Ryan Murray, PhD’s Post

All that you need to know about enabling #mRNA delivery for transient protein expression with minimal toxicity via #LNP to address a range of manufacturing and safety challenges facing CAR-T Cell Therapy. #nanomedicine #cartcelltherapy #celltherapy

By enabling messenger RNA (mRNA) delivery for transient protein expression with minimal toxicity, lipid nanoparticles have the potential to address a range of manufacturing and safety challenges facing the CAR T-cell therapy field. As new studies are published demonstrating their advantages, it may not be long before LNPs start to displace electroporation in the non-viral CAR T delivery field. Read this article to learn more about the role LNPs are playing to advance CAR T cell therapies. #mrna #LNP #cartcelltherapy #tcells

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

Leading CAR-T investigators put together commentaries toward the US FDA's concerns on T cell malignancies, including chimeric antigen receptor (CAR)-positive lymphoma, in patients who received treatment with BCMA- or CD19-directed autologous CAR-T cell immunotherapies. The risk of T cell malignancies is applicable to all 6 approved BCMA-directed and CD19-directed genetically modified autologous CAR-T therapies (including Kymriah, Breyanzi, Yescarta, Tecartus, Abecma, Carvykti). Peter Marks, Director of the Center for Biologics Evaluation and Research, U.S. FDA stated on Monday (at ARM Cell & Gene State of the Industry Briefing) that "The overall benefits of (approved) CAR-T products continue to outweigh their potential risks for all approved uses" (Youtube Link: https://lnkd.in/ep4jhnXM). The CAR-T products were approved with the requirement to conduct 15-year long-term follow-up observational safety studies to assess the long-term safety and the risk of secondary or subsequent malignant neoplasms occurring after treatment. Peter now states that patients and participants of clinical trials receiving treatment with these products should be monitored life-long for new malignancies. The FDA hopes to provide the community with further information in early 2024. Dr. Bruce Levine posted a thread yesterday, along with the Nat Med comments, for some insightful discussions: https://lnkd.in/eTZjfP_s Nat Med publication link: https://lnkd.in/eibceMS5

Cartesian Therapeutics, an mRNA cell therapy biotech, announced its BCMA-directed CAR-T therapy Descartes-08 met the primary endpoint in a Phase 2b study for the nerve communication-destroying disease myasthenia gravis (MG). The biotech switched its primary endpoint from the MG-ADL symptom scale to MGC. Descartes-08 helped 71% of patients achieve 5-point or greater improvement on a 60-point scale of the disease. That compared to 25% of patients who received placebo, with a p-value of 0.018. Cartesian did share results from a per-protocol population that included 31 patients. In that population, the MGC response rates in the Descartes-08 and placebo arms were 69% and 33%, respectively, resulting in a p-value of 0.048

We need to further define the terms 'organoids' and 'spheroid' cells. Personally, I disagree with labeling patient-derived cells as 'organoids,' as discussed in this paper. However, this patient-derived cell screening approach could provide a valuable opportunity to discover novel therapeutic options, despite some limitations in immuno-oncology. https://lnkd.in/geVM3pKm

#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

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

Cartesian Therapeutics celebrates a Phase II win with their mRNA CAR-T therapy for myasthenia gravis. Descartes-08 showed a 71% improvement in patients, surpassing the 25% in the placebo group. Notably, this therapy does not require lymphodepleting chemotherapy, allowing for outpatient administration. Insight - Cartesian secured $130M in financing, fueling further development of their innovative mRNA platform and expanding into other autoimmune diseases like systemic lupus erythematosus. Key questions - How will Cartesian's mRNA CAR-T therapy reshape treatment for autoimmune diseases? What implications does this have for future cell therapies? #Biotech #CAR_T #AutoimmuneDisease #HealthcareInnovation #CartesianTherapeutics

The Evolving Treatment Landscape of Multiple Myeloma: GPRC5D-Targeted CAR T-Cell Therapy: Chimeric antigen receptor (CAR) T-cell therapy can engineer T cells to target malignant cells in multiple myeloma. #finance #pharmacy #lifesciences