Cell and Gene Therapy Today—June 18, 2026

This week’s Gene and Cell Therapy update highlights early clinical progress, pivotal trial planning, platform expansion, financing activity, and advancing next-generation gene and cell therapy approaches across multiple indications.

In Today’s Newsletter

Dive deeper

🧬 RZ-001 early activity in recurrent glioblastoma [1] [KR • 15 Jun 2026]

https://www.koreabiomed.com/news/articleView.html?idxno=32031
Context: Rznomics’ RZ-001 is an RNA trans-splicing gene therapy in an ongoing Phase 1/2a trial in recurrent glioblastoma.
Key point: Interim data from 10 treated patients showed no DLTs, no treatment-related Grade 4+ AEs, and early disease-control signals.
Implication: May influence prescriber choice and payer reviews pending full data.

👁️ Opus Genetics expands inherited retinal disease pipeline [2] [US • 16 Jun 2026]

https://www.globenewswire.com/news-release/2026/06/16/3313010/0/en/opus-genetics-highlights-five-gene-therapy-programs-targeting-significant-unmet-need-in-patients-with-inherited-retinal-diseases.html
Context: Opus Genetics highlighted OPGx-RDH12, OPGx-MERTK, OPGx-RHO, OPGx-LCA5, and OPGx-BEST1 in inherited retinal diseases.
Key point: RDH12, MERTK, and RHO programs are expected to enter clinical testing over the next 12–18 months.
Implication: Signals pipeline investment and modality expansion.

🔊 SonoThera raises $125M for ultrasound-mediated genetic medicines [3] [US • 11 Jun 2026]

https://www.biospace.com/business/sonothera-bags-125m-series-b-to-advance-safer-gene-therapies
Context: SonoThera is developing non-viral genetic medicines delivered through ultrasound-mediated technology.
Key point: The $125M Series B will support lead programs in Duchenne muscular dystrophy and ADPKD.
Implication: Signals pipeline investment and modality expansion.

🧪 AviadoBio broadens vMiX RNAi gene silencing rights [4] [UK • 16 Jun 2026]

https://www.biospace.com/press-releases/aviadobio-expands-vmix-precision-gene-silencing-platform-exclusive-license-agreement-with-kings-college-london-beyond-neurological-diseases-to-all-human-therapeutic-areas
Context: AviadoBio and King’s College London expanded an exclusive vMiX RNAi platform license beyond neurological diseases.
Key point: AviadoBio can now apply vMiX across all human therapeutic areas, including cardiovascular, nephrology, and oncology.
Implication: Signals pipeline investment and modality expansion.

❤️ Lexeo finalizes LX2006 pivotal trial design in Friedreich ataxia [5] [US • 15 Jun 2026]

https://ir.lexeotx.com/news-releases/news-release-details/lexeo-therapeutics-announces-regulatory-update-and
Context: LX2006 is a gene therapy candidate for Friedreich ataxia cardiomyopathy.
Key point: SUNRISE-FA 2 will use LVMI as the primary endpoint, with a 6-month topline efficacy analysis.
Implication: May influence prescriber choice and payer reviews pending full data.

🩸 Caribou reports 17.1-month median PFS for vispa-cel [6] [11 Jun 2026]

https://www.fiercebiotech.com/biotech/caribou-boosts-case-shelf-car-t-matches-autologous-drugs-ahead-phase-3
Context: Caribou’s vispa-cel is an allogeneic anti-CD19 CAR-T for second-line large B cell lymphoma.
Key point: A 27-patient Phase 1 dataset showed 17.1-month median PFS after a single optimized CAR-T dose.
Implication: Could streamline initiation and adherence via remote prescribing and logistics.

🧫 Legend reports in vivo CAR-T proof-of-concept for LB2501 [7] [US • 14 Jun 2026]

https://www.globenewswire.com/news-release/2026/06/14/3311435/0/en/legend-biotech-establishes-clinical-proof-of-concept-for-lb2501-a-potential-first-in-class-in-vivo-cd19-cd20-dual-targeting-car-t-in-relapsed-refractory-b-cell-non-hodgkin-lymphoma.html
Context: Legend Biotech’s LB2501 is an in vivo CD19/CD20 dual-targeting CAR-T for relapsed/refractory B-cell NHL.
Key point: In Phase 1, DL2 achieved 100% ORR and 83.3% CR after a single infusion without lymphodepletion.
Implication: May expand screening, initiation, and follow-up at scale.

🧬 CARsgen presents CT0596 and CT1190B allogeneic CAR-T data [8] [China • 14 Jun 2026]

https://www.prnewswire.com/news-releases/carsgen-presents-allogeneic-car-t-cell-products-ct0596-and-ct1190b-at-eha-2026-302798952.html
Context: CARsgen presented CT0596 in R/R multiple myeloma and primary plasma cell leukemia, plus CT1190B in B-cell NHL.
Key point: CT0596 showed responses in 8 evaluable patients, while CT1190B showed 91.7% ORR in 12 evaluable patients.
Implication: May expand screening, initiation, and follow-up at scale.

Why it matters

Next-generation gene delivery remains active across oncology, ophthalmology, neuromuscular, renal, and cardiac indications.
Non-viral and in vivo approaches are gaining attention as companies try to reduce complexity and improve access.
Allogeneic CAR-T data continue to test whether off-the-shelf products can approach autologous outcomes.
Multiple programs are now moving from platform validation toward pivotal or first-in-human milestones.

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FAQ

What is RZ-001 (Rznomics)?

RZ-001 is an RNA trans-splicing gene therapy for recurrent glioblastoma. Interim Phase 1/2a data showed no DLTs and early activity signals [1].

Which Opus Genetics programs are closest to clinical entry?

OPGx-RDH12, OPGx-MERTK, and OPGx-RHO are expected to enter clinical testing over the next 12–18 months [2].

What makes SonoThera’s platform different from AAV gene therapy?

SonoThera uses ultrasound-mediated delivery rather than viral vectors. The company says this may avoid some liver and off-target issues linked to AAV delivery [3].

What changed for AviadoBio’s vMiX platform?

AviadoBio expanded its King’s College London license from neurological diseases to all human therapeutic areas [4].

What is the main endpoint in Lexeo’s SUNRISE-FA 2 trial?

The LX2006 pivotal study will use left ventricular mass index, assessed by cardiac MRI, as the primary endpoint [5].

How are LB2501 and CT1190B different from conventional autologous CAR-T?

LB2501 is designed as an in vivo CAR-T approach without lymphodepletion, while CT1190B is an allogeneic CAR-T product [7,8].

Entities / Keywords

Rznomics, RZ-001, RNA trans-splicing, recurrent glioblastoma, Phase 1/2a
Opus Genetics, OPGx-RDH12, OPGx-MERTK, OPGx-RHO, OPGx-LCA5, OPGx-BEST1, inherited retinal diseases
SonoThera, ultrasound-mediated delivery, non-viral genetic medicines, DMD, ADPKD
AviadoBio, King’s College London, vMiX, RNAi, gene silencing, AVB-406
Lexeo Therapeutics, LX2006, SUNRISE-FA 2, Friedreich ataxia cardiomyopathy, LVMI
Caribou Biosciences, vispacabtagene regedleucel, vispa-cel, allogeneic CAR-T, LBCL
Legend Biotech, LB2501, in vivo CAR-T, CD19/CD20, B-cell NHL
CARsgen Therapeutics, CT0596, CT1190B, BCMA, allogeneic CAR-T, R/R MM, pPCL, B-cell NHL