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Text of the page (random words):
he use of car t in neurology 5 months ago 1 19 the challenge of t cell therapy timing 6 months ago 0 31 bad t cells lead to failed cancer treatments 6 months ago 0 42 why one target isn t enough the future of neuroblastoma therapy 6 months ago 1 02 what should the medical community know about deramiocel 7 months ago 0 48 where is neuromuscular medicine heading in the future 7 months ago 0 47 the potential of bringing hemophilia b gene therapy to younger patients 7 months ago 0 41 the need for more data for sickle cell gene therapy 7 months ago previous slide next slide podcasts view more april 3 2026 immunologic episode 9 rewiring immunotherapy for high risk neuroblastoma with kristopher r bosse md february 10 2026 immunologic episode 8 surgery meets immunotherapy a one two punch for breast cancer with julia c tchou md phd facs january 2 2026 immunologic bonus episode chemo optional data mandatory a year end recap december 24 2025 immunologic bonus episode ash 2025 practice changers on the ground and on the wi fi december 2 2025 immunologic episode 7 turbocharging car t cells the il 9 orthogonal signal with anusha kalbasi md previous slide next slide videos previous slide next slide brian lin phd on using genetics biomarkers and clinical trial readiness to enhance als care steven w pipe md on integrating gene therapy into clinical care for hemophilia b angela lek phd on the priorities and agenda for mda s 2026 conference interview highlights from ash s 2025 meeting emily brown mgc cgc on models for expanding access to cardiac genetic counseling steven w pipe md on the implications of the final data from hemgenix s hope b trial rabi hanna md on practical considerations for integrating gene therapy steven w pipe md on the end of study results from hemgenix s hope b trial rabi hanna md on evaluating gene editing therapy reni cel for severe sickle cell disease deevyashali parekh mbbs on evaluating car t in patients with multiple myeloma and renal failure continuing medical education video cpe track community oncology connections leveraging recent advances in the treatment of relapsed refractory follicular lymphoma new york lori a leslie md bachar samra md view more video cpe track community oncology connections leveraging recent advances in the treatment of relapsed refractory follicular lymphoma north carolina krish patel md tara graff do ms view more video cpe track community oncology connections leveraging recent advances in the treatment of relapsed refractory follicular lymphoma montana wyoming lori a leslie md jack w erter iii md mmhc view more video cpe track community oncology connections leveraging recent advances in the treatment of relapsed refractory follicular lymphoma washington krish patel md girish kunapareddy md view more case based simulation simulated partnering for precision a framework for personalized care planning in acute lymphoblastic leukemia jae park md view more video 9th annual school of nursing oncology beth faiman phd msn apn bc bmtcn aocn faan fapo beth sandy msn crnp fapo lindsay adkins msn fnp bc bmtcn jeneth aquino dnp fnp bc casey gormley msn fnp c aocnp heather j jackson phd fnp bc kelsey martin ag acnp bc aocnp nerina t mcdonald pa c lauren verity moore dnp msn agacnp bc faith a mutale dnp crnp tiffany richards phd anp bc aocnp emily skotte dnp msn acnp bc leslie smith dnp rn aprn cns aocns bmtcn saneese stephen pa c mpas sara m tinsley vance phd aprn aocn view more video cpe track community oncology connections leveraging recent advances in the treatment of relapsed refractory follicular lymphoma nevada krish patel md matthew ulrickson md view more video cpe track community oncology connections leveraging recent advances in the treatment of relapsed refractory follicular lymphoma kentucky lori a leslie md sushil bhardwaj md facp view more video cases and conversations optimizing outcomes in pv and et evolving standards symptom management and therapeutic innovation john o mascarenhas md marina kremyanskaya md phd douglas a tremblay md view more video oncology town hall shaping the future of all treatment through innovation in t cell therapies claire roddie md phd elias jabbour md hagop kantarjian md view more multimedia community practice connections next generation therapies in aml menin inhibitors idh1 2 combinations and beyond naval daver md view more video cme track tackling oncologic emergencies in patients treated with high dose methotrexate shazia nakhoda md shruti gupta md mph anitha varghese msn aprn agpcnp c view more video medical crossfire experts debate the hottest topics in aml flt3 idh1 2 kmt2ar npm1 secondary aml maintenance novel combinations and emerging therapies naval daver md daniel a pollyea md gail j roboz md amer zeidan mbbs mhs view more