This week’s Cell and Gene therapy update covers major regulatory approvals, global clinical trial momentum, funding rounds, platform manufacturing expansions, and translational gene-editing advances—plus more developments across the cell and gene therapy landscape.
In Today’s Newsletter
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🧬 FDA approves Novartis Itvisma for older SMA patients [1] [US • 24 Nov 2025]
Context: Based on Phase 3 STEER and Phase 3b STRENGTH, 52-week follow-up; intrathecal administration.
Key point: Single-dose SMN1 gene replacement now approved for SMA patients aged ≥2, with motor function improvement and stabilization reported; safety consistent with prior data.
Implication: May influence prescriber choice and payer reviews pending full data.
🧠 First child dosed with Hunter syndrome gene therapy shows recovery [2] [UK • 24 Nov 2025]
Context: Royal Manchester Children’s Hospital; child dosed Dec 2024, 1-year follow-up; observational media report.
Key point: Clinicians report rapid improvement in development post investigational therapy; trial ongoing with two-year observation.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
💪 FDA okays sirolimus regimen study with Elevidys in non-ambulatory DMD [3] [US • 25 Nov 2025]
Context: Endeavor Cohort 8 to enroll ~25 non-ambulatory DMD patients; endpoints include acute liver injury incidence and dystrophin expression.
Key point: FDA cleared enhanced immunosuppression with sirolimus pre- and post-Elevidys infusion to address liver risk in AAV gene therapy.
Implication: May influence prescriber choice and payer reviews pending full data.
🌳 Aspen Neuroscience raises $115M to advance Parkinson’s cell therapy [4] [20 Nov 2025]
Context: Phase 1/2 autologous iPSC-derived neuron transplant (ANPD001); 14 patients dosed; Kite joins board.
Key point: Funding supports enriched trial, manufacturing scale-up, and potential Phase 3 planning.
Implication: Signals pipeline investment and modality expansion.
🧫 Non-viral long ssDNA donors enable efficient HSPC knock-ins [5] [21 Nov 2025]
Context: Nature Communications paper by Moligo and Cellectis on enzymatic long ssDNA, including circular formats.
Key point: Circular ssDNA donors showed 3–5× higher knock-in efficiency vs linear in HSPCs and better engraftment retention vs AAV6 in murine models.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧪 Aspen closes $115M Series C to accelerate personalized cell therapies [6] [20 Nov 2025]
Context: Co-led by OrbiMed, ARCH, Frazier, Revelation; Kite’s EVP joins board.
Key point: Proceeds fund ANPD001 clinical work, manufacturing scale-up, and pipeline for neurological indications.
Implication: Signals pipeline investment and modality expansion.
👁️ AGC Biologics to manufacture AAVantgarde dual-vector ocular AAVs [7] [EU • 21 Nov 2025]
Context: Programs AAVB-039 (Stargardt, Ph1/2 US/UK/EU) and AAVB-081 (Usher 1B RP, Ph1/2).
Key point: CDMO pact uses BravoAAV platform to produce dual-vector AAVs addressing genes exceeding AAV capacity.
Implication: Signals pipeline investment and modality expansion.
🩸 Nature Medicine: FIX-Padua AAV gene therapy Ph1/2 and Ph3 in China [8] [China • 20 Nov 2025]
https://www.nature.com/articles/s41591-025-04012-y
Context: BBM-H901 at 5×1012 vg/kg; Ph1/2 n=6 safety; Ph3 n=26 efficacy over 52 weeks.
Key point: Ph3 met primary endpoint with mean ABR 0.60 (95% CI 0.18–1.99) and mean FIX:C 41.9 IU/dl at week 52; transaminitis most common AE.
Implication: May influence prescriber choice and payer reviews pending full data.
🧯 GEMMABio gets Brazil CTA for SMA1 gene therapy GB221 [9] [Brazil • 24 Nov 2025]
Context: ANVISA safe-to-proceed; ICM delivery of AAVhu68-SMN1; first dosing expected in coming months.
Key point: Preclinical data showed motor neuron transduction with reduced liver exposure in NHPs; Brazil sites planned and tech transfer via Fiocruz.
Implication: May expand screening, initiation, and follow-up at scale.
🫁 Genprex publishes Acclaim-1 Phase 1 lung cancer gene therapy data [10] [US • 24 Nov 2025]
Context: REQORSA (quaratusugene ozeplasmid) + osimertinib in EGFR-mutant NSCLC post-osimertinib; n=12 dose escalation.
Key point: No DLTs; RP2D 0.12 mg/kg; prolonged disease control in select patients including one ongoing partial response beyond 3 years per report.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 EC approves Breyanzi for relapsed or refractory mantle cell lymphoma [11] [EU • 24 Nov 2025]
Context: Based on TRANSCEND NHL 001 MCL cohort; third-line plus setting.
Key point: ORR 82.7% and CR 71.6% with sustained responses; safety consistent with known profile.
