Significant Advancement in DMD Treatment by Sarepta Therapeutics! While Pfizer's Phase 3 CIFFREO trial for DMD therapy faced setbacks, last Thursday marked a positive shift with Sarepta's ELEVIDYS label receiving FDA approval, expanding treatment options significantly. Previously, only DMD boys between aged 4 and 5 were FDA approved to receive the therapy, and now, all young DMD boys and men aged 4 and above will have access per the FDA expansion. In addition, previously, there were strict ambulatory restrictions whereby if the child required assistance with ambulation, they were excluded from having access, whereby, now, this milestone FDA expansion includes full approval for non-wheelchair-dependent patients and accelerated approval for those who are wheelchair-dependent. Despite initial challenges, secondary clinical trial results demonstrating improved motor functions supported this decision, bringing renewed hope to families affected by this devastating disease. We anticipate many DMD boys who were denied access over the past year, may now meet guidelines for access to this potentially life-changing treatment option per the FDA expansion. Symphony Benefit Solutions is staying on top of developing stories like this in the gene and cell therapy arena. We are excited for the 7/1 launch of our fully integrated program, which will tackle high-cost gene and cell therapy treatments while remaining at the forefront of this rapidly changing environment. Reach out to us to learn more about how Symphony Benefits Solution can help at [email protected] This development highlights the continued progress and breakthroughs in the fight against DMD. #DMD #GeneTherapy #SareptaTherapeutics #FDAApproval #MedicalBreakthrough #SymphonyBenefitSolutions
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FDA grants accelerated approval to repotrectinib for adult and pediatric patients with NTRK gene fusion-positive solid tumors On June 13, 2024, the Food and Drug Administration granted accelerated approval to repotrectinib (AUGTYRO, Bristol-Myers Squibb Company) for adult and pediatric patients 12 years and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and that have progressed following treatment or have no satisfactory alternative therapy. #fda #drugapproval #oncology https://lnkd.in/g2tx7wg6
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In a conversation with Luisa Torres, PhD of Drug Discovery News, Kyverna Therapeutics Chief Medical Officer James Chung discusses the potential of CAR T-cell therapy for multiple sclerosis, gives an update on #KYV101’s clinical progress, and expresses his enthusiasm for the future of autoimmune disease research at Kyverna. Chung also shares how there is an opportunity to use CAR T cells to test the hypothesis about B cell depletion in a more complete way and to see if there could be unprecedented levels of efficacy. #CARTcelltherapy #AutoimmuneDisease #MultipleSclerosis https://lnkd.in/eqTBG8Yw
Kyverna Therapeutics tests CAR T cells for autoimmune disease
drugdiscoverynews.com
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CEO and Founder of Genoplex.ai | Comms Committee @ ASGCT | Host of Rx for Biotech Podcast & TEDx Speaker
#celltherapy to treat #autoimmune disease is the next treatment frontier as adoptive cellular therapies move beyond oncology and into other larger, more prevalent diseases with high unmet clinical need. Learn more about this emerging treatment modality in the story below... #advancedtherapies #cartcelltherapy
What is the potential for CAR-T cell therapies to treat autoimmune diseases? BioPharma Dive reviews how these therapies work, how they are different, and which companies are involved in their development, such as ARM members, Bristol Myers Squibb, Kyverna Therapeutics, ImmPACT Bio, Cabaletta Bio, CRISPR Therapeutics, and Autolus Therapeutics. Read the full story: https://lnkd.in/gcpEbyPF
CAR-T for lupus: the ‘tip of the iceberg' for cell therapy in autoimmune disease
biopharmadive.com
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If you are interested in #cellcycle science and its impact on innovative #breastcancer therapeutics, check out our recent work seamlessly integrating biological and #drugdevelopment perspectives in Clinical and Translational Medicine
Seize the engine: Emerging cell cycle targets in breast cancer
onlinelibrary.wiley.com
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More than 2 years ago, Healio reported on the groundbreaking cohort of #lupus patients undergoing #CART cell therapy and the explosive potential to treat the most severe and refractory forms of #AutoimmuneDisease 💥 2 years later, are we still waiting for the boom? 💥 “All five SLE patients are in remission and drug-free,” Dr. Georg Schett, whose lab produced the landmark 2022 study, said. “All of them are now 2 years disease-free, and the first of them is now 3 years disease free.” Since then, the potential for #CART cell therapy has proliferated across the rheumatology spectrum: 🔬 New data accrued for autoimmune myositis, #SystemicSclerosis and #MyastheniaGravis 💉 First treatments given in neuromyelitis optica and #MultipleSclerosis 👩⚕️ Potential indications for #RheumatoidArthritis, #Sjogrens syndrome, ANCA-associated #vasculitis and blistering skin diseases It is too soon to call CAR T cell therapy a “cure,” but this kind of excitement is rare in the #rheumatology space, and we are cautiously optimistic that – despite the checkered history of rheumatology therapies – this will lead to effective treatments in the most hard-to-treat populations 🤞 Join the conversation 💬
