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

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

Amanda Piquet, MD, FAAN, discussed phase 2 data from a trial evaluating the CAR-T therapy mivocabtagene autoleucel for the treatment of stiff person syndrome.

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

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

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

The pediatric neurologist at Johns Hopkins Medicine discussed the FDA's recent approval of a higher-dose formulation of nusinersen and the evolving SMA treatment landscape.

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

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

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

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.

The therapy will notably be provided for free in the United States.

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

Jerel A. Banks, MD, PhD, 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 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.

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.

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

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.

Published: August 16th 2024 | Updated: August 19th 2024

February 7th 2025