Implication: Introduces competition that may affect pricing and formulary access.
🇬🇧 NICE recommends AUCATZYL (obe-cel) for adult r/r B-ALL [12] [UK • 25 Nov 2025]
Context: Draft guidance for routine NHS commissioning in England and Wales; MHRA conditional MA April 2025 based on FELIX study.
Key point: Autologous CD19 CAR T now recommended for adults ≥26 with r/r B-ALL in the NHS pathway.
Implication: Introduces competition that may affect pricing and formulary access.
Why it matters
- Expansions in SMA and DMD programs reflect active regulator engagement on safety and access in AAV therapies.
- Non-viral editing and dual-vector AAV manufacturing point to platforms tackling payload and safety limits.
- European HTA and regulatory moves expand CAR T access in hematologic malignancies.
- Capital flows into autologous neurology cell therapies continue, supporting late-stage readiness.
- Early oncology gene therapy signals possible combination paths with TKIs.
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FAQ
What did FDA approve for SMA this week?
Itvisma (onasemnogene abeparvovec-brve) gained FDA approval for children ≥2, teens, and adults with SMA, intrathecal single dose, supported by STEER and STRENGTH data [1].
How is Sarepta addressing Elevidys liver safety in non-ambulatory DMD?
FDA cleared an Endeavor Cohort 8 protocol adding sirolimus before and after infusion to mitigate acute liver injury risk in AAV therapy [3].
Which CAR T therapies expanded access in Europe and the UK?
EC approved Breyanzi for r/r mantle cell lymphoma after ≥2 lines including a BTKi [11]. NICE recommended AUCATZYL for adult r/r B-ALL via routine NHS commissioning [12].
What is notable about GEMMABio’s SMA1 program in Brazil?
ANVISA granted CTA for GB221, an AAVhu68 SMN1 therapy delivered via ICM, with tech transfer to Fiocruz and plans to initiate dosing in Brazil [9].
Are there advances in non-viral gene insertion?
Moligo and Cellectis reported that circular long ssDNA donors can drive efficient knock-ins in HSPCs and outperform AAV6 in engraftment retention in murine models [5].
Any pivotal efficacy data in hemophilia B gene therapy?
A Nature Medicine paper reported Ph3 BBM-H901 reduced mean ABR to 0.60 at 52 weeks with mean FIX:C 41.9 IU/dl; transaminitis was the most common AE [8].
Entities / Keywords
Novartis Itvisma onasemnogene abeparvovec-brve; SMA; Sarepta Elevidys sirolimus; Aspen Neuroscience ANPD001; Moligo Technologies long ssDNA CssDNA; Cellectis TALEN; AGC Biologics BravoAAV; AAVantgarde AAVB-039 AAVB-081 dual-vector; BBM-H901 FIX-Padua; GEMMABio GB221 AAVhu68 ICM; Genprex REQORSA quaratusugene ozeplasmid; BMS Breyanzi liso-cel; Autolus AUCATZYL obe-cel; Hunter syndrome MPS II.
References
- https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-itvisma-only-gene-replacement-therapy-children-two-years-and-older-teens-and-adults-spinal-muscular-atrophy-sma
- https://www.geo.tv/latest/635140-first-child-to-receive-gene-therapy-for-hunter-syndrome-shows-stunning-recovery
- https://www.fiercepharma.com/pharma/sarepta-gets-fdas-go-ahead-add-new-immunosuppressive-regimen-elevidys-study-key-population
- https://www.fiercebiotech.com/biotech/aspen-enriches-parkinsons-cell-therapy-trial-kite-backed-115m-series-c
- https://www.prnewswire.com/news-releases/moligo-technologies-and-cellectis-demonstrate-potential-of-enzymatic-single-stranded-dna-for-non-viral-gene-editing-in-nature-communications-302622945.html
- https://www.prnewswire.com/news-releases/aspen-neuroscience-announces-115-million-series-c-financing-to-accelerate-personalized-cell-therapy-programs-302621124.html
- https://www.agcbio.com/news/agc-biologics-to-manufacture-aavantgardes-dual-vector-gene-therapies-for-inherited-retinal-disorders
- https://www.nature.com/articles/s41591-025-04012-y
- https://www.prnewswire.com/news-releases/gemmabio-announces-clinical-trial-authorization-for-new-sma1-gene-therapy-302623436.html
- https://www.prnewswire.com/news-releases/data-from-genprexs-acclaim-1-phase-1-gene-therapy-clinical-trial-published-in-clinical-lung-cancer-302623852.html
- https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Receives-Approval-from-the-European-Commission-to-Expand-Use-of-CAR-T-Cell-Therapy-Breyanzi-for-Relapsed-or-Refractory-Mantle-Cell-Lymphoma/default.aspx
- https://autolus.gcs-web.com/news-releases/news-release-details/nice-recommends-aucatzylr-obecabtagene-autoleucel-treatment