Cell and Gene Therapy Today—February 19, 2026

In Today’s Newsletter

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❌ Regenxbio’s PDE6A gene therapy fails to show visual improvement in retinitis pigmentosa trial [1] [EU • 13 Feb 2026]

https://www.ophthalmologytimes.com/view/no-significant-visual-gains-seen-with-pde6a-gene-supplementation-in-early-phase-trial
Context: Study involved nine patients with PDE6A-associated retinitis pigmentosa.
Key point: AAV8-based PDE6A gene therapy did not yield significant improvements in visual function over a year in patients with retinitis pigmentosa, and risks such as retinal thinning were observed.
Implication: This failure underscores the challenge in targeting slow-progressing inherited retinal diseases with gene therapies.

🤝 Ultragenyx to cut 10% of workforce following failed trials [2] [US • 13 Feb 2026]

https://www.fiercebiotech.com/biotech/ultragenyx-lays-10-workforce-wake-phase-3-brittle-bone-trial-fails
Context: The company is now restructuring to focus on its core gene therapy programs and aims to be profitable by 2027.
Key point: Ultragenyx laid off 10% of its workforce after two failed trials in brittle bone disease and setbacks in advancing gene therapy UX111 for Sanfilippo syndrome.
Implication: The restructuring may delay the commercial path for certain gene therapies, but future success hinges on UX111 approval and other pipeline advancements.

💉 ExCellThera’s Zemcelpro® receives NUB Status 1 in Germany [3] [EU • 16 Feb 2026]

https://www.prnewswire.com/news-releases/zemcelpro-dorocubicel-receives-nub-status-1-in-germany-302688424.html
Context: The therapy targets blood cancers and has seen growing interest, with 220 hospitals expressing interest for 2026.
Key point: Zemcelpro® (dorocubicel) was granted NUB Status 1 in Germany, enabling hospitals to apply for reimbursement while further evaluations continue.
Implication: This status opens the door to broader access for patients in Germany, positioning Zemcelpro® for future growth in the European market.

🛑 Boehringer Ingelheim halts inhaled gene therapy after cystic fibrosis trial failure [4] [EU • 17 Feb 2026]

https://www.fiercebiotech.com/biotech/boehringer-axes-inhaled-gene-therapy-after-viewing-cystic-fibrosis-data
Context: The Phase 1/2 trial showed no clinical benefit, despite an acceptable safety profile.
Key point: Boehringer Ingelheim discontinued the Lenticlair 1 trial after its lentiviral inhaled gene therapy BI 3720931 failed to show efficacy in cystic fibrosis.
Implication: The decision could affect the company’s future strategy in the cystic fibrosis space, though other CF treatments remain in development.

🧬 Andelyn Biosciences to manufacture AAV for NKH gene therapy [5] [US • 17 Feb 2026]

https://www.prnewswire.com/news-releases/andelyn-biosciences-to-apply-the-aav-curator-platform-to-manufacture-clinical-grade-aav-for-drake-rayden-foundation-for-the-treatment-of-nonketotic-hyperglycinemia-302688707.html
Context: NKH is a rare, severe condition with limited treatment options.
Key point: Andelyn Biosciences has partnered with the Drake Rayden Foundation to manufacture AAV vectors for a gene therapy aimed at treating Nonketotic Hyperglycinemia (NKH).
Implication: This partnership underscores the potential of gene therapy to address ultra-rare diseases, highlighting Andelyn’s growing role in the gene therapy manufacturing sector.

💡 Tozaro raises €6.9 million to reduce gene therapy production costs [6] [EU • 16 Feb 2026]

https://www.eu-startups.com/2026/02/platform-designed-to-cut-cell-and-gene-therapy-production-costs-attracts-e6-9-million-for-uk-startup-tozaro/
Context: The platform aims to improve manufacturing yields and reduce costs for therapies like CAR-T and AAV.
Key point: Tozaro has raised €6.9 million to advance its Smart Polymer platform, designed to reduce the cost of gene therapy production.
Implication: Tozaro’s technology could lower the production barriers for gene therapies, improving patient access.

