News

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

Investigators discuss interim phase 1/2 INSPIRE DUCHENNE data and the emerging biomarker and safety profile of SGT-003 in Duchenne muscular dystrophy.

Diana Castro, MD, highlights early phase 1b findings on salanersen and its potential to reshape treatment burden in spinal muscular atrophy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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

The new PDUFA action date for the BLA has been set at September 19, 2026.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In episode 9 of ImmunoLogic, Kristopher Bosse, MD, and the cohosts discussed how immunotherapy is reshaping treatment for high-risk neuroblastoma.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The child neurologist at Children’s Hospital of Philadelphia discussed long-term givinostat safety for Duchenne at MDA's 2026 conference.

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

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

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.

Review top news and interview highlights from the week ending February 13, 2026.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

In episode 8 of ImmunoLogic, Julia C. Tchou, MD, PhD, and the cohosts discussed the potential of combining immunotherapy approaches and surgery in the treatment of breast cancer.

ACP says to stop calling doctors “providers”; in the modern age, the term blurs expertise and weakens trust in patient care.