Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2022-12-29

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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PIER MARIA FORNASARI
2022-12-28

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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PIER MARIA FORNASARI
2022-12-07

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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PIER MARIA FORNASARI
2022-12-07

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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PIER MARIA FORNASARI
2022-11-24

Gene-delivering viruses reach the brain in a step toward gene therapy for neurological diseases

Researchers have engineered a family of adeno-associated viral vectors that cross the blood-brain barrier in primate models. Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed. Researchers have engineered viruses called adeno-associated viruses (AAVs) to deliver

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PIER MARIA FORNASARI
2022-11-23

CRISPR Technology applied to CAR T cells for Cancer Therapy

Researchers find that combining novel gene-editing CRISPR technology with CAR T therapy could simplify and improve CAR T therapy in one fell swoop. Traditional CAR T Therapy A remarkable feat in cancer care, today people with difficult-to-treat blood cancers can receive CAR T therapy, a personalized “drug” made from their own immune cells. Chimeric Antigen Receptor T cell (CAR T)

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