Cell and Gene Therapy Today—February 12, 2026

In Today’s Newsletter

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❌ FDA CRL for RGX-121 in Hunter syndrome (Regenxbio) [1] [US • 09 Feb 2026]

https://www.fiercebiotech.com/biotech/fda-rejects-regenxbio-hunter-syndrome-gene-therapy-recently-hit-hold-unrelated-brain-tumor
Context: PDUFA date moved to 08 Feb 2026 from 09 Nov 2025; separate FDA clinical holds noted after an RGX-111 brain tumor case, but not cited as CRL reasons.
Key point: FDA rejected RGX-121, raising concerns on population definition, natural-history control, and heparan sulfate as surrogate endpoint (accelerated approval path).
Implication: May influence prescriber choice and payer reviews pending full data.

🤝 Lilly to acquire Orna Therapeutics for in vivo CAR-T in autoimmune disease [2] [US • 09 Feb 2026]

https://investor.lilly.com/news-releases/news-release-details/lilly-acquire-orna-therapeutics-advance-cell-therapies
Context: Goal is durable in vivo cell engineering to reduce ex vivo CAR-T complexity, cost, and logistics.
Key point: Definitive deal for up to $2.4B, centered on circular RNA + LNP platform; lead ORN-252 targets CD19 in vivo for B-cell-driven autoimmune diseases (clinical-ready).
Implication: Signals pipeline investment and modality expansion.

⏸ uniQure pauses AMT-191 dosing in Fabry after DLTs [3] [06 Feb 2026]

https://www.fiercebiotech.com/biotech/uniqure-pauses-fabry-gene-therapy-dosing-2-groups-after-toxicities-emerge
Context: Phase 1/2a, 11 patients across 3 doses; corticosteroids effective; lower doses without serious AEs; efficacy signals include ↑α-Gal A and ERT discontinuations in some.
Key point: Two asymptomatic Grade 3 liver enzyme elevations at a mid dose triggered a protocol-defined pause of mid- and high-dose cohorts.
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 UX111 shows long-term benefit in Sanfilippo A (Ultragenyx) [4] [09 Feb 2026]

https://www.biospace.com/drug-development/ultragenyxs-gene-therapy-shows-long-term-efficacy-in-two-sanfilippo-studies
Context: FDA declined approval in Jul 2025 for CMC reasons; resubmitted 30 Jan 2026 with a 6-month review expectation.
Key point: Across two studies (n=33; follow-up to 8.5 years), younger patients had significant Bayley-III cognitive gains; older patients retained key functions; CSF HS reduced.
Implication: May influence prescriber choice and payer reviews pending full data.

👁 First patient dosed in REVISE LHON study (GenSight, 15-20 Hospital) [5] [EU • 10 Feb 2026]

https://www.biospace.com/press-releases/the-15-20-national-hospital-and-gensight-biologics-announce-the-treatment-of-the-first-patient-in-the-gs010-lumevoq-revise-study
Context: 15-20 Hospital is the sole EU site for REVISE and AAC; GenSight aims to start a new Phase 3 by year-end.
Key point: Open-label, single-center, 14-patient, dose-ranging study of GS010/LUMEVOQ for ND4-LHON began in Paris; AAC named-patient program authorized in France.
Implication: May expand screening, initiation, and follow-up at scale.

🧪 Bruker launches iNTApharma for native-state nanoparticle QC [6] [US • 10 Feb 2026]

https://www.biospace.com/press-releases/bruker-introduces-intapharma-a-label-free-platform-for-nanoparticle-characterization-in-mrna-drug-and-gene-therapy-development-and-qc
Context: Introduced at SLAS 2026; early-access placements H1 2026, broader availability H2 2026; supports well-plate, automated workflows.
Key point: Label-free iSCAT-based platform measures size and concentration of 50–300 nm bioparticles, targeting LNPs, AAV/AdV, lentiviral vectors, and EVs.
Implication: Signals pipeline investment and modality expansion.

