Latest Conference Coverage

Phase 3 HOPE-3 data further support deramiocel’s potential to preserve both skeletal and cardiac function in Duchenne muscular dystrophy while slowing progression of myocardial fibrosis.

Srikanth Muppidi, MD, spoke about data from the KYSA-6 trial evaluating Kyverna's CAR-T mivocabtagene autoleucel in MG.

New interim ASGCT 2026 findings suggest investigational AAV9 gene therapy GS-100 may improve motor and cognitive outcomes in children with NGLY1 Deficiency, while informing dose optimization and immune management strategies.

New ASGCT 2026 data suggest Encoded Therapeutics’ investigational gene therapy ETX101 may meaningfully reduce seizures and alter developmental trajectory in children with SCN1A-positive Dravet syndrome.

The clinical professor of adult neurology at Stanford University discussed data from a phase 2 study assessing Kyverna Therapeutics’ CAR-T.

The director of the autoimmune neurology program at the University of Colorado discussed data from a phase 2 single-arm registrational trial evaluating Kyverna Therapeutics’ CAR-T.

Jerel A. Banks, MD, PhD, the CEO of Benitec Biopharma, discussed data he presented at MDA's 2026 conference.

The chief research officer of the Muscular Dystrophy Association discussed the context, evidence, and ongoing questions regarding the approval.

The chief research officer of the Muscular Dystrophy Association discussed highlights and themes from the organization's 2026 Clinical and Scientific Conference.

The CEO of Benitec Biopharma discussed data he presented at MDA's 2026 conference.

The president and chief executive officer of the Biotechnology Innovation Organization discussed insights from his keynote address at MDA's 2026 conference.

FDA restarts review of deramiocel for DMD cardiomyopathy after HOPE-3 data submission; the cell therapy treatment eyes August 2026 decision.

Wave Life Sciences’ RNA therapy WVE-N531 generates sustained dystrophin and improves early function in exon 53–skipping Duchenne muscular dystrophy trial.

Robin E. Miller, MD, director of the Sickle Cell Disease Program at Nemours Children’s Hospital, discussed what clinics need in order to start administering gene therapy effectively.

The director of the Sickle Cell Disease Program at Nemours Children’s Hospital discussed what clinics need in order to start administering gene therapy effectively.

Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed the tracks at the upcoming 2026 MDA Meeting.

The chief medical advisor of the Muscular Dystrophy Association spoke about what attendees can expect at the 2026 MDA Meeting, with a focus on this year's tracks.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, also discussed open questions that remain for the future of the hemophilia B gene therapy.

For Glaucoma Awareness Month, CGTLive® interviewed the chair of ophthalmology at the Byers Eye Institute at Stanford University about the potential of cell and gene therapy for the condition.

The research portfolio director at the Muscular Dystrophy Association discussed how ongoing advances in ALS research may inform therapeutic development approaches.

The professor of pediatric hematology/oncology at the University of Michigan Health discussed what clinics need in order to start administering Hemgenix effectively.

The chief research officer of the Muscular Dystrophy Association discussed emerging scientific priorities and community-driven initiatives shaping the organization's 2026 Clinical and Scientific Conference.

We showcased several key moments from our on the ground coverage of the 67th American Society of Hematology Annual Meeting and Exposition.

Colleen Caleshu, MS, CGC, the senior director of research and real world data at Genome Medical, discussed important considerations for genetic counselors thinking about using AI tools in their practice.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, also discussed open questions that remain for the future of the hemophilia B gene therapy.

The genetic counselor at Johns Hopkins Hospital spoke on how cardiac genetics clinics are adopting alternative care models to broaden access to genetic testing and counseling.

The professor of pediatric hematology/oncology at the University of Michigan Health also discussed open questions that remain for the future of the hemophilia B gene therapy.

The chairman of the Division of Pediatric Hematology & Oncology and BMT at Cleveland Clinic Children's discussed learnings from the institutions integration of gene therapy for sickle cell disease.