Cell and Gene Therapy Today—February 5, 2026

In Today’s Newsletter

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🧬 Ultragenyx Announces Positive Long-Term Data for UX111 Gene Therapy in Sanfilippo Syndrome (MPS IIIA) [1] [US • 3 Feb 2026]

https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-longer-term-data-demonstrating
Context: Ultragenyx shares data from up to 8.5 years of follow-up for its UX111 gene therapy in children with Sanfilippo Syndrome (MPS IIIA).
Key Point: Significant reductions in cerebrospinal fluid (CSF) heparan sulfate and improvements in cognitive and motor functions were observed.
Implication: These results support the continued development of UX111 as a potential treatment for this rare and devastating disease.

🌱 REGENXBIO Faces FDA Clinical Hold on RGX-111 and RGX-121 for MPS I and MPS II [2] [US • 28 Jan 2026]

https://regenxbio.gcs-web.com/news-releases/news-release-details/regenxbio-announces-regulatory-update-ultra-rare-mps-programs
Context: The FDA has placed a clinical hold on REGENXBIO’s gene therapies RGX-111 and RGX-121 after the discovery of a neoplasm in a patient treated with RGX-111.
Key Point: Investigations into the cause of the adverse event are ongoing, with no tumor found in other patients.
Implication: This clinical hold may delay the development of these therapies, which are targeting rare genetic diseases.

🧬 Taysha Gene Therapies Doses First Patient in Pivotal Phase 3 Trial for Rett Syndrome [3] [US • 30 Jan 2026]

https://www.neurologylive.com/view/taysha-doses-first-patient-pivotal-trial-rett-syndrome-gene-therapy-tsha-102-aligns-fda-bla-plans
Context: Taysha begins dosing patients in the REVEAL Phase 3 trial for TSHA-102, a gene therapy for Rett syndrome.
Key Point: The company has aligned with the FDA for a potential biologics license application (BLA) based on positive data from previous trials.
Implication: TSHA-102 could become a breakthrough treatment for Rett syndrome, which has limited therapeutic options.

💡 ZipBio and MeiraGTx Partner to Develop AAV Gene Therapy for Geographic Atrophy [4] [US • 3 Feb 2026]

https://investingnews.com/zipbio-and-meiragtx-enter-into-exclusive-license-agreement-to-advance-first-in-class-aav-gene-therapy-for-geographic-atrophy/
Context: ZipBio and MeiraGTx enter into a licensing deal to advance a first-in-class AAV gene therapy targeting the complement pathway for Geographic Atrophy.
Key Point: ZipBio’s AI-driven platform will help design next-generation gene therapies, while MeiraGTx will handle clinical development and manufacturing.
Implication: This collaboration could speed up the development of gene therapies for retinal diseases with high unmet medical needs.

👁️ Ocugen’s OCU410ST Gene Therapy Shows Positive Results in Stargardt Disease [5] [US • 3 Feb 2026]

https://www.neurologylive.com/view/ocugen-stargardt-gene-therapy-ocu410st-provides-6-letter-gain-bcva-evaluable-patients-phase-trial
Context: Ocugen announces a +6-letter gain in visual acuity and reduced lesion growth in patients treated with OCU410ST for Stargardt disease.
Key Point: The therapy showed a 54% reduction in lesion expansion compared to untreated eyes.
Implication: OCU410ST may provide a significant treatment option for patients with Stargardt disease, a condition with limited current therapies.

🧬 Siren Biotechnology Receives FDA IND Clearance for Cancer Gene Therapy Trial [6] [US • 28 Jan 2026]

https://sirenbiotechnology.com/siren-biotechnology-announces-fda-clearance-of-first-ind-advancing-company-to-clinical-stage/
Context: Siren Biotechnology receives FDA clearance for its first IND application, allowing the company to begin its first human clinical trial in cancer.
Key Point: The trial will evaluate the company’s Universal AAV Immuno-Gene Therapy platform in patients with recurrent high-grade glioma.
Implication: This marks a major milestone for Siren as it transitions into clinical-stage operations, potentially paving the way for new cancer therapies.