🔬 Lyell Immunopharma initiates Phase 3 CAR T-cell trial for large B-cell lymphoma [7] [US • 12 Feb 2026]

https://www.globenewswire.com/news-release/2026/02/12/3237094/0/en/lyell-immunopharma-announces-initiation-of-patient-dosing-in-first-of-its-kind-phase-3-head-to-head-car-t-cell-clinical-trial-in-aggressive-large-b-cell-lymphoma.html
Context: The trial is designed to assess efficacy in aggressive large B-cell lymphoma, a highly unmet need.
Key point: Lyell Immunopharma began dosing patients in its Phase 3 head-to-head CAR T-cell trial comparing ronde-cel to other CD19-targeting therapies.
Implication: This study could redefine treatment standards for relapsed/refractory large B-cell lymphoma if ronde-cel proves superior.

🧬 CareDx’s AlloHeme™ shows early promise for relapse detection in AML and MDS [8] [US • 12 Feb 2026]

https://www.businesswire.com/news/home/20260212430487/en/CareDx-Announces-Clinical-Validation-Results-for-AlloHeme-the-First-AIPowered-NGS-Surveillance-Solution-for-AML-and-MDS-PostCell-Therapy
Context: The test, which uses next-generation sequencing, demonstrated high sensitivity and specificity in a clinical trial.
Key point: CareDx’s AI-powered AlloHeme™ test successfully detected cancer relapse in AML and MDS patients earlier than traditional methods.
Implication: AlloHeme™ offers a non-invasive and more accurate way to monitor relapse, potentially improving survival outcomes post-transplant.

🦵 MEDIPOST files IND amendment for stem cell therapy in knee osteoarthritis [9] [US • 13 Feb 2026]

https://www.biospace.com/press-releases/medipost-inc-submits-ind-amendment-with-u-s-food-and-drug-administration-to-initiate-phase-iii-trial
Context: The therapy is derived from umbilical cord blood and aims to address cartilage defects.
Key point: MEDIPOST submitted an IND amendment to initiate a Phase III trial of its mesenchymal stem cell therapy for knee osteoarthritis.
Implication: This submission marks a significant step forward in regenerative medicine for osteoarthritis, potentially offering new treatment options for patients.

Why It Matters

Gene therapy advancements: This week saw both successes and setbacks in gene therapy, from groundbreaking partnerships and funding to trial failures.
Manufacturing and cost reduction: The rise of platforms like Tozaro’s Smart Polymer technology signals a shift toward more accessible and scalable gene therapy production.
Cell therapy progress: Lyell’s CAR T-cell trial and CareDx’s relapse detection test show promise in improving patient outcomes for blood cancers and cell therapy.

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FAQ

Why was Ultragenyx restructuring?

Ultragenyx laid off 10% of its workforce following failed trials and slow progress with FDA approval for its Sanfilippo A gene therapy. The company aims to reduce expenses and focus on its most promising programs.

What is Zemcelpro® and why is it significant?

Zemcelpro® is a cell therapy for blood cancers that has recently gained NUB Status 1 in Germany, offering hospitals reimbursement while further evaluations take place.

How does CareDx’s AlloHeme™ improve relapse detection in AML and MDS?

AlloHeme™ uses AI-powered NGS to detect relapse earlier than traditional methods, offering a non-invasive and highly sensitive monitoring solution for post-transplant patients.

Entities / Keywords

Regenxbio; PDE6A; retinitis pigmentosa; AAV8
Ultragenyx; UX111; Sanfilippo A; layoffs
ExCellThera; Zemcelpro®; NUB Status 1; blood cancers
Boehringer Ingelheim; BI 3720931; cystic fibrosis; gene therapy
Andelyn Biosciences; AAV Curator®; Nonketotic Hyperglycinemia
Tozaro; Smart Polymer; gene therapy production costs
Lyell Immunopharma; ronde-cel; CAR T-cell; large B-cell lymphoma
CareDx; AlloHeme™; AML; MDS
MEDIPOST; mesenchymal stem cell therapy; knee osteoarthritis