Conference Coverage
13 days ago
Evaluating Miv-Cel for Stiff Person Syndrome20 days ago
Assessing Miv-Cel for Myasthenia Gravisabout 1 month ago
Srikanth Muppidi, MD, on Evaluating Miv-Cel in Myasthenia Gravisabout 2 months ago
Evaluating Gene Therapy BB-301 for OPMD-Related Dysphagiaabout 2 months ago
Angela Lek, PhD, on the FDA's Approval of High-Dose Nusinersen in SMALatest News
May 27, 2026 | News
Around the Helix: Cell and Gene Therapy Company Updates – May 27, 2026
Evaluating Miv-Cel for Stiff Person Syndrome
May 22, 2026
Around the Helix: Cell and Gene Therapy Company Updates – May 20, 2026
May 20, 2026
Phase 3 HOPE-3 Data Reinforce Skeletal and Cardiac Benefits of Deramiocel in Duchenne Muscular Dystrophy
May 18, 2026
Assessing Miv-Cel for Myasthenia Gravis
May 15, 2026
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Shorts
0:32
The FDA’s Approval of High-Dose Nusinersen: What Doctors Should Know
a month ago
0:46
More on misconceptions about CAR-T in neurology
a month ago
1:09
What are some of the misconceptions your colleagues might have about the use of CAR-T in neurology?
a month ago
1:19
The Challenge of T-Cell Therapy Timing
a month ago
0:31
Bad T-cells lead to failed cancer treatments
a month ago
0:42
Why One Target Isn't Enough: The Future of Neuroblastoma Therapy
2 months ago
1:02
What should the medical community know about Deramiocel?
3 months ago
0:48
Where is neuromuscular medicine heading in the future?
3 months ago
0:47
The potential of bringing hemophilia B gene therapy to younger patients
3 months ago
0:41
The need for more data for sickle cell gene therapy
3 months ago
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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.
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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.
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.
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.
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.
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