Authors
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Jonathan W. Weinsaft, MD, on Integrating Genetic Research into Cardiovascular MedicineThe chief of cardiology at Weill Cornell Medical College discussed the center’s increased focus on genetic research.
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Bart P. Leroy, MD, PhD, on Aligning IRD Gene Therapy Outcome Measures With Activities of Daily LivingThe head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital discussed the need to reevaluate requirements for regulatory approval for ophthalmology gene therapies.
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Evan Weber, PhD, on Improving CAR T-Cell FitnessThe assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.
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Detecting Translocations in Gene Edited CellsVanee Pho, PhD, the chief product officer at Mission Bio, discussed the company’s Genome Editing Solution.
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Deevyashali Parekh, MBBS, on Evaluating the Prevalence of Second Primary Malignancies After CAR T-Cell TherapyThe internal medicine resident at SUNY Upstate Medical University Hospital discussed the implications of findings from a deidentified database of EMR data.
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Tiffany Chen, PhD, on the Role of Preclinical Models in Cell Therapy Research for Autoimmune DiseaseThe vice president of discovery at GentiBio discussed the panel she participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.
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Efficient Gene Editing in Monogenic DiseasesAndré Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies.
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Stephanie Cherqui, PhD, on Bringing Attention to Cystinosis and Gene TherapyThe associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.
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Genovefa (Zenia) Papanicolaou, MD, on Managing Infectious Diseases in Patients Receiving Stem Cell Transplant or Cell TherapyThe infectious diseases specialist at Memorial Sloan Kettering Cancer Center discussed the session she chaired for the Infectious Diseases Track at Tandem’s 2024 Meeting.
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BASECAMP-1 Trial Identifies Patients With Solid Tumors to Assess Novel CAR T-Cell TherapyJ. Randolph Hecht, MD, discusses the challenges researchers have faced in the development of cellular therapy for patients with solid tumors, and details how A2B530 could potentially overcome some of these obstacles.
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Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.
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Sickle Cell Disease Gene Therapy Exa-Cel's Ability to Prevent VOCsHaydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, discussed the latest data update from the CLIMB SCD-121 trial evaluating exa-cel.
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Robert J. Hariri, MD, PhD, on the Unique Potential of Chimeric TherapyThe founder, chairman, and CEO of Celularity shared his view on the concept of “chimeric vigor” and the potential benefits arising from it in the cell therapy field.
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Weijia Fang, MD, and Guoqiang Ai, MD, on the Future of IMC001 in Gastric and Colorectal CancerFang commented on a limitation of the clinical trial pointed out at the European Society for Medical Oncology (ESMO) 2022 Congress.
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Faraz Ali, MBA, on Trends in Precision Medicine for Cardiovascular DiseaseThe chief executive officer of Tenaya Therapeutics discussed the growing interest in genomic medicines in cardiology.
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The Power of Partnership in CMT Gene Therapy ResearchSusan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the origins of the CMTRF.
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Michael Severino on In Vivo Gene Editing With RNA Gene WritersThe chief executive officer of Tessera Therapeutics discussed ongoing research into developing its platform for sickle cell disease, CAR T-cell therapy, and more.
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Alfred L. Garfall, MD, MS, on Ide-Cel and Lenalidomide Maintenance in MM Following Suboptimal ASCT ResponseThe director of the Autologous Hematopoietic Cell Transplantation, Cell Therapy and Transplant Program at the Hospital of the University of Pennsylvania discussed data from the phase 2 BMT CTN 1902 trial.
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Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene-Edited Cell Therapy in PKDThe clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.
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Selecting Cell Therapy Targets in CancerThe chief executive and medical officers of Cytovia Therapeutics discussed the company's therapeutic targets and cell therapy scaffold.
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Vlad Hogenhuis, MD, MBA, on Developing Controllable CAR Ts for Solid TumorsThe chief executive officer of Chimera Bioengineering discussed CBIO-007 and its planned IND submission for colorectal and triple negative breast cancer.
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Brett Kopelan, MA, on the Future Treatment of Epidermolysis Bullosa With Advanced TherapeuticsIn observance of Epidermolysis Bullosa Awareness Week, the executive director of debra of America discussed how a combination of advanced and topical approaches may represent the future of EB treatment.
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Dr. Anderson on Addressing Unmet Needs in Myeloma With Descartes-11Kenneth C. Anderson, MD, discusses potentially addressing unmet needs in patients with multiple myeloma through the use of the mRNA-based CAR T-cell therapy Descartes-11.
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Jason Fontenot, PhD, on Overcoming the Challenge of Delivery for Zinc Finger Epigenetic RepressorsThe chief scientific officer of Sangamo Therapeutics discussed the company’s AAV capsid evolution research.
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As Gene Editing Technology Advances, Numerous Potential Modalities EmergeJanice Chen, PhD, the cofounder and chief technology officer of Mammoth Biosciences, discussed the importance of diverse approaches to gene editing to address a variety of indications.
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CARsgen Therapeutics’ CT041 Shows Promise in Advanced Gastric/Gastroesophageal Junction AdenocarcinomaThe chimeric antigen receptor-modified autologous T cells targeting CLDN18.2 had a manageable safety/tolerability profile and promising efficacy.
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Addressing Unmet Needs With Gene TherapyEric Crombez, PhD, senior vice president and chief medical officer of Ultragenyx Gene Therapy, discussed indications the company is targeting.
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Aimee Donald, MBChB, PhD, on Making Progress With Gene Therapy in Lysosomal Storage DiseasesThe pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.
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Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhDThe chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.
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Selecting Cell Therapy Targets in CancerThe chief executive and medical officers of Cytovia Therapeutics discussed the company's therapeutic targets and cell therapy scaffold.
