Category REGENERATIVE MEDICINE

PIER MARIA FORNASARI
2022-10-14

CART cells for relapsed or refractory multiple myeloma (CARTITUDE-1): a phase 1b–2, open-label study on health-related quality of life published in The Lancet Haematology

CARTITUDE-1 is a phase 1b–2 study evaluating ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor T cell therapy with two B-cell maturation antigen–targeting single-domain antibodies, in patients with relapsed or refractory multiple myeloma. Primary efficacy outcomes have previously been reported. In this paper are reported health-related quality of life (HRQOL) secondary outcomes evaluated using patient-reported outcomes.

Read More

No Comments InCAR-T
PIER MARIA FORNASARI
2022-10-14

Results of a phase 1 UCART19 trial, a first-in-class allogeneic anti-CD19 CART-cell therapy for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia (CALM) published in The Lancet Haematology

The prognosis for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia remains poor. UCART19, an allogeneic genome-edited anti-CD19 chimeric antigen receptor (CAR) T-cell product derived from healthy donors and available for immediate clinical use, offers a potential therapeutic option for such patients. The CALM trial is a first-in-human study evaluating the safety and antileukaemic

Read More

No Comments InCAR-T
PIER MARIA FORNASARI
2022-10-10

Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness, Penn Study Finds

Patients’ low-light sensitivity improved by factors of thousands in a clinical trial Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. The patients

Read More

No Comments InGENE THERAPY RARE DISEASES
PIER MARIA FORNASARI
2022-09-29

 Short documentary video from the New England Journal of Medicine on Sickle Cell Disease and Gene Therapy from Patient and Physician Perspectives

In this short documentary video from the New England Journal of Medicine, patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy. Patients share their own stories of interactions with the health care system and explore

Read More

No Comments InGENE THERAPY RARE DISEASES
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

Read More

No Comments InCAR-T
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

Read More

No Comments InGENE EDITING
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

Read More

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

Read More

No Comments InADVANCED THERAPY MEDICINAL PRODUCT
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,

Read More

No Comments InADVANCED THERAPY MEDICINAL PRODUCT GENE THERAPY RARE DISEASES