News of the week: Rare Pediatric Disease designations granted for gene therapies, Tentative FDA approval granted to generic drop for eye redness, acquisition news, and more. Read all about it in EyeWorld Weekly https://bit.ly/4cFgplU
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First autologous CD34+ hematopoietic stem/progenitor cells therapy, genetically modified by Lenti-transduced ASMA gene, approved today by FDA to treat Metachromatic Leukodystrophy (MLD). The trial recruitment of MLD is in EU, https://lnkd.in/eZ3PpuHZ The trial sponsor published the Ph1/2 clinical trial result in Lancet in 2022: https://lnkd.in/eRejuC_J This Phase I/II clinical trial consists of the application of lentiviral vector-based gene therapy to patients affected by Metachromatic Leukodystrophy (MLD), a rare inherited Lysosomal Storage Disorder (LSD) resulting from mutations in the gene encoding the Arylsulfatase A (ARSA) enzyme. The medicinal product consists of autologous CD34+ hematopoietic stem/progenitor cells in which a functional ARSA cDNA is introduced by means of 3rd generation VSV-G pseudotyped lentiviral vectors. Treatment with arsa-cel resulted in sustained, clinically relevant benefits in children with early-onset MLD by preserving cognitive function and motor development in most patients, and slowing demyelination and brain atrophy. Congrat toUK based Orchard Therapeutics
Today, we approved the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). https://lnkd.in/epcpJtrT MLD is a debilitating, rare genetic disease affecting the brain and nervous system caused by a deficiency of an enzyme called arylsulfatase A, leading to a buildup of sulfatides or fatty substances in the cells.
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RAPTAR AUTOMATED SAFETY CASE PROCESSING - ADVANCED THERAPIES Is your Pharmacovigilance as advanced as your therapies? In order to help with Pharmacovigilance safety case processing PharSafer has invented RAPTAR - An automated safety case processing system with configurable follow up to monitor safety in all types of products - drugs, devices, vaccines, biologics, advanced therapies and cosmetics. As new and necessary treatments are devised and approved RAPTAR can ensure Regulatory compliance for safety reporting allowing for the accurate collection of safety data globally in a cost effective way. PharSafer remains at the cutting edge of global drug safety to ensure Companies can easily comply with multi-national safety requirements in a compliant manner. 😀 Follow us on: #RAPTAR ⬇ https://lnkd.in/exZFGiw #PharSafer ⬇ https://lnkd.in/eimySG2 #drugdevelopment #drugsafety #pharmacovigilance #medicaldevices #cosmeticsindustry #cosmetics #compliance #regulatoryaffairs #regulatorycompliance #regulatoryintelligence #PBRER #periodic reports #vaccinedevelopment #materiovigilance #inspections #gcp #auditing #audits #auditservices #IND #PSMF #PRAC #validation #artificialintelligence #GAMP #21CFRPart11 #clinicaltrials #clinicaloperations #ICH #trainingdevelopment #training #QRcode #ITautomation #FDA #EMA #PRAC #Brexit #DSUR #MHRA #ICSR #SUSAR #caseprocessing #automation #signaldetection #medicalinformation #medicalaffairs #productinformation #productcomplaints #training #SDEA #medicaldevice #device #regulation #pregnancy #advancedtherapies
Today, we approved the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). https://lnkd.in/epcpJtrT MLD is a debilitating, rare genetic disease affecting the brain and nervous system caused by a deficiency of an enzyme called arylsulfatase A, leading to a buildup of sulfatides or fatty substances in the cells.
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Special Issue on Genes and Gene Therapies in Chronic Renal Disease aims to highlight the transformative role of molecular and medical genetics in enhancing the understanding, diagnosis, and treatment of patients with chronic renal disease. Read more at: https://lnkd.in/dYu8x-7Y
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Gene therapy emerges as a treatment option for neurodengerative disease. This article explores how the promotion of interleurkin-10 (IL-10) expression could alleviate symptoms of Parkinson's disease - read more below! https://lnkd.in/eD_ffZQc
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Advanced therapy medicinal products, including Cell & Gene Therapies (CGTs), are transforming the healthcare landscape, offering hope to those battling rare diseases, cancers, and autoimmune disorders. Read the latest bioMérieux's blog article to learn how collaborative research and quality control supports CGTs development & manfacturing. #WeArebioMérieux
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Read one of our latest articles! Gene therapy an emerging treatment option for Parkinson’s disease
Gene therapy emerges as a treatment option for neurodengerative disease. This article explores how the promotion of interleurkin-10 (IL-10) expression could alleviate symptoms of Parkinson's disease - read more below! https://lnkd.in/eD_ffZQc
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Advanced therapy medicinal products, including Cell & Gene Therapies (CGTs), are transforming the healthcare landscape, offering hope to those battling rare diseases, cancers, and autoimmune disorders. Read the latest bioMérieux's blog article to learn how collaborative research and quality control supports CGTs development & manfacturing. #WeArebioMérieux
Collaborative Research and Quality Control Supports Cell & Gene Therapy Development & Manufacturing
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#podcast Ha Tran, Medical Head, Cell and Gene Therapy at Astellas Pharma is developing gene therapy approaches to fight neuromuscular diseases, central nervous system disorders, and ophthalmology conditions. There is strong evidence that this approach can be a way to treat and possibly cure rare diseases. Ha explains the challenges of running gene therapy trials for rare diseases and the unique patient journey required to run a successful trial. #AstellasPharma #GeneTherapy #RareDiseases #Allogeneic #ClinicalTrials #CNS #NeuromuscularDiseases astellas.com/us/ https://lnkd.in/dmCtkGAn
Empowered Patient Podcast: Path to Developing Gene Therapies for Treating Rare Diseases with Ha Tran Astellas Pharma
empoweredpatientradio.com
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Our Chief Medical Officer Kenji Fujita, MD, had the pleasure of speaking with Tyler Menichiello about the rise of gene therapies being developed for #InheritedRetinalDiseases and Atsena’s clinical programs in #LCA1 and #XLRS. Check out the article in Cell & Gene. ⬇ #GeneTherapy #ophthalmology
The Rising Tide Of Ophthalmologic Gene Therapies https://lnkd.in/es6Wswes In recent years, there’s been an increase in gene therapy clinical trials for inherited retinal diseases (IRDs) — a group of genetic conditions that can lead to vision loss and blindness.
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Cell and gene therapies are revolutionizing how clinicians treat previously intractable diseases such as cancers, blindness, and metabolic disorders. In this tech note, examine integration site analysis techniques that are a critical component of patient safety testing of these therapies: https://hubs.ly/Q02LmRR20 #ClinicalTrials #CellTherapy #GeneTherapy #PatientSafety
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