Category GENE THERAPY RARE DISEASES

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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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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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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Novel Supramolecular CRISPR–Cas9 Carrier Enables More Efficient Genome Editing

Fifth generation polyrotaxane (PRX) carriers can effectively deliver CRISPR-Cas9 ribonucleoproteins (RNPs) The PRX carrier can cross the cell membrane, avoid endosomal action, and release Cas9 RNP for entry into the nucleusImage courtesy: Kumamoto University, The carriers, aminated polyrotaxanes, can flexibly and reversibly bind with Cas9 ribonucleoprotein and protect it from intracellular endosomal degradation CRISPR-Cas9 is

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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1–2 dose-escalation study. METHODS Was conducted an open-label, single-group, multicenter, phase 3

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Huge step forward towards gene therapy and diagnosis for genetic eye disease

New opportunities towards gene therapy and diagnosis for the blinding eye disease, retinal dystrophy, may now become available following work done by the Eye Genetics Research Unit at Children’s Medical Research Institute. This work was published in the Journal of Personalized Medicine today. The team looked at the RPGR gene which is involved in maintaining

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Gene Therapy for Thalassemia Ends Need for Transfusions in Young Children

Phase 3 clinical trial included children younger than 12 years Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients

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First gene therapy for Tay-Sachs disease successfully given to two children

Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development. Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a fatlike substance that normally exists in very small, harmless amounts in the brain. Without HexA, however, this fatlike substance can accumulate

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