Authors
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Carrie Miceli, PhD, on the Potential of Needle Biopsy Findings to Improve DMD Gene TherapyThe professor of microbiology, immunology, and molecular genetics at UCLA discussed how a better understanding of treatment impact on a cellular level could help improve future gene therapy approaches.
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Casey Maguire, PhD, on Potential Advantages of Encapsulated AAVsThe associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.
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Gene Therapy for Sickle Cell DiseaseExperts discuss the safety profile and cost of gene therapy for the management of sickle cell disease.
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Developing Gene Therapies for AMD: Brandi L. Williams, PhD; Gregory S. Hageman, PhDDirectors from the Moran Eye Center discussed researching gene therapy approaches targeting HTRA1 in AMD.
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IRD Gene Therapy OCU400’s Phase 1/2 TrialArun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.
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CARTITUDE Trials in Multiple MyelomaExperts discussed safety and efficacy data of cilta-cel for R/R MM from the CARTITUDE-1 and CARTITUDE-2 studies.
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Matthew D. Weitzman, PhD, and Matthew Charman, PhD, on Further Investigation of the Viral Reproduction ProcessThe investigators from Children’s Hospital of Philadelphia discussed the follow-up studies they are conducting in light of their recent findings regarding DNA virus replication.
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Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart FailureThe medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.
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Debora Mazzetti, MS, on Multitargeting MicroRNA in GlioblastomaThe research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.
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CAR T Therapy Yields Remission in Systemic Lupus ErythematosusFive patients with lupus treated with anti-CD19 CAR T-cell therapy are now in remission.
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Zachary Crees, MD, on the Potential of Cell Therapy to Treat or Prevent Viral Infections After HSCTThe assistant clinical director of the Center for Gene and Cellular Immunotherapy at Washington University in St. Louis discussed Atara Biotherapeutics' tabelecleucel and AlloVir’s posoleucel.
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Future Research With NK and CAR T Therapy in Multiple Myeloma: Irene Ghobrial, MDThe director at Dana-Farber Cancer Institute discussed future efforts to prevent the development of multiple myeloma.
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Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet SyndromeThe chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.
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Strategies to Reverse Multiple Sclerosis Neurodegeneration: Robert Zivadinov, MD, PhDThe director of the Buffalo Neuroimaging Analysis Center and professor of Universtiy of Buffalo discussed avenues of research into reversing neurodegeneration in MS.
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Kim A. Reiss Binder, MD, on the Potential of CAR Macrophages in Solid TumorsThe assistant professor of medicine at University of Pennsylvania Hospital stressed the importance of tumor sequencing.
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Two ACGT all-stars celebrated with ‘Oscars of Science’ awardCarl June, MD, and Michel Sadelain, MD, PhD, were recognized for their pioneering achievements in treating cancer.
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Ivan Horak, MD, on Early Clinical Efficacy of Allogeneic CD30.CAR EBVST Cell Therapy in LymphomasThe chief medical and scientific officer of Tessa Therapeutics discussed data from the BESTA and CHARIOT studies presented at ASH 2022.
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Advantages of Developing Platform-Agnostic Gene TherapiesThe chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.
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Everett Meyer, MD, PhD, on Reducing GvHD in Leukemia TreatmentThe assistant professor of medicine and pediatrics at Stanford University discussed updated data from 2 studies of Orca-T cell therapy.
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Matthew Gornet, MD, on Treating Degenerative Disc Disease With Cell TherapyThe spine surgeon from The Orthopedic Center of St. Louis discussed recent data on IDCT from the phase 1/2 DGX-A01 study.
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PJ Brooks, PhD, on Improved Newborn Screening, Non-Viral Gene Editing: New Frontiers for Neuromuscular DiseaseThe deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed areas that the agency is targeting.
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Madeleine Powys, MBBS, on Lessons Learned With LibmeldyThe locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.
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Farah Sheikh, PhD, on Preclinical Research on Arrhythmogenic Cardiomyopathy Gene Therapy LX2020The professor of medicine at University of California San Diego discussed promising preclinical findings she presented at the American Heart Association’s Scientific Sessions 2023.
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Deyaa Adib, MD, on Optimizing Manufacturing of Solid Tumor Cell TherapiesThe chief medical officer of Triumvira Immunologics discussed the company’s future plans.
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Christa Krupski, DO, MPH, on Reinfusion of Tisagenlecleucel in B-ALLThe hematologist/oncologist at Cincinnati Children’s discussed unfavorable outcomes with tisa-cel reinfusion in patients with B-cell acute lymphocytic leukemia.
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Managing Safety in Fitusiran Treatments of Hemophilia: Guy Young, MDThe director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed mitigation strategies in trials and clinic.
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Matthew D. Weitzman, PhD, and Matthew Charman, PhD, on Further Investigation of the Viral Reproduction ProcessThe investigators from Children’s Hospital of Philadelphia discussed the follow-up studies they are conducting in light of their recent findings regarding DNA virus replication.
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Raj Mehra, PhD, and Krishna Subramanian, PhD, on Parkinson Disease Gene Therapy Mouse StudiesMehra and Subramanian discussed preclinical safety studies with the gene therapy SLS-004 and plans for future research.
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Atul Malhotra, MD, PhD, on Treating Preterm Infants With Autologous Stem Cell TherapyThe head of the early neurodevelopment clinic at Monash Children's Hospital discussed clinical trials in the field of neonatal cell therapy.
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Raj Mehra, PhD, and Krishna Subramanian, PhD, on Parkinson Disease Gene Therapy Mouse StudiesMehra and Subramanian discussed preclinical safety studies with the gene therapy SLS-004 and plans for future research.
