Leigh Ramos-Platt, MD, on Sharing Experience With Gene Therapy Administration
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her learning experience administering gene therapies for the first time.
Daniel Hart, PhD, on CRISPR-Mediated In Vivo Epigenomic Activation
The Senior Director and Head of Technology Development at Epic Bio discussed potential applications of the new technology.
Luke Roberts, MBBS, PhD, on Developing Gene Therapy for Congestive Heart Failure
The medical director of clinical development at AskBio discussed the progress of an early clinical trial of AB-1002.
PJ Brooks, PhD, on Improved Newborn Screening, Non-Viral Gene Editing: New Frontiers for Neuromuscular Disease
The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed areas that the agency is targeting.
Sowmya Viswanathan, PhD, on Translating Cell Therapies to the Clinic at ISCT 2024
The scientist from Krembil Research Institute shared takeaways from the session she chaired.
Omar Nadeem, MD, on Initial Efficacy of GPRC5D-CAR in R/R Multiple Myeloma
The medical oncologist and clinical director of Myeloma Cellular Therapies at Dana-Farber Cancer Institute emphasized promising safety in patients with high-risk disease.
Omer A. Abdul Hamid, MD, on Improving Gene Therapy’s Effect and Accessibility
The pediatric neurologist at Nemours Children’s Health discussed next steps in the field to fully enable the benefits of gene therapy.
George Tachas, PhD, on Tackling DMD Treatment From Multiple Angles
The lead scientist at Percheron Therapeutics discussed research on antisense oligonucleotide therapies in mouse models of DMD.
David Suhy, PhD, on Using DNA Nanoplasmids to Detect Cancer
The cofounder and chief scientific officer at Earli discussed the company’s unique approach to cancer diagnosis.
Deepak L. Bhatt, MD, MPH, MBA, on Incorporating AI into Genetic Research for Cardiovascular Disease
The director of the Mount Sinai Fuster Heart Hospital discussed how artificial intelligence complements data-driven research.
Jeffrey Chamberlain, PhD, on Helping Progress Cell and Gene Therapy Development
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed the role the ASGCT plays in the field.
Jonathan W. Weinsaft, MD, on Integrating Genetic Research into Cardiovascular Medicine
The chief of cardiology at Weill Cornell Medical College discussed the center’s increased focus on genetic research.
Jacques Galipeau, MD, on Highlights from ISCT 2024’s Presidential Plenary
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared highlights from the discussion.
Vanee Pho, PhD, on Detecting Translocations in Gene Edited Cells
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
Michael Wang, MD, on Liso-Cel's Approval in Mantle Cell Lymphoma
The professor in the Department of Lymphoma/Myeloma at MD Anderson Cancer Center discussed the advantages the newly approved CAR-T has for older, frailer patients.
Robert J. Hopkin, MD, on Looking Deeper into Fabry Disease Biology
The associate professor of clinical pediatrics at Cincinnati Children's discussed further investigations into novel therapies and new research.
Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations
The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed a sub analysis of the HOPE-B trial.
Rawan Faramand, MD, on Evaluating Orca-T in Patients With Acute Myeloid Leukemia
The assistant professor at Moffit Cancer Center discussed the subgroup analysis she presented at ASCO’s 2024 Annual Meeting.
Marcus Conant, MD, on More Research Needed With HIV Therapies
The Chief Medical Officer of Addimmune discussed the unmet need with HIV and research the company is conducting.
Manali Kamdar, MD, on Liso-Cel's Continued Efficacy in Second-Line LBCL at 3-Year Follow-up
The associate professor of medicine at University of Colorado discussed updated follow-up data from the phase 3 TRANSFORM trial presented at the 2024 ASCO meeting.
Omid Hamid, MD, on Clinic Experience With TIL vs CAR-T Therapy Administration
The professor from Cedars Sinai also discussed additional investigations evaluating lifileucel for other advanced subtypes of melanoma.
Jeffrey Chamberlain, PhD, on Continuing the Marathon of Muscular Dystrophy Research
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington shared his outlook on the trajectory of research in the field.
N. Nora Bennani, MD, on Diving Deeper Into T-Cell Lymphomas
The assistant professor at Mayo Clinic School of Medicine shared her outlook and predictions on research with T-cell lymphomas.
Anjali Pradhan, MS, on Using Single Cell Sequencing for Gene Editing Quality Control
Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic Leukodystrophy
The deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.
Brian Kim, MBA, on the Importance of Single Cell Analysis to Cell and Gene Therapy
The CEO of Mission Bio discussed the company’s Tapestri platform for single cell sequencing.
Xandra Breakefield, PhD, on Trying New Approaches to AAV Therapy for Glioblastoma
The investigator of neurology at Mass General Research Institute discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.
Nathan Yozwiak, PhD, on Researching AAV Gene Therapy Delivery to the Brain
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.
Salvador Rico, MD, PhD, on Informing Clinical Trials With Natural History Studies in Dravet
The chief medical officer of Encoded Therapeutics discussed the company’s strategy with gene therapy development for Dravet syndrome.
Zheng-Yi Chen, DPhil, on International Collaboration on Clinical Trials
The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.
