Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

Posted on: 30 June 2025, source: Medical Xpress
Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle cell disease experience increased brain ischemia, where oxygen is not delivered properly to brain tissues, potentially leading to strokes. A part of the risk for these events comes from increased blood flow speed in the brain. Findings from three patients in a gene therapy clinical trial showed that gene therapy treatment significantly improved blood flow in the brain. These results demonstrate that people with these risk factors may benefit from gene therapy and should be considered for future clinical trials of gene therapy. The findings were published in the American Journal of Hematology.

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Posted on: 18 June 2025, source: Forbes
On June 16, Sarepta Therapeutics announced a second patient death following treatment with its gene therapy, Elevidys, for Duchenne muscular dystrophy. This news has sent shockwaves through the rare disease and biotech communities. It raises questions about the safety of adeno-associated virus-based gene therapies. The latest fatality, a 15-year-old, died from acute liver failure, a recognized but rare complication of adeno-associated virus-based gene transfer. This death follows a similar case reported in March.

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Posted on: 29 May 2025, source: Nature
This study presents a novel cancer vaccine strategy using genetically engineered bacteriophages. These phages display tumor-associated peptides on their surface and were tested in a mouse melanoma model, both alone and in combination with gene electrotransfer (GET) of interleukin-12 (IL-12) plasmids. Both treatments significantly delayed tumor growth, and their combination had a synergistic effect, leading to complete tumor regression in 30% of cases. Tissue analysis showed increased immune activation, including higher levels of necrosis and infiltration by macrophages, CD4+, and CD8+ T cells. This is the first time a combination of three different engineered M13 phages with intratumoral IL-12 GET has been tested, demonstrating strong therapeutic potential. The results highlight the promise of nanotechnological approaches like phage-based vaccines in cancer treatment.

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Posted on: 2 May 2025, source: PM Live
The US Food and Drug Administration (FDA) has approved Abeona Therapeutics’ Zevaskyn (prademagene zamikeracel) for use in recessive dystrophic epidermolysis bullosa (RDEB). The decision makes Zevaskyn the first autologous cell-based gene therapy approved in the US to treat wounds in adult and paediatric patients with the rare skin disorder. RDEB is an incurable connective tissue disease that causes the skin to become very fragile. It is characterised by extensive blistering and severe wounds that often cover more than 30% of a patient’s body surface.

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Posted on: 11 April 2025, source: China Daily
Japanese pharmaceutical company Takeda and domestic biotech company Belief BioMed jointly announced on Thursday that the gene therapy drug Dalnacogene Ponparvovec Injection has received approval from China's National Medical Products Administration for the treatment of moderate to severe hemophilia B in adult patients, marking the first approved gene therapy drug for the disease in the country. This innovative injection was developed and produced by Belief BioMed, while Takeda China is responsible for its commercialization on the Chinese mainland as well as Hong Kong and Macao. The two parties will leverage their respective strengths to accelerate the provision of this gene therapy to patients and jointly explore new frontiers in hemophilia B treatment.

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Posted on: 2 April 2025, source: news-medical.net
Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the genetic material to the tissue and cell type of interest. To help improve gene therapy, a multidisciplinary team led by researchers at Baylor College of Medicine, the Jackson Laboratory and the University of Massachusetts Medical School has generated a comprehensive atlas that researchers can use to select the most effective viral vehicle for their target organ. The study appeared in Molecular Therapy.

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Posted on: 3 March 2025, source: hcplive.com
Pfizer has announced that it is discontinuing development and commercialization of fidanacogene elaparvovec an adeno-associated virus (AAV) vector-based gene therapy for the treatment of hemophilia B. The therapy was approved by the FDA under the name Beqvez less than a year ago, in April 2024. Reuters reported the news on February 21, stating that Pfizer cited limited interest in gene therapies for hemophilia as a reason for discontinuation. No patients had been dosed yet in the commercial setting with the therapy, which was priced at a whopping $3.5 million.

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