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Evaluating Miv-Cel for Stiff Person Syndrome3 months ago
Assessing Miv-Cel for Myasthenia GravisLatest News
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The FDA’s Approval of High-Dose Nusinersen: What Doctors Should Know
3 months ago
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More on misconceptions about CAR-T in neurology
4 months ago
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What are some of the misconceptions your colleagues might have about the use of CAR-T in neurology?
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The Challenge of T-Cell Therapy Timing
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Bad T-cells lead to failed cancer treatments
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Why One Target Isn't Enough: The Future of Neuroblastoma Therapy
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What should the medical community know about Deramiocel?
5 months ago
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Where is neuromuscular medicine heading in the future?
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The potential of bringing hemophilia B gene therapy to younger patients
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The need for more data for sickle cell gene therapy
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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.
REGENXBIO will receive a $100 million milestone from AbbVie following the first dosing in NAAVIGATE, a pivotal trial evaluating one-time suprachoroidal gene therapy in nonproliferative diabetic retinopathy without center-involved macular edema.
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.
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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.
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.
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 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.
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.