video real world insights on advances in the management of myeloproliferative neoplasms how the experts apply the latest developments to clinical practice naveen pemmaraju md shannon elf phd angela fleischman md phd bethan psaila md view more video per tumor board charting a new course in the treatment of newly diagnosed multiple myeloma ajai chari md maria victoria mateos md phd mohamad mohty md phd view more video cases and conversations optimizing patient outcomes in pediatric aya and adult all lbl with asparaginase therapies and combinations daniel j deangelo md phd shira dinner md stephen p hunger md jae h park md view more video advances in all management across the patient spectrum integrating innovation and precision care elias jabbour md hagop kantarjian md aaron logan md phd mphil rob pieters md phd claire roddie md phd view more adaptive learning medical crossfire navigating chronic gvhd prophylaxis and treatment targeted strategies to elevate patient outcomes corey cutler md mph frcp c amin m alousi md mehdi hamadani md anna sureda md phd view more video cme track medical crossfire menin inhibitors in aml dissecting the data to define the role naval daver md courtney dinardo md eunice wang md view more adaptive learning show me the data how today s evidence is shaping tomorrow s management and prophylaxis of chronic gvhd carrie l kitko md miguel angel perales md amandeep salhotra md view more video cpe track tackling oncologic emergencies in patients treated with high dose methotrexate shazia nakhoda md shruti gupta md mph anitha varghese msn aprn agpcnp c view more previous slide next slide advertisement all news advertisement fda extends deramiocel review for duchenne muscular dystrophy august 24th 2026 the fda extended its review of deramiocel for duchenne muscular dystrophy to november 22 2026 after accepting additional hope 3 data as a major bla amendment ptc wins sangamo s fabry gene therapy in bankruptcy auction august 24th 2026 ptc therapeutics wins sangamo s bla stage fabry disease gene therapy st 920 for 111 million in a bankruptcy auction with staar data supporting accelerated approval pariglasgene brecaparvovec opnr approved for glycogen storage disease 1a by matt hoffman august 20th 2026 fda granted accelerated approval to pariglasgene brecaparvovec opnr a one time liver directed aav8 gene therapy based on a 31 reduction in cornstarch intake versus placebo miv cel earns rmat designation for progressive multiple sclerosis august 20th 2026 fda grants kyverna s miv cel rmat designation in non active secondary progressive ms based on phase 1 data showing car t expansion and b cell reconstitution j j secures 2 58b option to acquire sail biomedicines august 17th 2026 johnson johnson secures an exclusive option to acquire sail biomedicines for 2 58 billion backing the biotech s in vivo car t platform for immune mediated disease fda panel votes against deramiocel for duchenne cardiomyopathy august 14th 2026 fda advisory committee votes 9 3 against deramiocel s effectiveness for duchenne cardiomyopathy pdufa decision due august 22 2026 rugonersen s phase 3 beacon trial doses first participant by marco meglio august 12th 2026 oak hill bio announced dosing of the first participant in beacon a pivotal phase 3 trial evaluating rugonersen an antisense oligonucleotide originally developed by roche for angelman syndrome cell therapy tg c misses primary endpoints in phase 3 knee osteoarthritis trial by marco meglio july 23rd 2026 the phase 3 activion ii trial found that a single intra articular injection of tg c did not improve pain or function compared with placebo at 12 months in patients with knee osteoarthritis missing both co primary endpoints liver directed gene therapy shows durable effect in mma mouse model by marco meglio july 21st 2026 a single systemic dose of an immune shielded lentiviral vector delivering the mmut gene produced lasting metabolic correction in a mouse model of methylmalonic acidemia according to newly published preclinical data aspen neuroscience completes aspiro cohort 3 and 4 dosing advancing sasineprocel toward phase 3 by marco meglio july 9th 2026 the milestone advances the commercial ready cryopreserved formulation of the autologous ipsc derived therapy into broader clinical experience as aspen prepares for phase 3 initiation advertisement first patient dosed in phase 2b 3 naavigate trial of gene therapy surabgene lomparvovec in diabetic retinopathy by marco meglio july 7th 2026 regenxbio will receive a 100 million milestone from abbvie following the first dosing in naavigate a pivotal trial evaluating one time suprachoroidal gene therapy in nonproliferative diabetic retinopathy without center involved macular edema fda approves tregzi for matched donor hsct in hematologic malignancies by cgtlive staff june 30th 2026 the fda approved tregzi