Videos

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed the statistical findings she presented at the 2024 WORLDSymposium.

The chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed challenges on the horizon in this rapidly emerging field.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the importance of rigorous clinical trial design for determining whether CAR-T will truly be of benefit in autoimmune disease.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham pointed out access to a sickle cell specialist as one such priority.

The chief medical officer of Encoded Therapeutics discussed ETX101 gene therapy and other initiatives in the company’s overall Dravet program.

The Associate Professor of Medicine at Harvard Medical School shared her outlook on the trajectory of CAR T-cell therapy for treating solid tumors.

The executive director of global clinical development at Ultragenyx Pharmaceutical discussed UX111, the company’s investigational gene therapy for MPSIIIA.

The clinical research director of the UCSF Multiple Sclerosis Center discussed the current standard of care for B-cell driven autoimmune diseases and how CAR-T could address unmet needs.

The associate professor of otolaryngology from Harvard Medical School also shared other big takeaways from the landmark trial for the gene therapy field.

The clinical assistant professor at Stanford Medicine discussed potential applications for machine learning in analyzing data in medicine.

The chief scientific officer at Omega Therapeutics discussed how the MYCHELANGEO-1 trial may validate the company’s OEC platform.

The chief scientific officer of CureDuchenne discussed how nonviral approaches may address issues including gene size and redosability.

The associate professor of dermatology at Stanford University discussed the potential impact of Vyjuvek’s approval in the derm field.

The assistant professor at MD Anderson Cancer Center discussed a trial she is leading at MD Anderson with NK cells and cetuximab in colorectal cancer.

The professor in residence of pediatrics at University of California San Francisco discussed trends in research for SCD gene therapy.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed work the National Alliance of Sickle Cell Centers is conducting.

The Associate Professor of Medicine at Harvard Medical School discussed CAR T’s journey to treating solid tumors.

The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.

The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed how a personalized gene editing approach may help patients avoid development of FVIII inhibitors.

The chairman, chief executive officer, and cofounder of Ocugen discussed programs including OCU400 and OCU410.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed the organization’s research imperatives of 2024.

The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.

The associate professor of otolaryngology from Harvard Medical School discussed investigating gene therapy for DFNB9 deafness.

The internal medicine resident physician at University of Kansas Medical Center also discussed highlights from the ASH 2023 meeting.

The chief medical advisor of the Muscular Dystrophy Association discussed research he is working on with the goal of increasing eligibility for gene therapies and opening the door to redosing.

The clinical assistant professor at Stanford Medicine also shared his excitement on the recent approvals of lovo-cel and exa-cel.

The chief medical advisor of the Muscular Dystrophy Association discussed what seasoned clinicians and newcomers to the field alike can look forward to at the 2024 MDA Meeting.

The associate professor at Fred Hutch Cancer Center discussed trends he observed in the field in 2023 and at ASH 2023.