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: 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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Posted on: 8 February 2025, source: HCP live
More than 90% of patients with hemophilia B eliminated factor IX prophylaxis 4 years after treatment with etranacogene dezaparvovec (HEMGENIX) gene therapy. Hemophilia B Gene Therapy Achieves Sustained Benefit Over 4 Years. A one-time infusion of etranacogene dezaparvovec-drlb (HEMGENIX) gene therapy continued to demonstrate long-term sustained efficacy and safety in adults with hemophilia B over 4 years in the pivotal Phase 3 HOPE-B study. Announced by CSL on February 7, 2025, these data, presented at the 18th Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD), showed the gene therapy delivered sustained factor IX activity levels, offered greater bleed protection than prophylactic treatment, and maintained favorable safety over time.

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Posted on: 1 February 2025, source: evrimagaci.org
On February 1, 2025, the National Institute for Health and Care Excellence (NICE) announced the approval of exagamglogene autotemcel (exa-cel), marking what many are calling a significant breakthrough for the treatment of sickle cell disease. This groundbreaking gene therapy utilizes the CRISPR gene-editing technology, which has transformed medical approaches to genetic disorders, enabling some patients suffering from this debilitating condition to access potentially life-changing treatment.

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Posted on: 4 January 2025, source: pmlive.com
Novartis has shared positive top-line results from a late-stage study of its investigational gene therapy in patients aged two to less than 18 years with the rare neuromuscular disease spinal muscular atrophy (SMA). The phase 3 STEER study has been evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with SMA type 2 who were able to sit but had never walked independently. Results were compared against a sham control, which is designed to mimic the administration of an investigational drug without delivering any active treatment.

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Posted on: 17 December 2024, source: Indian Express
In a significant medical breakthrough, India’s first human gene therapy with a lentiviral vector for severe haemophilia-A has been found to be successful with zero bleeding rate in patients. Haemophilia is a serious bleeding disorder caused by blood deficiency of clotting factors (factor VIII and factor IX proteins). It can lead to spontaneous bleeding episodes, joint damage, and a reduced quality of life for patients and their families. The new gene therapy has shown promising results during the first phase trial on five patients, aged between 22 and 41. The annualised bleeding rate has been observed to be zero in all five patients over a cumulative follow-up of 81 months. Supported by the Department of Biotechnology, this first-in-human gene therapy for haemophilia-A patients, has been developed by Centre for Stem Cell Research (CSCR) at CMC, Vellore.

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