Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2022-09-27

Calibr, a Division of Scripps Research, reports promising results from first-in-human clinical trial of switchable CAR-T (CLBR001 + SWI019), a next-generation universal CAR-T platform designed to enhance the versatility and safety of cell therapies

In preliminary results from a Phase I study of CLBR001 + SWI019 for patients with B cell malignancies: 7 of 9 patients responded and 6 of 9 had a complete response (78% ORR, 67% CR) CLBR001 cells engrafted at higher levels than approved CAR-T cell products without causing an increase in the incidence of CRS

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PIER MARIA FORNASARI
2022-09-25

New University of California Irvine-led report Illustrates potential of precision genome editing in treating inherited retinal diseases

Improvements in technology and delivery techniques could revolutionize treatment of genetic disorders of vision In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). The study, titled, “Precision genome editing in the eye,” was published this week

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PIER MARIA FORNASARI
2022-08-18

FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions

The U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.  “Today’s approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions,” said Peter

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One comment InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2022-08-17

Stem cell-based therapy for human diseases: a complete review

Recent advancements in stem cell technology open a new door for patients suffering from diseases and disorders that have yet to be treated. Stem cell-based therapy, including human pluripotent stem cells (hPSCs) and multipotent mesenchymal stem cells (MSCs), has recently emerged as a key player in regenerative medicine. hPSCs are defined as self-renewable cell types

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PIER MARIA FORNASARI
2022-08-12

Two patients treated with Novartis Zolgensma gene therapy died due to acute liver injury.

Two children who received a Novartis gene therapy for their neuromuscular disease died following treatment, spotlighting its risks and renewing questions about the safety of genetic medicines like it. The patients developed acute liver failure between five and six weeks after infusion with the gene therapy, called Zolgensma and approved to treat spinal muscular atrophy,

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No Comments InADVANCED THERAPY MEDICINAL PRODUCT GENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2022-08-09

Higher doses of CAR T cell therapy associate with improved outcomes in young patients with B-ALL : a report from the pediatric real-world CAR consortium

Young people who received doses of tisagenlecleucel, a chimeric antigen receptor T cell (CAR-T) therapy, at the higher end of the FDA-approved dosing range had significantly better survival rates at one year compared with those who received lower doses within this range, according to research published today in Blood Advances.   Since its approval as the first

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PIER MARIA FORNASARI
2022-08-06

St. Jude Researchers improved CAR T-cell therapy for T-cell acute lymphoblastic leukemia

Scientists at St. Jude Children’s Research Hospital developed a simple method to select for more effective cancer-destroying CAR T cells for patients with relapsed T-ALL. Scientists at St. Jude Children’s Research Hospital are improving chimeric antigen receptor (CAR) T-cell therapy. Their new simplified approach selected for an advantageous T-cell type and showed promise in the lab against relapsed T-cell

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PIER MARIA FORNASARI
2022-08-03

Gene Therapy Approach Shows Promise in Treating ALS

In rodent models, introduced neuroprotective protein slowed disease progression and increased life span Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the progressive loss of motor neurons in the brain and spinal cord responsible for voluntary movements and muscle control. In a new study, published July 11, 2022 in the journal Theranostics, researchers at

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No Comments InGENE THERAPY RARE DISEASES