🧩 Kincell Bio–RegCell partnership on epigenetically reprogrammed Tregs [7] [US • 10 Feb 2026]

https://www.businesswire.com/news/home/20260210768570/en/Kincell-Bio-Announces-Partnership-with-RegCell-to-Advance-Treg-Cell-Therapies-into-Clinical-Trials-for-Autoimmune-Diseases
Context: Platform reprograms CD4+ T cells to lineage-stable Tregs without viral DNA or gene editing, aiming to simplify manufacturing.
Key point: Kincell to provide CMC and GMP manufacturing to advance RegCell’s antigen-matched, non-edited Tregs toward IND and trials.
Implication: Signals pipeline investment and modality expansion.

🧬 Gyala starts first-in-human CD84 CAR-T in AML and T-ALL [8] [EU • 10 Feb 2026]

https://www.prnewswire.com/news-releases/gyala-therapeutics-initiates-phase-iiia-clinical-trial-of-a-car-t-cell-therapy-in-acute-leukemias-with-limited-treatment-options-302681392.html
Context: Target CD84; sites in Valencia and Barcelona; €3.7M public funding; no approved CAR-Ts for these indications.
Key point: Phase I/IIa single-arm study of GYA01 begins in Spain for R/R AML or T-ALL, with dose escalation then expansion.
Implication: Signals pipeline investment and modality expansion.

Why It Matters

FDA’s CRL for RGX-121 underscores rising scrutiny of surrogate endpoints and natural-history controls in neurogene therapy.
In vivo CAR-T could broaden autoimmune treatment access by bypassing ex vivo manufacturing.
Safety management in AAV programs remains central, with dosing strategy and steroid protocols under focus.
Long-term follow-up data are increasingly pivotal for neurocognitive gene therapies and regulatory reassessment.
EU-based access routes and investigator-site leadership can maintain momentum while pivotal plans mature.

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FAQ

What did the FDA cite in rejecting Regenxbio’s RGX-121?

Trial population definition, reliance on a natural-history control, and use of heparan sulfate as a surrogate endpoint in the accelerated approval filing [1]. The FDA noted clinical holds elsewhere but did not cite them as CRL reasons.

What exactly is Lilly acquiring from Orna?

A circular RNA + LNP platform for in vivo cell engineering, led by ORN-252 (CD19 in vivo CAR-T) for B-cell autoimmune disease; deal value up to $2.4B, contingent on milestones [2].

Why did uniQure pause dosing in Fabry?

Two mid-dose patients had asymptomatic Grade 3 liver enzyme elevations, triggering a protocol pause of mid- and high-dose groups while assessment continues; steroid response was observed [3].

How durable are UX111 effects in Sanfilippo A?

Across two studies with follow-up up to 8.5 years, younger children showed significant Bayley-III gains and CSF HS reductions, while older patients retained key functions; the BLA was resubmitted on 30 Jan 2026 [4].

What is REVISE and how does it relate to access in France?

REVISE is a 14-patient, open-label dose-ranging study of GS010/LUMEVOQ in ND4-LHON at 15-20 Hospital; ANSM also authorized named-patient AAC, with requests under review [5].

What is Bruker’s iNTApharma used for?

Label-free, native-state size and concentration measurements for 50–300 nm bioparticles, supporting LNP and viral-vector development and QC, with early-access in H1 2026 [6].

Entities / Keywords

Regenxbio; RGX-121; RGX-111; Hunter syndrome; MPS II; heparan sulfate; FDA CRL
Eli Lilly; Orna Therapeutics; ORN-252; circular RNA; LNP; in vivo CAR-T
uniQure; AMT-191; Fabry disease; AAV5; α-galactosidase A; ERT
Ultragenyx; UX111; Sanfilippo A; Bayley-III; CSF HS; WORLDSymposium
GenSight Biologics; LUMEVOQ; GS010; ND4-LHON; 15-20 Hospital; ANSM; AAC
Bruker; iNTApharma; iSCAT; nanoparticle tracking; LNP; AAV; QC
Kincell Bio; RegCell; Tregs; epigenetic reprogramming; CMC; GMP
Gyala Therapeutics; GYA01; CD84; AML; T-ALL; AEMPS