GENE THERAPY RARE DISEASES – Page 3 – REGENHEALTHSOLUTIONS (RHS)

PIER MARIA FORNASARI
2022-11-09

US FDA grants Rare Pediatric Disease Designation to gene therapy OTOF-GT for the treatment of otoferlin gene-mediated hearing loss

OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency Sensorion, a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, announces that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to the Company’s lead therapy gene

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PIER MARIA FORNASARI
2022-10-17

First AAV gene therapy approved in Europe for direct infusion into the brain as a treatment for AADC Deficiency.

Gene Therapy Sees Children With Rare Genetic Condition Walk And Talk For First Time Aromatic l-amino acid decarboxylase (AADC) deficiency is a genetic disorder of the nervous system. Only about 120 cases have ever been reported, and around half of these have been in people of Asian ancestry. The condition is caused by mutations in the DDC gene, which

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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

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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

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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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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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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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PIER MARIA FORNASARI
2022-07-27

How to turn muscle into a protein factory for advanced gene therapy

Interdisciplinary team of researchers from UMass Amherst and UMass Chan Medical School maps the cellular production of protein and discovers how to boost its output In a major new development in the quest to develop better gene therapies with which to treat a host of diseases, researchers at the University of Massachusetts Amherst and UMass

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PIER MARIA FORNASARI
2022-05-12

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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PIER MARIA FORNASARI
2022-04-07

Gene-therapy gel shows promise for blistering skin disease in clinical trial

Stanford researchers have been working on gene therapies for epidermolysis bullosa, or “butterfly disease,” for over a decade. A new gel helped wounds heal and stay healed in a clinical trial. A gene therapy gel applied to the wounds of nine people — three of whom were children — with the blistering skin disease epidermolysis

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Category GENE THERAPY RARE DISEASES

US FDA grants Rare Pediatric Disease Designation to gene therapy OTOF-GT for the treatment of otoferlin gene-mediated hearing loss

First AAV gene therapy approved in Europe for direct infusion into the brain as a treatment for AADC Deficiency.

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

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

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

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

Gene Therapy Approach Shows Promise in Treating ALS

How to turn muscle into a protein factory for advanced gene therapy

Novel Supramolecular CRISPR–Cas9 Carrier Enables More Efficient Genome Editing

Gene-therapy gel shows promise for blistering skin disease in clinical trial