News

PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.

FDA granted accelerated approval to pariglasgene brecaparvovec-opnr, a one-time liver-directed AAV8 gene therapy, based on a 31% reduction in cornstarch intake versus placebo.

FDA grants Kyverna's miv-cel RMAT designation in non-active secondary progressive MS, based on Phase 1 data showing CAR T expansion and B-cell reconstitution.

Johnson & Johnson secures an exclusive option to acquire Sail Biomedicines for $2.58 billion, backing the biotech's in vivo CAR T platform for immune-mediated disease.

FDA advisory committee votes 9-3 against deramiocel's effectiveness for Duchenne cardiomyopathy; PDUFA decision due August 22, 2026.

Oak Hill Bio announced dosing of the first participant in BEACON, a pivotal phase 3 trial evaluating rugonersen, an antisense oligonucleotide originally developed by Roche, for Angelman syndrome.

The Phase 3 ACTiVION-II trial found that a single intra-articular injection of TG-C did not improve pain or function compared with placebo at 12 months in patients with knee osteoarthritis, missing both co-primary endpoints.

A single systemic dose of an immune-shielded lentiviral vector delivering the MMUT gene produced lasting metabolic correction in a mouse model of methylmalonic acidemia, according to newly published preclinical data.

The milestone advances the commercial-ready cryopreserved formulation of the autologous iPSC-derived therapy into broader clinical experience as Aspen prepares for phase 3 initiation.

The FDA approved Tregzi as the first allogeneic regulatory T cell-based immunotherapy based on Precision-T phase 3 data showing a significant 1-year cGVHD-free survival rate of 78% vs 38% with conventional alloHSC.

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.

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.

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.

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.