Category REGENERATIVE MEDICINE

Results on 5 gene therapy trials expected in the first 2023 semester

Can gene therapy trials for vision loss, hemophilia, and melanoma break through in 2023? fter another turbulent year in gene therapy development, all eyes are on five major trial readouts set for H1 2023. The clinical studies target rare blood disorders, inherited retinal diseases, and metastatic melanoma—each of which poses distinct trial design challenges. First, two small

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Coadministration of CD19- and CD22-Directed Chimeric Antigen Receptor T-Cell Therapy in Childhood B-Cell Acute Lymphoblastic Leukemia: A Single-Arm, Multicenter, Phase II Trial with a 99% response rate

An innovative immunotherapy combination has shown a stunning 99% response rate in children with relapsed leukemia. The phase 2 trial, run jointly between researchers at St. Jude Children’s Research Hospital in Memphis and Shanghai Children’s Hospital in China tested the therapy in 225 children who had relapsed after conventional treatment. The work published in the Journal

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Two synthetic biology experiments offer hope for future cancer treatments with CAR-T cells

New immunotherapies can allow healing cells to be activated or deactivated according to the stage of the disease, while more accurately targeting tumors While CAR T therapy represents one of the most impressive innovations in cancer care, the treatment can cause autoimmune-like side effects. Many have contended with the dilemma of maximizing the therapy’s benefits

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Pfizer Announces Positive Top-Line Results from Phase 3 Study of Hemophilia B Gene Therapy Candidate

 Pfizer announced positive top-line results from the Phase 3 BENEGENE-2 study (NCT03861273) evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B. The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion

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Wafer-thin device has potential to transform the field of islet cell transplantation

Implantable platform provides prolonged treatment of Type 1 diabetes A quarter-sized device created at Houston Methodist could drastically alter the course of treatment for Type 1 diabetes, a chronic condition that impacts millions of Americans and does not have a cure. In a study published in the Dec. 26 issue of Nature Communications, a research team led

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Liver cancer study encourages caution with certain gene therapies

A newly discovered link between protein misfolding and liver cancer could help improve gene therapy for hemophilia Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world’s deadliest diseases. The findings, published

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New treatments for blood cancers, new hope for patients: a new STAT e-book on CAR-T cells

Blood cancers like multiple myeloma have long been considered incurable, but in the last decade, the development of precision immunotherapy treatments has offered patients months or even years of extended life. One such treatment, known as CAR-T cell therapy, turns immune system T cells into killers of cancer cells. They have been so transformative that

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