This week’s Gene & Cell Therapy news delivers the latest updates, developments, and progress from leading biopharma companies.
In Today’s Newsletter
🧬 Intellia pauses phase 3 MAGNITUDE dosing for nex-z in ATTR [1] [27 Oct 2025]
https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-provides-update-magnitude-clinical-trials
Context: Temporary pause in MAGNITUDE (ATTR-CM) and MAGNITUDE-2 (ATTR-PN) after Grade 4 LFTs and hyperbilirubinemia in one patient dosed Sep 30, 2025; >650 enrolled in MAGNITUDE, 47 in MAGNITUDE-2.
Key point: Protocol-defined criteria triggered pause; patient hospitalized and under care while company consults regulators and experts.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
👁️ Lilly to acquire Adverum, adding phase 3 Ixo-vec for wAMD [2] [24 Oct 2025]
https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-adverum-biotechnologies
Context: Tender offer at $3.56 cash plus CVR up to $8.91 per share; Adverum receives up to $65M secured loan pre-close.
Key point: Ixo-vec is intravitreal, one-time AAV gene therapy in ARTEMIS phase 3, with US RMAT and FDA Fast Track.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🧪 REQORSA preclinical poster in ALK+ NSCLC (EORTC-NCI-AACR) [3] [US • 23 Oct 2025]
https://www.prnewswire.com/news-releases/genprex-collaborators-to-present-positive-preclinical-data-on-the-use-of-reqorsa-gene-therapy-for-the-treatment-of-lung-cancer-at-the-2025-aacr-nci-erotc-international-conference-on-molecular-targets-and-cancer-therapeutics-302592389.html
Context: University of Michigan collaborators present TUSC2 plasmid nanoparticle data.
Key point: In models, QO alone or with alectinib reduced tumor size versus control; supports potential future clinical path (preclinical, not randomized clinical).
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
🧫 NLS/Kadimastem–TargetGene collaboration on gene-edited cell therapies [4] [27 Oct 2025]
https://www.prnewswire.com/news-releases/nls-pharmaceutics-and-kadimastem-announce-strategic-collaboration-between-kadimastem-and-targetgene-to-advance-next-generation-gene-edited-cell-therapies-302595227.html
Context: MOU to pair Kadimastem stem-cell platforms (AstroRx, IsletRx) with TargetGene editing; to continue under NewCelX post-merger.
Key point: Aims for multiplex, DNA-guided editing to enhance efficacy and safety in ALS and diabetes cell therapies.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🛡️ BMS CD19 NEX-T CAR-T shows early autoimmune signals at ACR [5] [25 Oct 2025]
https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-Encouraging-Data-from-Phase-1-Breakfree-1-Study-of-CD19-NEX-T-CAR-T-Cell-Therapy-in-Three-Chronic-Autoimmune-Diseases-at-ACR-Convergence-2025/default.aspx
Context: Phase 1 Breakfree-1 in SSc, SLE, IIM, n=71 treated; uses liso-cel construct with next-gen manufacturing.
Key point: 94% of evaluable patients off chronic IST at analysis; safety acceptable with mostly low-grade CRS and limited ICANS.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧘 CAR-Tregs: Quell preclinical QEL-005 data ahead of CTA [6] [25 Oct 2025]
https://www.globenewswire.com/news-release/2025/10/25/3174167/0/en/Quell-Therapeutics-to-Unveil-Promising-Data-for-QEL-005-a-Novel-CAR-Treg-Cell-Therapy-for-Complex-Autoimmune-Diseases-at-ACR-Convergence-2025.html
Context: CD19-directed CAR-Tregs with Foxp3 Phenotype Lock; RA and SSc planned.
Key point: Preclinical data show non-cytolytic, multi-lineage immunomodulation; CTA filings targeted Q4 2025, CHILL study H1 2026.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🗂️ Regeneron ends bbT369 CAR-T for lymphoma [7] [24 Oct 2025]
https://www.biospace.com/business/regeneron-cans-2seventy-acquired-car-t-candidate-for-lymphoma-in-strategic-move
Context: bbT369 came via 2seventy pipeline purchase; was early-stage, pre-phase 2.
