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Thoughts on this? >> BIX Korea 2024: Promise of mRNA technology for cell and gene therapy - BioWorld Online >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #competitivemarketing #biotech #healthcare #pharmaceutical

Thoughts on this? >> Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort | Gene ... - Nature.com >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #biotech #pharmaceutical #pharma #competitivemarketing

Thoughts on this? >> Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort | Gene ... - Nature.com >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #biotech #healthcare #pharmaceutical #pharma #competitivemarketing

Thoughts on this? >> ASGCT: 'From darkness to light' in ocular gene therapy - BioWorld Online >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #competitivemarketing #healthcare #biotech #pharma #pharmaceutical

Today was a day full of updates on the advancement of gene therapy 🧬✂️ 📌 Pfizer reported that their Phase III trial in Duchenne gene therapy was unsuccessful. This adds more pressure on the FDA to meet Sarepta’s requests for revising the Elevidys label. 📌 PTC Therapeutics shared 12-month data from the PIVOT-HD trial, testing the oral HTT-lowering drug PTC-518. Although the trial was designed to assess safety, the results are encouraging, showing promising signs for biomarkers and some clinical metrics. The journey of gene therapy continues to be one of highs and lows, underscoring the critical need for ongoing research and development 👩🏻🔬🧑🏼🔬👨🏽🔬. #GeneTherapy #Biotech #Pharmaceuticals #ClinicalTrials #Duchenne #HuntingtonsDisease

Thoughts on this? >> Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson Disease - CGTLive™ >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #pharma #competitivemarketing #biotech #pharmaceutical

Why this matters? 1. Orchard Therapeutics Launch: Orchard Therapeutics has announced the U.S. commercial launch of Lenmeldy (atidarsagene autotemcel), a gene therapy for metachromatic leukodystrophy (MLD), with a wholesale acquisition cost of $4.25 million. #GeneTherapy #OrchardTherapeutics #Lenmeldy 2. Pricing Justification: The company justifies the high cost by highlighting the potential long-term impact of the treatment on healthcare utilization and the improved quality of life for patients and caregivers. #HealthcareCosts #PatientCare #QualityOfLife 3. ICER Assessment: The pricing was determined after a comprehensive health technology assessment by the Institute for Clinical and Economic Review (ICER), which set Lenmeldy’s health benefit price benchmark at up to $3.94 million. #ICER #CostEffectiveness #HealthEconomics 4. MLD Impact: Metachromatic leukodystrophy is a rare genetic metabolic disorder affecting one in every 100,000 live births, leading to severe cognitive and physical impairments. #RareDisease #MetabolicDisorder #MLD 5. Gene Therapy Benefits: Lenmeldy has shown significant improvement in survival and developmental milestones in treated children, positioning it as a paradigm-shifting medicine for early-onset MLD. #ClinicalTrials #TreatmentAdvancements #MedicalInnovation 6. Comparison with Other Therapies: Lenmeldy’s price continues the trend of high costs for gene therapies, with Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy priced at $2.2 million and bluebird bio’s Lyfgenia at $3.1 million. #GeneTherapyCosts #PharmaceuticalPricing #HealthcareIndustry

Thoughts on this? >> 12 Year Old Begins Newly Approved Sickle Cell Gene Therapy - Black Enterprise >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #pharmaceutical #competitivemarketing #biotech #healthcare

Thoughts on this? >> Preclinical safety and efficacy of intein-based dual-AAV gene therapy for Stargardt disease - BioWorld Online >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #competitivemarketing #pharma #healthcare #pharmaceutical #biotech

How are cell and gene therapy products classified and regulated in the EU and US? Click here to read more: https://lnkd.in/gb9ZKsg #celltherapy #genetherapy #pharmaceutical #pharma #drugdevelopment #clinicaltrials #clinicalresearch #drugsafety #pharmacovigilance #classification #drugclassification