Jennifer Taylor-Cousar, MD, MSCS, on Early Signals of Efficacy With Cystic Fibrosis Gene Therapy
The professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.
Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.
Paul Y. Song, MD, on Taking a Holistic Approach to Treating Parkinson Disease
The chairman and chief executive officer of NKGen discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.
Manali Kamdar, MD, on Evaluating Liso-Cel in Mantle Cell Lymphoma by Lines of Therapy, Prior BTKi
The associate professor of medicine at University of Colorado discussed a post hoc analysis study on subgroup analyses of liso-cel response.
Ignacio Mata, PhD, on the Research Needed to Bring Gene Therapy to PD
The associate professor of neurology at the Cleveland Clinic Lerner Institute discussed the challenges that will need to be overcome to apply gene therapy to a complex neurological disorder like PD.
Subhash Tripathi, PhD, on Developing Safe, Specific Engineered Treg Cell Therapy
The senior researcher at Seattle Children’s discussed advantages of engineered Tregs compared with natural Tregs.
Ignacio Mata, PhD, on the Challenges of Developing New Therapies in Parkinson Disease
The associate professor of neurology at the Cleveland Clinic Lerner Institute pointed out that genetic forms of PD may be the best place to start for new therapeutic research.
Mark Walters, MD, on the Future of Sickle Cell Disease’s Landscape of Care
The professor in residence for pediatrics at the Sickle Cell Center of Excellence at the University of California discussed gene therapy’s ongoing transformation of the treatment landscape.
Jacques Galipeau, MD, on International Insights Into Cell and Gene Therapy Development
The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison emphasized similar variables in the way of therapy uptake regardless of country.
Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMD
The cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.
Manali Kamdar, MD, on Bringing Liso-Cel to Earlier Lines of Treatment
The associate professor of medicine at University of Colorado discussed data in the primary relapsed LBCL setting.
Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson Disease
The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
Omid Hamid, MD, on Assessing TIL Combination Therapies, Expanding Past Melanoma
The professor from Cedars Sinai discussed further research being conducted in the field after lifileucel’s approval.
Nathan Yozwiak, PhD, on Collaboration for Cell and Gene Therapy Development
The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed work the center is engaged in with cell and gene therapy.
Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet Syndrome
The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.
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.
