Cell and Gene Therapy Video Recap—November 27, 2025

This biweekly Cell and Gene Therapy video reacap covers major regulatory approvals, long-term durability data, cell and gene therapy advances, preclinical innovation, and health-technology assessments—plus more.

🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Two Weeks

🗓️ Explore weekly details and sources

Week 13–19 November 2025
Week 20–26 November 2025

📚 See the full Cell and Gene Therapy archive on our research hub page.

Transcript

Welcome to the latest edition of Gene & Cell Therapy Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.

Waskyra moved a step closer to European availability as the EMA’s CHMP issued a positive opinion recommending EU marketing authorisation. It is an ex vivo autologous gene therapy for Wiskott-Aldrich syndrome that will be provided at Ospedale San Raffaele, and a biologics license application is under review at the FDA.

Long-term outcomes from a multicenter cohort for ADA-deficient severe combined immunodeficiency reported sustained survival, immune recovery, and metabolic correction after treatment with autologous lentiviral vector–modified CD34+ cells following busulfan conditioning, with no vector-related safety signals reported.

In metabolic disease, the FDA approved Redemplo, plozasiran, an siRNA therapy targeting APOC3, as an adjunct to diet for familial chylomicronemia syndrome. The approval is based on the Phase 3 PALISADE trial, where the primary and key secondary endpoints were met, representing Arrowhead’s first FDA approval and the first of its class for FCS.

An NCI-led preclinical study published in Cell Reports Medicine found that Eureka’s ARTEMIS CAR T cells outperformed conventional CAR T cells in neuroblastoma models, demonstrating stronger tumor infiltration and control at low antigen density, while clinical trials in other settings continue.

For spinal muscular atrophy, the FDA approved Itvisma, onasemnogene abeparvovec-brve, as a single-dose intrathecal gene replacement therapy for children two years and older, teens, and adults with an SMN1 mutation. The decision was supported by the Phase 3 STEER and Phase 3b STRENGTH studies, which reported improvement and stabilization of motor function.

In Duchenne muscular dystrophy, the FDA cleared the addition of a sirolimus-based immunosuppression regimen to Sarepta’s Elevidys in the Endeavor Cohort 8 study for non-ambulatory patients, with approximately 25 participants. Primary endpoints include the incidence of acute liver injury and the level of dystrophin expression.

In hematologic oncology, the European Commission approved Breyanzi for relapsed or refractory mantle cell lymphoma after at least two prior lines of therapy including a BTK inhibitor, based on the TRANSCEND NHL 001 MCL cohort. Reported outcomes included an overall response rate of 82.7 percent and a complete response rate of 71.6 percent, with safety consistent with the known profile.

Finally, NICE issued draft guidance recommending AUCATZYL, also known as obe-cel, for routine NHS commissioning for adults 26 and older with relapsed or refractory B-ALL in England and Wales, following MHRA conditional authorisation in April 2025 based on the FELIX study.

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Why It Matters

Regulators advanced AAV, siRNA, and CAR T access across US, EU, UK.
Ex vivo LVV programs for PIDs show durable outcomes, supporting curative intent.
Preclinical platforms aim to overcome solid-tumor barriers for engineered T cells.

🗓️ Explore weekly details and sources

Week 13–19 November 2025
Week 20–26 November 2025

📚 See the full Cell and Gene Therapy archive on our research hub page.

FAQ

What did EMA’s CHMP say about Waskyra for WAS?

It issued a positive opinion recommending EU marketing authorisation for the ex vivo therapy developed at SR-Tiget, with US BLA under FDA review [1].

Which SMA gene therapy change happened in the US?

FDA approved Novartis’s intrathecal Itvisma for children ≥2, teens, and adults with SMA and SMN1 mutation, expanding the eligible population beyond infants [5].

How is Sarepta addressing Elevidys safety in DMD?

FDA cleared an Endeavor Cohort 8 protocol adding sirolimus to immunosuppression for non-ambulatory patients to reduce acute liver injury risk [6].

What new CAR T access decisions landed in Europe and the UK?

EC approved Breyanzi for relapsed or refractory mantle cell lymphoma after ≥2 prior lines including a BTKi [7]. NICE recommended AUCATZYL for adult r/r B-ALL in England and Wales [8].

What is Arrowhead’s Redemplo approval?

FDA approved the siRNA plozasiran for familial chylomicronemia syndrome as adjunct to diet, based on the Phase 3 PALISADE study, marking Arrowhead’s first approved medicine [3].

Entities / Keywords

Fondazione Telethon Waskyra; Wiskott-Aldrich syndrome; SR-Tiget; ADA-SCID lentiviral gene therapy; Arrowhead Redemplo plozasiran APOC3; Novartis Itvisma onasemnogene abeparvovec-brve SMA; Sarepta Elevidys sirolimus Endeavor Cohort 8; BMS Breyanzi lisocabtagene maraleucel MCL; Autolus AUCATZYL obe-cel FELIX NHS.