Key point: Discontinuation framed as strategic; broader cell therapy portfolio continues.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
❤️ Medera doses first patient in phase 2 MUSIC-HFrEF (SRD-001) [8] [27 Oct 2025]
https://www.medera.bio/medera-biopharm-s-sardocor-showcases-positive-interim-data-from-heref-gene-therapy-phase-1/medera-announces-first-patient-dosed-in-phase-2-portion-of-music-hfref-phase-trial
Context: Phase 2 randomized, double-blind, placebo-controlled; n=50 planned; intracoronary infusion method.
Key point: First phase 2 patient treated Sep 5, 2025 following DSMB go-ahead from phase 1b.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧠 BridgeBio LGMD2I/R9 phase 3 interim success reiterated [9] [27 Oct 2025]
https://www.biospace.com/drug-development/bridgebio-pops-on-late-stage-limb-girdle-muscular-dystrophy-data
Context: FORTIFY met primary and secondary endpoints; BBP-418 oral small molecule.
Key point: Nearly doubled αDG at 3 months, 82% CK drop at 12 months, plus functional gains; NDA expected H1 2026.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧾 BioMarin to divest Roctavian after weak sales [10] [US, EU • 28 Oct 2025]
https://www.fiercepharma.com/pharma/biomarin-resigned-ending-journey-hemophilia-gene-therapy-roctavian-seeking-divestiture-amid
Context: Q3 sales $3M; narrowed to US, Germany, Italy earlier; plant idled pending demand.
Key point: Company pursuing out-licensing to maintain access while exiting commercial ownership.
Implication: Regulatory/generics: Introduces competition that may affect pricing and formulary access.
🩺 Gamida Cell: omidubicel in SAA shows fast engraftment; PDUFA Dec 10, 2025 [11] [28 Oct 2025]
https://www.biospace.com/press-releases/gamida-cell-presents-positive-initial-results-on-treating-severe-aplastic-anemia-saa-with-omidubicel
Context: Open-label single-center NHLBI study, n=14; reduced-intensity conditioning.
Key point: 92.9% rapid neutrophil recovery, median 7 days; no Grade III–IV acute or chronic GvHD reported.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.
🧯 Zag Bio debuts with $80M series A for thymus-targeted Tregs [12] [28 Oct 2025]
https://www.fiercebiotech.com/biotech/3-biopharma-heavies-contribute-80m-series-round-tregs-specialist-zag-bio
Context: Investors include AbbVie, Regeneron, Sanofi; founded by Polaris and T1D Fund.
Key point: Platform delivers antigens to thymus to expand antigen-specific Tregs and delete Teff; T1D candidate aims for phase 1 next year.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
Why it matters
- Cell and gene therapy momentum persists despite setbacks, with capital, M&A, and diversified modalities progressing in ophthalmology, cardiology, neuromuscular, and autoimmune.
- Autoimmunity shift: multiple approaches emerge, from B-cell–depleting CAR-T to non-cytolytic CAR-Tregs and thymus-targeted Tregs.
- Regulatory catalysts continue to shape portfolios, from pauses and divestitures to priority reviews and near-term filings.
- Clinical design rigor increases, with placebo-controlled phase 2 transitions and functional endpoints alongside biomarkers.
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FAQ
What triggered Intellia’s pause in MAGNITUDE?
A MAGNITUDE patient experienced Grade 4 transaminase elevation and increased bilirubin, meeting protocol pausing criteria. Dosing and screening are temporarily paused while the event is investigated [1].
What exactly is Lilly buying from Adverum?
Ixo-vec, an intravitreal, one-time AAV gene therapy in phase 3 for wet AMD, plus a CVR structure tied to approval and sales milestones; Adverum also gets up to $65M in secured financing pre-close [2].
