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
The chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
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.
Janice Chen, PhD, on Novel Approaches to Gene Editing
The cofounder and chief technology officer of Mammoth Biosciences discussed the importance of diverse approaches to gene editing to address a variety of indications.
Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing Therapies
The cofounder and chief executive officer of Verve Therapeutics discussed the company’s pipeline.
Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe Disease
The chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.
Leigh Ramos-Platt, MD, on Allowing Access and Ensuring Preparation for Gene Therapies
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her talk on preparing for gene therapy administration.
John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1
The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.
Erika Fullwood Augustine, MD, MS, on Optimizing End Points for Rare Disease Clinical Trials
The associate chief science officer of the Kennedy Krieger Institute discussed an aspect of clinical trial design highly relevant to gene therapy development for rare diseases.
Maria Escolar, MD, on Continued Positive Results With Gene Therapy and UCBT in Krabbe Disease
The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.
Casey Maguire, PhD, on Potential Advantages of Encapsulated AAVs
The associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.
Faraz Ali, MBA, on Trends in Precision Medicine for Cardiovascular Disease
The chief executive officer of Tenaya Therapeutics discussed the growing interest in genomic medicines in cardiology.
Faraz Ali, MBA, on Adding Gene Editing Programs to Tenaya’s Cardiovascular Disease Pipeline
The chief executive officer of Tenaya Therapeutics discussed the company’s decision to supplement the gene transfer programs in its pipeline with gene editing programs.
Evan Weber, PhD, on Improving CAR T-Cell Fitness
The assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.
Faraz Ali, MBA, on Continued Innovation With Cardiovascular-Targeted AAV Gene Therapy
The chief executive officer of Tenaya Therapeutics discussed the company’s research on capsids, promoters, and manufacturing improvements.
Shankar Ramaswamy, MD, on Bringing Gene Therapy to Common Diseases
The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.
Kevin Campbell, PhD, on the Challenges of Gene Therapy Approaches in Advanced Muscular Dystrophy
The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.
Debora Mazzetti, MS, on Multitargeting MicroRNA in Glioblastoma
The research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.
