Authors
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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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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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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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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.
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Donald Kohn, MD, on the Trajectory of Gene TherapyThe renowned professor from University of California Los Angeles reflected on receiving the Outstanding Achievement Award from the ASGCT.
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Jeffrey S. Chamberlain, PhD, on Collaborating to Cure DMDThe professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.
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Challenges With Conducting Clinical Trials in Gaucher Disease: Neil Weinreb, MDThe chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.
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CGTLive’s Weekly Rewind – April 8, 2022Review top news and interview highlights from the week ending April 8, 2022.
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Developing Controllable Tumor Infiltrating Lymphocyte TherapiesJan ter Meulen, MD, PhD, chief scientific officer, Obsidian Therapeutics, discussed the potential of the cytoDRIVE platform.
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Breanna DiAndreth, PhD, on Improving Selective Targeting of Tumor CellsThe scientist at A2 Biotherapeutics discussed the company’s dual-receptor Tmod technology.
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Looking Forward in Relapsed/Refractory Multiple Myeloma TreatmentsJames Hoffman, MD, leads a discussion about recently approved therapies and exciting future treatment options for relapsed/refractory multiple myeloma.
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Jennifer Kwon, PhD, on Reducing LDL Cholesterol in NHPs With Epigenome EditingThe senior scientist at Tune Therapeutics discussed preclinical research presented at the ASGCT 2023 meeting.
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Tackling Deceased Kidney Transplant and Severe Scleroderma With Cell TherapyScott Requadt, chief executive officer, Talaris Therapeutics, discussed the phase 2 FREEDOM-3 trial and preclinical studies of the cell therapy FCR-001.
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Gene Editing's Potential for COVID-19 and Other IndicationsNew human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.
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Vishal Patel, MD, on the Potential Advantages of a Gene Therapy Approach for Polycystic Kidney DiseasePatel discussed the potential advantages of his team’s gene therapy approach compared to an investigational PKD drug.
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Working Toward a Single Dose Gene Therapy for Type 1 Gaucher DiseaseOlga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.
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Unique Challenges With Gene Therapy in Duchenne Muscular DystrophyThe co-founder, president, and chief executive officer of Solid Biosciences, whose own son has DMD, discussed challenges in developing gene therapies for DMD.
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Weijia Fang, MD, and Tianhang Luo, MD, on EpCAM-Targeted CAR-T Therapy in Advanced Colorectal and Gastric CancerFang recently presented at the European Society for Medical Oncology (ESMO) 2022 Congress.
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Carol Miao, PhD, on Delivering Gene Editing Tools With Nonviral Methods in Hemophilia A ModelsThe principal investigator at Seattle Children’s Research Institute discussed her lab’s preclinical research on nonviral delivery methods for gene editing tools in the context of treating hemophilia A.
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Optimizing COVID-19 Vaccines in Patients Receiving CAR T Therapy: Muhammad Bilal Abid, MD, MRCPThe assistant professor of medicine at the Medical College of Wisconsin discussed immune-compromising factors that are indigenous to CAR T-cell therapy recipients.
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Gavin Macbeath, PhD, on Screening for Off-Target EffectsThe chief scientific officer at TScan Therapeutics discussed the company’s SafetyScan technology.
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Gene Therapy Optogenetics Combination Efficacious in Retinitis Pigmentosa: José-Alain Sahel, MDThe professor from University of Pittsburgh School of Medicine discussed the combination of an optogenetics device and gene therapy in treatment of RP.
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Developing Safer, Next-Generation CAR T-Cell TherapiesRick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s programs and future plans.