How strong is BMS’s Breakfree-1 signal across autoimmune cohorts?
Early phase 1 data show robust CAR-T expansion, B-cell depletion, and 94% of evaluable patients off chronic IST at analysis, with mostly low-grade CRS and manageable ICANS across SSc, SLE, and IIM cohorts [5].
What distinguishes Quell’s CAR-Tregs from classic CD19 CAR-T?
QEL-005 is designed for non-cytolytic, multi-lineage immunomodulation via CAR-Tregs with a Foxp3 lock, aiming for safety and durable control rather than B-cell depletion alone (preclinical) [6].
Why is BioMarin divesting Roctavian?
Persistent commercial underperformance and narrowed market focus led BioMarin to seek out-licensing to maintain access while reallocating resources to other assets [10].
What is Zag Bio’s thymus-targeting approach?
It delivers antigens to the thymus to restore central tolerance, expanding antigen-specific Tregs and depleting Teff, with an initial Type 1 diabetes program planned for phase 1 next year [12].
Entities / Keywords
Intellia Therapeutics; nex-z; MAGNITUDE; Eli Lilly; Adverum; Ixo-vec; wAMD; Genprex; REQORSA; TUSC2; NLS Pharmaceutics; Kadimastem; TargetGene; NewCelX; Bristol Myers Squibb; CD19 NEX-T; Breakfree-1; systemic sclerosis; SLE; IIM; Quell Therapeutics; QEL-005; CAR-Tregs; Regeneron; bbT369; Medera; SRD-001; MUSIC-HFrEF; BridgeBio; BBP-418; LGMD2I/R9; BioMarin; Roctavian; Gamida Cell; omidubicel; SAA; Zag Bio; Tregs; thymus-targeted tolerance.
References
- https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-provides-update-magnitude-clinical-trials
- https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-adverum-biotechnologies
- https://www.prnewswire.com/news-releases/genprex-collaborators-to-present-positive-preclinical-data-on-the-use-of-reqorsa-gene-therapy-for-the-treatment-of-lung-cancer-at-the-2025-aacr-nci-erotc-international-conference-on-molecular-targets-and-cancer-therapeutics-302592389.html
- https://www.prnewswire.com/news-releases/nls-pharmaceutics-and-kadimastem-announce-strategic-collaboration-between-kadimastem-and-targetgene-to-advance-next-generation-gene-edited-cell-therapies-302595227.html
- https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-Encouraging-Data-from-Phase-1-Breakfree-1-Study-of-CD19-NEX-T-CAR-T-Cell-Therapy-in-Three-Chronic-Autoimmune-Diseases-at-ACR-Convergence-2025/default.aspx
- https://www.globenewswire.com/news-release/2025/10/25/3174167/0/en/Quell-Therapeutics-to-Unveil-Promising-Data-for-QEL-005-a-Novel-CAR-Treg-Cell-Therapy-for-Complex-Autoimmune-Diseases-at-ACR-Convergence-2025.html
- https://www.biospace.com/business/regeneron-cans-2seventy-acquired-car-t-candidate-for-lymphoma-in-strategic-move
- https://www.medera.bio/medera-biopharm-s-sardocor-showcases-positive-interim-data-from-heref-gene-therapy-phase-1/medera-announces-first-patient-dosed-in-phase-2-portion-of-music-hfref-phase-trial
- https://www.biospace.com/drug-development/bridgebio-pops-on-late-stage-limb-girdle-muscular-dystrophy-data
- https://www.fiercepharma.com/pharma/biomarin-resigned-ending-journey-hemophilia-gene-therapy-roctavian-seeking-divestiture-amid
- https://www.biospace.com/press-releases/gamida-cell-presents-positive-initial-results-on-treating-severe-aplastic-anemia-saa-with-omidubicel
- https://www.fiercebiotech.com/biotech/3-biopharma-heavies-contribute-80m-series-round-tregs-specialist-zag-bio