Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2022-02-15

Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency. “These results confirm the stability and good

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PIER MARIA FORNASARI
2022-02-06

Gene therapy in transfusion-dependent beta-thalassemia

A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine. The therapy represents a potentially curative treatment option for patients who must otherwise rely on life-long red blood cell transfusions. This approach provides

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PIER MARIA FORNASARI
2022-02-06

Study of Penn Patients with Decade-Long Leukemia Remissions after CAR T Cell Therapy Reveals New Details About Persistence of Personalized “Living Drug” Cells

Two patients represent longest-known CAR T cell response to date, providing insight into treatment effects and outcomes In the summer of 2010, Bill Ludwig and Doug Olson were battling an insidious blood cancer called chronic lymphocytic leukemia (CLL). They’d both received numerous treatments, and as remaining options became scarce, they volunteered to become the first

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PIER MARIA FORNASARI
2022-01-25

Landing therapeutic genes safely in the human genome

Researchers at Harvard’s Wyss Institute, Harvard Medical School, and the ETH Zurich predict and validate genomic safe harbors for therapeutic genes, enabling safer, more efficient, and predictable gene and cell therapies. Many future gene and cell therapies to treat diseases like cancer, rare genetic and other conditions could be enhanced in their efficacy, persistence, and

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PIER MARIA FORNASARI
2022-01-25

New gene therapy treatment resulted in sustained, clinically relevant benefits in children with Metachromatic Leucodystrophy: long term results published.

An investigational gene therapy showed benefit in pediatric patients with early-onset metachromatic leukodystrophy (MLD), a small study showed. Treatment with atidarsagene autotemcel (arsa-cel) preserved motor development and cognitive function in MLD patients enrolled in a prospective phase I/II trial or treated through expanded access programs, reported Alessandro Aiuti, MD, of the San Raffaele Telethon Institute for Gene

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PIER MARIA FORNASARI
2022-01-19

Immunomodulatory effects of mesenchymal stem cells in peripheral nerve injury: a review

Peripheral nerve injury (PNI) refers to varying degrees of trauma to peripheral nerve stems or branches. PNIaccounts for 1.5–4.0% of global trauma cases annually and is one of the most challenging health issues at present. Nerve regeneration is a complicated cellular process involving infammation, neurotrophic factors, neurotransmitters, adhesion, the formation of axons and growth cones,

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PIER MARIA FORNASARI
2022-01-19

Epilepsy Research Boosts Case for New Gene Therapy

Research from the School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. The gene therapy, developed by Stoke Therapeutics, is now in clinical trials. Because most Dravet syndrome cases are caused by a mutation in the SCN1A gene, resulting in

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PIER MARIA FORNASARI
2022-01-11

Engineered Mesenchymal Stem Cells as a Biotherapy Platform for Targeted Photodynamic Immunotherapy of Breast Cancer

Interleukin-12 (IL12) is a pleiotropic cytokine with promising prospects for cancer immunotherapy. Though IL12 gene-based therapy could overcome the fatal hurdle of severe systemic toxicity, targeted delivery and tumor-located expression of IL12 gene remain the challenging issues yet to be solved. Photo-immunotherapy emerging as a novel and precise therapeutic strategy, which elaborately combines immune-activating agents

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PIER MARIA FORNASARI
2022-01-11

CAR-T-cell therapy has the potential to replace chemoimmunotherapy for second-line treatment of patients with refractory or relapsed large B-cell lymphoma (LBCL)

In the ZUMA-7 trial, at a median follow-up of 24.9 months, patients randomly assigned to receive CAR T-cell therapy with axicabtagene ciloleucel, or axi-cell (Yescarta) had a median event-free survival (EFS) of 8.3 months, compared with 2 months for patients randomly assigned to standard-of-care chemoimmunotherapy, reported Frederick L. Locke, MD, from the Moffitt Cancer Center

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