as the first allogeneic regulatory t cell based immunotherapy based on precision t phase 3 data showing a significant 1 year cgvhd free survival rate of 78 vs 38 with conventional allohsc around the helix cell and gene therapy company updates may 27 2026 by noah stansfield may 27th 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies evaluating miv cel for stiff person syndrome by amanda l piquet md noah stansfield may 22nd 2026 amanda piquet md faan discussed phase 2 data from a trial evaluating the car t therapy mivocabtagene autoleucel for the treatment of stiff person syndrome around the helix cell and gene therapy company updates may 20 2026 by noah stansfield may 20th 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies phase 3 hope 3 data reinforce skeletal and cardiac benefits of deramiocel in duchenne muscular dystrophy by marco meglio may 18th 2026 phase 3 hope 3 data further support deramiocel s potential to preserve both skeletal and cardiac function in duchenne muscular dystrophy while slowing progression of myocardial fibrosis assessing miv cel for myasthenia gravis by srikanth muppidi md noah stansfield may 15th 2026 srikanth muppidi md spoke about data from the kysa 6 trial evaluating kyverna s car t mivocabtagene autoleucel in mg asgct 2026 gs 100 gene therapy demonstrates early activity in ngly1 deficiency by marco meglio may 14th 2026 new interim asgct 2026 findings suggest investigational aav9 gene therapy gs 100 may improve motor and cognitive outcomes in children with ngly1 deficiency while informing dose optimization and immune management strategies etx101 gene therapy shows early developmental seizure benefits in dravet syndrome by marco meglio may 13th 2026 new asgct 2026 data suggest encoded therapeutics investigational gene therapy etx101 may meaningfully reduce seizures and alter developmental trajectory in children with scn1a positive dravet syndrome around the helix cell and gene therapy company updates may 13 2026 by noah stansfield may 13th 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies beacon therapeutics reports sustained 12 month visual gains with laru zova in xlrp by marco meglio may 10th 2026 new phase 2 dawn data presented at arvo 2026 showed sustained improvements in visual function and favorable safety with laru zova in patients with x linked retinitis pigmentosa around the helix cell and gene therapy company updates may 6 2026 by noah stansfield may 6th 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies around the helix cell and gene therapy company updates april 29 2026 by noah stansfield april 29th 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies lonvo z advances toward approval as first in vivo crispr therapy for hereditary angioedema by marco meglio april 27th 2026 intellia has initiated a rolling fda submission for lonvo z after phase 3 data showed significant reductions in hereditary angioedema attacks and treatment burden with a single dose regeneron nets accelerated approval from fda for otoferlin related hearing loss gene therapy lunsotogene parvec by noah stansfield april 24th 2026 the therapy will notably be provided for free in the united states around the helix cell and gene therapy company updates april 22 2026 by noah stansfield april 22nd 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies evaluating gene therapy bb 301 for opmd related dysphagia by jerel a banks md phd noah stansfield april 19th 2026 jerel a banks md phd the ceo of benitec biopharma discussed data he presented at mda s 2026 conference early biomarker signals from sgt 003 highlight ongoing progress in duchenne gene therapy by marco meglio aravindhan veerapandiyan md april 17th 2026 investigators discuss interim phase 1 2 inspire duchenne data and the emerging biomarker and safety profile of sgt 003 in duchenne muscular dystrophy reimagining sma treatment salanersen and the push for durable low frequency therapies by marco meglio diana castro md april 16th 2026 diana castro md highlights early phase 1b findings on salanersen and its potential to reshape treatment burden in spinal muscular atrophy around the helix cell and gene therapy company updates april 15 2026 by noah stansfield april 15th 2026 catch up on the latest news breakthroughs and announcements from biotechnology companies making advancements in cell and gene therapies view all news advertisement development watch latest regulatory safety events across tracked therapies september 8 2026 update lonvo z fda accepts bla with priority review pdufa target march 10 2027 july 1 2026 update casgevy fda expands both indications to ages 2 april 27 2026 update lonvo z positive phase 3 topline rolling bla submission initiated how we track this advertisement about us editorial contact us terms and conditions do not sell my 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