for Cell Therapies

As more cell therapy products reach the “finish line” of U.S. Food and Drug Administration (FDA) approval and new regenerative medicine products begin the clinical development process, the need for expanded manufacturing capacity that can support long-term commercial business models is increasing. Recently, David Smith, Alex Klarer, Thomas Heathman, Courtney LeBlon, Yasuhiko Tada and Brian

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Asia Bioprocessing Capacity Trends: Overall Growth, But Cell Therapy Shortages Loom

Overall, the growth in Asia’s bioprocessing capacity, particularly in China, has been impressive, albeit from a low baseline. Capacity growth over the past five years has been over 20 percent annually.1 New facility construction and expansions reflect the demand for biologics for domestic consumption, while other facilities are beginning to develop manufacturing strategies for GMP production

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University of Minnesota achieves transplant in monkeys without long-term immune-fighting drugs

The technique could greatly increase the number and safety of organ transplants.  University of Minnesota researchers are reporting a breakthrough in what has long been viewed as a holy grail in the field of organ transplantation — success without the permanent need for immunosuppressive drugs. Their technique could greatly increase the number and safety of

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Mesenchymal Stem Cells for Perianal Crohn’s Disease

Perianal fistulizing Crohn’s disease (PFCD) is associated with significant morbidity and might negatively impact the quality of life of CD patients. In the last two decades, the management of PFCD has evolved in terms of the multidisciplinary approach involving gastroenterologists and colorectal surgeons. However, the highest fistula healing rates, even combining surgical and anti-TNF agents,

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