News

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.

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.

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

New phase 2 DAWN data presented at ARVO 2026 showed sustained improvements in visual function and favorable safety with laru-zova in patients with X-linked retinitis pigmentosa.

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.

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.

Intellia has initiated a rolling FDA submission for lonvo-z after phase 3 data showed significant reductions in hereditary angioedema attacks and treatment burden with a single dose.

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.

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.

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.