Biogen’s Post

Great Relief for ALS Patients! New treatment for rare motor neurone disease recommended for approval EMA has recommended granting a marketing authorisation in the European Union for a new therapy for the treatment of adult patients with amyotrophic lateral sclerosis (ALS), a rare and often fatal disease that causes muscles to become weak and leads to paralysis. #Qalsody (#tofersen) is indicated for the treatment of adults with #ALS, who have a mutation in the superoxide dismutase 1 (SOD1) gene. #NeuroMotorDisease #RaReDisease #EMA #amyotrophiclateralsclerosis #Biogen

Role of kynurenine monooxygenase in experimental acute pancreatitis The researchers characterize a metabolic link between 3-hydroxykynurenine generated by KMO activity and priming of innate immune gene transcription during systemic inflammation. Therapeutic blockade using the highly specific KMO inhibitor GSK898 rescues the phenotype and protects against excess morbidity and death from multiple organ failure in experimental acute pancreatitis. #sciencenewshighlights #ScienceMission https://lnkd.in/gt6yFk2Y

Rare disease drug development is thriving – thanks to promising gene therapies, advances in AI and a supportive regulatory environment, among other influences. But more work needs to be done, urgently. Listen to the latest episode of our podcast series, Preparing for a Cell and Gene Future as Kim MacDonnell, BScN, MSc, RN, CGC and I address the use of surrogate endpoints and regulatory acceptance of surrogate endpoints to demonstrate clinical benefit. https://lnkd.in/gSNNm8VR #cellandgenetherapy #raredisease

Rare disease drug development is thriving – thanks to promising gene therapies, advances in AI and a supportive regulatory environment, among other influences. But more work needs to be done, urgently. Listen to the latest episode of our podcast series, Preparing for a Cell and Gene Future as Jamie Pierson and Kim MacDonnell, BScN, MSc, RN, CGC address the use and regulatory acceptance of surrogate endpoints to demonstrate clinical benefit. https://lnkd.in/gDMdMTvC

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

On July 19, 2023, we had the pleasure of meeting and discussing with Professor Juan Bueren, President of the European Society of Gene and Cell Therapy (ESGCT). We are excited to explore the potential of establishing a productive #collaboration towards delivering a sustainable solution on #access to #genetherapy for rare disease #patients. Our mission is to continue advocating for the rights of all #thalassaemia, #sicklecell disease and other #rareanaemia patients, who need cell and gene therapies or opt in getting this type of #treatments as a #cure. #ThalassaemiaInternationalFederation #TIF #ESGCT #GeneTherapy #CellAndGene #AdvancedTherapies #HopeForCure

Souad Messahel, PhD, Elpida Therapeutics on Clinical Trials in Rare Diseases - Strategies for Resource Management, exploring the development costs for a biotech for a gene therapy. #ASGCT24, within the “Why Do Rare Disease Clinical Trials Often Miss The Mark” workshop

Join the #ICLE2025 program by submitting your abstract on “Non-viral CAR/TCR gene targeting”! Showcase your research in promoting integrations into lymphocyte genomes by means of nucleases or transposons. Submit your abstract today: https://lnkd.in/dCxhi_iE #ICLE2025 #lymphocytes #lymphocytesengineering #CARTcells

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

During the American Epilepsy Society Annual Meeting, Hongjie Yuan will deliver a poster presentation describing a standardized approach to characterize genetic variants in the N-methyl-D-aspartate (NMDA) receptor GRIN gene family as gain-of-function or loss-of-function. We hope this will have a positive impact on #clinicalresearch programs for GRIN-related disorders, including the ongoing trial we are supporting through GRIN Therapeutics. Learn more and see the #AES2023 agenda: https://lnkd.in/ePusYH4y