News and Trends 22 May 2023 FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment The U.S. Food and Drug Administration has approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The approval of Vyjuvek was granted to […] May 22, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
In Depth 19 May 2023 Can cardiovascular diseases be cured? The future of heart treatment Cardiovascular diseases, characterized as a group of disorders of the heart and blood vessels, are the leading cause of death worldwide. In fact, in the U.S. alone, it is thought that one person dies every 33 seconds from cardiovascular disease. Although clearly a huge health problem throughout the world, developing cures for cardiovascular diseases is […] May 19, 2023 - 11 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
News and Trends 18 May 2023 8 rare diseases selected for gene therapy trial portfolio The Foundation for the National Institutes of Health (FNIH) says the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. This portfolio pioneers a novel approach to gene therapy development for rare diseases by demonstrating that manufacturing and testing standards can provide a streamlined approval […] May 18, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 18 May 2023 Sania Therapeutics launches to advance neural circuit dysfunction medicines Sania Therapeutics, which develops genetic medicines for neural circuit dysfunction, has launched with the unveiling of its platforms at the American Society of Gene & Cell Therapy (ASGCT) conference. Sania Therapeutics’ approach combines the evolution of adeno-associated viruses (AAV) in human neural circuits, the fundamental components of the nervous system, and tuneable conditionally-activated ion channels. […] May 18, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 12 May 2023 Cell and gene therapy company NewBiologix exits stealth with $50M NewBiologix SA has emerged from stealth as a technology innovation company developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies. The company’s DNA-based platform is being developed to address key manufacturing challenges associated with the production of recombinant adeno-associated virus (rAAV) vectors used to […] May 12, 2023 - 3 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 10 May 2023 Delivering on the need for vector innovation in genetic medicine By Tuyen Ong, CEO Ring Therapeutics, CEO-Partner Flagship Pioneering Gene therapy is a life-changing class of medicine that can treat patients with once intractable diseases. While revolutionary, significant limitations remain for the vectors that carry them. Viruses are masters at delivering genetic material to the nucleus of the organisms they infect, and because of this, […] May 10, 2023 - 2 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 10 May 2023 Cell rejuvenation for age-related diseases By Sharon Rosenzweig-Lipson, CSO, Life Biosciences Cellular rejuvenation is quickly becoming a hot topic in the development of novel therapeutic modalities for a range of diseases. The leading approaches to cellular rejuvenation currently in development utilize epigenetic reprogramming to achieve this goal. Epigenetics is the study of how positive and negative lifestyle factors (exercise, smoking, […] May 10, 2023 - 3 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
Opinion 9 May 2023 Where is innovation needed in cell therapy? By Dr Ryan Roberts, cell processing lead at Cellular Origins Despite the rapidly emerging innovations in the field of cell and gene therapy, bottlenecks in cell therapy manufacturing processes are preventing these transformative medicines from reaching patients. There are several broad challenges to the scalability and affordability of cell therapies, including a reliance on manual […] May 9, 2023 - 5 minutesmins - By External Contributor Share WhatsApp Twitter Linkedin Email
News and Trends 9 May 2023 Gene & cell therapy manufacturer Ascend launches with more than $130M Ascend Gene & Cell Therapies (Ascend), a company innovating manufacturing processes to improve quality, potency, safety and cost enabling better reach for gene therapy technologies, has launched as a facilitator for gene therapy companies to help translate product ideas into clinical programs. Ascend Gene & Cell Therapies offers process development, clinical manufacturing, and analytical and […] May 9, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 8 May 2023 How thalassemia gene therapy approval is making a difference A newly-approved gene therapy is transforming the lives of patients with beta thalassemia – an inherited blood disorder – by helping to stop or significantly reduce their reliance on blood transfusions, says an expert from a leading global health system, Cleveland Clinic, on International Thalassemia Day on May 8. A gene therapy known as betibeglogene […] May 8, 2023 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 25 Apr 2023 Gene therapy and chemotherapy could be new osteosarcoma treatment option New research has identified a potential therapeutic target and developed a unique delivery system to treat osteosarcoma, a bone cancer that primarily affects children and adolescents. The standard-of-care treatment plan for osteosarcoma today is no different to when first introduced almost 50 years ago. However, nearly one third of patients’ relapse and need new interventions. […] April 25, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 24 Apr 2023 Life Biosciences gene therapy data shows visual function restored in primates Life Biosciences (Life Bio) has announced preclinical data in nonhuman primates (NHP) for its novel gene therapy candidate, which uses a partial epigenetic reprogramming approach to restore visual function. The approach has been shown to reverse aging, improve vision, and extend lifespan in mice, but whether epigenetic reprogramming would work in primates was not known. […] April 24, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email