CAR T-cell therapy may be a ‘miracle,’ but it is too soon to say ‘cure’
healio.com
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Brainstorm Cell Therapeutics Announces Peer-reviewed Publication of #Biomarker Data from NurOwn's® Phase 3 Clinical Trial in #ALS. NurOwn treatment resulted in a positive impact on important #CSF biomarkers relevant to ALS compared to placebo. Significant changes in multiple ALS disease pathways support NurOwn's mechanism of action and complement clinical effects observed in ALS. https://lnkd.in/gv7R7FY3
BrainStorm Cell Therapeutics Announces Peer-reviewed Publication of Biomarker Data from NurOwn's® Phase 3 Clinical Trial in ALS
ir.brainstorm-cell.com
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Antibody drug conjugates (ADCs) are fast-growing fields and attract strong interests from antibody professionals. We are highlighting selected ADCs papers published on Antibody Therapeutics (current CiteScoreTracker: 8.3), official journal of the Chinese Antibody Society. The 5th paper was entitled “Mechanisms contributing to ado-trastuzumab emtansine-induced toxicities: a gateway to better understanding of ADC-associated toxicities” contributed by Wenjin Wu from U.S. Food and Drug Administration (FDA). We also highly recommend the special Issue Collection: Antibody-targeted Cytotoxic agents – Immunotoxins and ADCs. This special issue collection includes 14 excellent review and research articles. Special thanks to the two guests, Dr. Antonella Antignani and Dr. David FitzGerald at National Cancer Institute (NCI) of The National Institutes of Health. You may find more ADCs collection through https://lnkd.in/e76efmRZ #ADCs #Antibody #Therapeutics #Immunotoxins
Mechanisms contributing to ado-trastuzumab emtansine-induced toxicities: a gateway to better understanding of ADC-associated toxicities
academic.oup.com
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I love examples of how relatively simple (not requiring complicated genetic engineering) process changes can lead to significant improvements in CAR T cell function. These authors show how glucose-based to galactose-based media leads to increased mitochondrial activity and improved tumor control: https://lnkd.in/gmQdWAnf. Maybe this approach could be combined with low oxygen culture...? #cellandgenetherapy #biotechnology #lifesciences #manufacturing #manufacturingreadiness #advancedtherapies #therapeutics #pharma #pharmaceuticals #manufacturingtools #carttherapies #oncology #efficacy #potency
Improved CAR-T cell activity associated with increased mitochondrial function primed by galactose - Leukemia
nature.com
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📉 Impact ⤵ TG Therapeutics, Inc. has secured FDA clearance to begin a Phase I trial of #AzercabtageneZapreleucel (azer-cel), a novel allogeneic #CARTtherapy for progressive multiple sclerosis. This milestone marks a significant step forward in exploring CAR T technology beyond oncology, potentially offering new hope for patients with #chronicautoimmunediseases. 💡 Insight ⤵ The expansion of allogeneic CAR T therapy into autoimmune diseases like multiple sclerosis reflects the growing potential of innovative treatments in addressing unmet medical needs. This partnership between Precision BioSciences and TG Therapeutics highlights the importance of collaboration in advancing groundbreaking therapies that could transform patient care. ❓ Questions to Think About ⤵ How will the success of azer-cel influence the future of CAR T therapies in autoimmune diseases? What challenges might arise in translating oncology-focused CAR T technology to autoimmune conditions? How could this trial reshape treatment approaches for multiple sclerosis? #PharmaNews #CARTTherapy #MultipleSclerosis #AutoimmuneDisease #PrecisionMedicine #TGtherapeutics #HealthcareInnovation #GeneEditing #Biotech
FDA grants IND clearance for TG Therapeutics’ trial of multiple sclerosis treatment
clinicaltrialsarena.com
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Great article on CAR-T therapies for Lupus. Thought I'd add to the report with some additional numbers pulled from Beacon Immune Tolerance. - 61 cell therapy assets investigating SLE - 22 of these have already published preclinical efficacy readouts - 33/61 being specifically CAR-T - 360 assets investigating SLE across all drug modalities - with 72 of these drugs in preclinical dev. vs. 244 in the clinic Happy to dive deeper into these numbers for anyone who's interested. #sle #autoimmune #cartcelltherapy https://lnkd.in/exSj8z7i
CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease
biopharmadive.com
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