Authors
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Targeting Cancers With CAR T and NK CellsJan Davidson, MD, PhD, chief medical officer, Wugen, discussed the company’s future plans and research.
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The Potential of ALLO-715 in Relapsed/Refractory Multiple Myeloma: Doris Hansen, MDThe hematologist from Moffitt Cancer Center discussed the promise of ALLO-715 in relapsed/refractory multiple myeloma.
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Gene Therapy Well-Tolerated in Late-Onset Pompe DiseaseInterim safety data from the FORTIS study were presented at WORLDSympsoium.
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Exploring Advanced Approaches for Pain ReliefThe chief executive officer and co-founder of Flexion Therapeutics discussed the company’s pain-focused pipeline, which includes a gene therapy that targets inflammation.
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Gene Therapy for APOE4 Homozygous Alzheimer DiseaseR. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
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Thomas Povsic, MD, PhD, on Ongoing Research With XC001 Angina Gene TherapyThe interventional cardiologist and professor, Duke University School of Medicine, discussed other populations that may benefit from XC001 and the challenges and opportunities with a surgical delivery of treatment.
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1-Year Data on Omidubicel Shows Sustained Benefits, Reduced Infection RatesThere was a continued trend toward improvement of overall survival with omidubicel at 73% compared with UCBT at 60%.
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The Growing Popularity of Gene Therapy for Retinal DiseasesFoundation Fighting Blindness is helping to support the development of gene therapies for retinal diseases.
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Advantages of CG0070 Over Standard Care for Non-Muscle Invasive Bladder CancerThe chief executive officer of CG Oncology discussed the role of CG0070 in patients who are unresponsive and responsive to Bacillus Calmette-Guerin immunotherapy.
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Re-Dosing With Gene Therapies in Rare DiseasesTakashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
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The Importance of Collaboration in Developing Next Generation Cell TherapiesDolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the company’s collaboration with 2seventy bio to develop TCR T-cell therapies.
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Gene Therapy for APOE4 Homozygous Alzheimer DiseaseR. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
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Devon J. Shedlock, PhD, on Shifting from Autologous to Allogeneic TherapiesThe chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.
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Mesenchymal Stem Cells and Other Upcoming Treatments for Progressive MS: Jeffrey Cohen, MDThe director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.
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Angela Genge, MD, FRCPC, eMBA, on Targeting STMN2 to Rescue Neurons in ALSThe chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.
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Next Steps for Car T-Cell Therapy in Multiple Myeloma: Faith E. Davies, MDThe director of the Clinical Myeloma Program at NYU Langone Health’s Perlmutter Cancer Center discusses the next steps for CAR T-cell therapy in the treatment of patients with relapsed/refractory multiple myeloma.
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Caspian Oliai, MD, MS, on Making Allo-Transplant Safer for Older PatientsThe medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.
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Clinical Implications of CAR T-Cell Therapy in R/R Myeloma: Frits van Rhee, MD, PhDThe professor and director of the Myeloma Center at the University of Arkansas for Medical Sciences discussed the clinical implications of CAR T-cell therapy for patients with relapsed/refractory multiple myeloma.
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Gene Therapy: The Future of Rare Disease TreatmentExperts shared opinions about the future of gene therapy in sickle cell and other rare diseases.
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Stem Cell vs Gene Therapy Processes in Sickle Cell DiseaseA discussion of the course of gene therapy compared with stem cell therapy.
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Stem Cell vs Gene Therapy Processes in Sickle Cell DiseaseA discussion of the course of gene therapy compared with stem cell therapy.
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Stem Cell vs Gene Therapy Processes in Sickle Cell DiseaseA discussion of the course of gene therapy compared with stem cell therapy.
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Carsten Bönnemann, MD, on Identifying At-Risk Genotypes for DMD Gene TherapyThe senior investigator from NINDS discussed findings investigating serious AEs across different trials.
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Ongoing Gene Therapy Trials in Stargardt Disease : Prof. Carel HoyngThe professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
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N. Nora Bennani, MD, on Diving Deeper Into T-Cell LymphomasThe assistant professor at Mayo Clinic School of Medicine shared her outlook and predictions on research with T-cell lymphomas.
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Transforming Challenges Into Action for SLC6A1In honor of Rare Disease Day, observed this year on February 28, Amber Freed, the founder of SLC6A1 Connect, shared thoughts on advocacy and efforts to raise awareness for rare genetic disease SLC6A1.
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B-VEC Shows Quality of Life Improvements in Dystrophic Epidermolysis BullosaKrystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.
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Giedre Krenciute, PhD, on Revitalizing T-cells in CAR T-cell TherapyThe assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.
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Pursuing Slow but Steady Gene Therapy DevelopmentMichael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.
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Aimee C. Talleur, MD, on the Challenge of Evaluating Late Effects from CAR-T in Pediatric PatientsThe physician from St. Jude Children’s Research Hospital discussed the importance of involvement from patients and families while researching the long-term impact of CAR-T.
