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Keys to a Successful Cell and Gene Therapy LaunchThe success of cell and gene therapies relies not just on approvability but on affordability and accessibility. Innovative manufacturing, automation, and digital tools are key to expanding patient access and ensuring commercial viability.
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Gene Therapy Will Be a Major Area of Interest for Attendees at MDA’s 2025 ConferencePaul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, discussed the growing interest in gene therapy for the annual meeting.
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Magdalena Cichewicz, PhD, on Improving AAV Gene Therapy With Synthetic PromotersThe scientist II at Senti Biosciences discussed the potential of synthetic promoters to improve target specificity in AAV vector-based gene therapies in retinal diseases and beyond.
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Clive Svendsen, PhD, on Unlocking the Potential of Astrocyte Cell TherapyThe director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
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Emilie Aschenbrenner, PharmD, BCOP, on CAR-T Versus Bispecific Antibodies for the Treatment of LBCLThe hematology coordinator for pharmacy at Froedtert and the Medical College of Wisconsin discussed the advantages of each of the 2 modalities in the third and second line settings.
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Delivering Ultracompact CRISPR Systems With AAVLucas Harrington, PhD, the cofounder and chief scientific officer of Mammoth Biosciences, discussed the company’s mouse model research on treating hypertriglyceridemia.
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David Weinstein, MD, on Delivering Gene Therapy Directly to the BrainThe senior vice president of clinical development at Passage Bio discussed intracisternal magna administration of PBGM01.
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Frank Borriello, MD, PhD, on Next Steps for SUPLEXA-101 for Solid TumorsThe scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed progress of the phase 1 trial and possible future evaluations of the therapy.
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Marty Giedlin, PhD, on Advantages of Allogeneic NK Cell Therapy ApproachesThe senior vice president of Technical Operations at Senti Biosciences discussed the company’s NK cell logic-gating platform and allogeneic approach.
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Noninflammatory Neurotoxicity Events in Patients Receiving CAR-TCaroline Diorio, MD, FRCPC, FAAP, an attending physician at the Cancer Center at Children's Hospital of Philadelphia, discussed a study she coauthored that was recently published in Blood.
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Unraveling the Genetics of GlaucomaDetails enhance knowledge of biological pathways that contribute to disease pathogenesis.
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Unraveling the Genetics of GlaucomaDetails enhance knowledge of biological pathways that contribute to disease pathogenesis.
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Dr. Hwu on the Uptake of Cellular Therapy in OncologyPatrick Hwu, MD, discusses the uptake of cellular therapy in the treatment of patients with cancer.
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Binod Dhakal, MD, on Benefit of Cilta-Cel in Earlier Lines of Multiple Myeloma TreatmentThe associate professor at Medical College of Wisconsin discussed current studies evaluating cilta-cel and ongoing research.
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Stem Cell Therapy for TBI: Masahito Kawabori, MD, PhDThe associate professor at Hokkaido University and investigator of STEMTRA also discussed other efforts in stroke, Alzheimer disease, and Parkinson disease.
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Gene Transfer Therapy Requires Further Investigation Despite Potential Approval, Review SaysAfter a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.
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Dr. Hutson on Challenges With Targeted Therapy in Non-Clear Cell RCCThomas Hutson, DO, PharmD, discusses challenges with targeted therapy in non-clear cell renal cell carcinoma.
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The Potential of Gene Transfer Therapy in SMAJohn Brandsema, MD, discusses gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.
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Dr. Till on the Potential to Utilize CAR T-Cell Therapy Earlier in MCLBrian Till, MD, discusses the potential to utilize CAR T-cell therapy earlier in the treatment of patients with mantle cell lymphoma.
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Cilta-Cel Demonstrates Durable Responses at 18 Months in Relapsed/Refractory Multiple MyelomaCiltacabtagene autoleucel, an investigational BCMA-directed CAR-T therapy, sustained efficacy and durable responses in heavily pretreated patients with relapsed/refractory multiple myeloma.
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The Role of CAR NK Cells: Competition or Compliment to CAR T-Cell Therapy?New studies into chimeric antigen receptor (CAR)–engineered natural killer (NK) cells has shown promising results, explained Ulrike Köhl, PhD, MD, professor of immune oncology and director of the Institute for Clinical Immunology at the University of Leipzig in Germany.
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Gene Transfer for Heart Failure Shows PromiseIntracoronary gene transfer among heart failure patients increased left ventricular function beyond standard heart failure therapy.
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Delays With Payers Mean Some Patients Miss Window for CAR T-Cell Therapy, Findings ShowAjeet Gajra, MD, MBBS, FACP, talks about identifying and removing the barriers for offering CAR T-cell therapy at the community practice level.
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DMD Trial Results Demonstrate Decrease in Creatine Kinase LevelsResults of a Phase 1/2a gene therapy clinical trial in children with Duchenne muscular dystrophy (DMD) revealed a significant decrease in levels of serum creatine kinase, an enzyme biomarker associated with muscle damage caused by DMD.
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Ophthalmology Overview: Gene Therapy Partially Restores Vision in Blind Patient, Presbyopia Therapy, and MoreHighlighting the latest ophthalmology-related news reported across MJH Life Sciences™.
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Scaling the Cell and Gene Therapy Supply Chain for GrowthWhile the cell and gene therapies approved so far are indicated for rare diseases with small patient populations, the successes of chimeric antigen receptor-T (CAR-T) therapies and expanding interest from biopharma stress the need to rapidly scale the supply chain as these therapies move toward commercial availability for more disease states and larger patient populations.
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Selective Inhibitor of FLT3 Allows High-Risk AML Patients to Bridge to Stem Cell TransplantA unique targeted therapy, quizartinib, was able to clear leukemia cells from the bone marrow in more than 33% of patients with an aggressive form of acute myeloid leukemia marked by a mutation in the FLT3 gene.
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Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
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Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