🤖 NanoMosaic Secures FDA AMT Designation for AAV Gene Therapy Analytics [7] [US • 29 Jan 2026]

https://www.prnewswire.com/news-releases/nanomosaic-secures-fda-advanced-manufacturing-technology-amt-designation-for-advanced-gene-therapy-analytics-302673514.html
Context: NanoMosaic receives FDA Advanced Manufacturing Technology (AMT) designation for its Nanoneedle platform, designed to enhance AAV gene therapy manufacturing.
Key Point: The platform allows for more efficient and scalable manufacturing of gene therapies.
Implication: The AMT designation could speed up the adoption of NanoMosaic’s technology across the gene therapy industry.

🏭 AstraZeneca Commits $15B to Boost Cell Therapy and Radioconjugate Capabilities in China [8] [China • 29 Jan 2026]

https://www.fiercebiotech.com/biotech/astrazeneca-vows-15b-china-investment-boost-cell-therapy-and-radioconjugate-capabilities
Context: AstraZeneca announces a $15 billion investment in China to expand its research and development in cell therapy and radioconjugates.
Key Point: The investment will focus on drug discovery, clinical development, and increasing AstraZeneca’s R&D footprint in China.
Implication: This commitment highlights China’s growing role in the global biopharmaceutical landscape.

🧬 ImmunityBio Launches Phase 2 Trial for CAR-NK Cell Therapy in Indolent Lymphoma [9] [US • 2 Feb 2026]

https://ir.immunitybio.com/news-releases/news-release-details/immunitybio-launches-phase-2-chemotherapy-free-car-nk-cell
Context: ImmunityBio initiates a Phase 2 trial evaluating its chemotherapy-free CAR-NK cell therapy for indolent non-Hodgkin lymphoma (iNHL).
Key Point: The therapy builds on promising Phase 1 results, where patients achieved durable responses without the need for chemotherapy.
Implication: If successful, this approach could offer a less toxic, more patient-friendly treatment for indolent B-cell malignancies.

Why It Matters

Regulatory Evolution: FDA actions such as clinical holds, IND clearances, and AMT designations underscore a more active regulatory role in balancing safety with accelerated development of cell and gene therapies.
Advancements in Rare Diseases: Progress in Sanfilippo syndrome, Rett syndrome, Stargardt disease, and other rare conditions highlights gene therapy’s expanding ability to address high unmet medical needs.
Strategic Partnerships and Investment: Collaborations like ZipBio–MeiraGTx and AstraZeneca’s $15B China investment demonstrate how partnerships and capital deployment are driving global cell and gene therapy growth.
Manufacturing and Platform Innovation: Advances in AAV analytics, scalable manufacturing technologies, and novel cell therapy platforms are critical to enabling broader access and long-term sustainability of these therapies.

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FAQ

What does Ultragenyx’s long-term data for UX111 mean for patients with Sanfilippo Syndrome?

The long-term data shows that UX111 can not only reduce biomarkers associated with the disease but also improve cognitive and motor functions, offering hope for patients who face rapid decline without intervention.

What is the significance of the FDA clinical hold on REGENXBIO’s RGX-111 and RGX-121?

While this hold is concerning, it highlights the need for caution in gene therapy development. It is crucial that patient safety remains a priority, and the ongoing investigation will help ensure these therapies’ safety and efficacy before moving forward.

What impact will AstraZeneca’s $15B investment in China have?

AstraZeneca’s investment could dramatically increase the pace of drug discovery and manufacturing in China, enabling faster access to cutting-edge treatments in Asia, while strengthening China’s role as a global biotech hub.

Why is ImmunityBio’s CAR-NK therapy for iNHL important?

ImmunityBio’s CAR-NK therapy offers a promising chemotherapy-free option for lymphoma patients, which could dramatically improve the quality of life for those with indolent lymphoma and other B-cell malignancies. This could be a game-changer for a population that has limited treatment options.

Entities / Keywords

Ultragenyx; REGENXBIO; Taysha Gene Therapies; ZipBio; MeiraGTx; Ocugen; Siren Biotechnology; NanoMosaic; AstraZeneca; ImmunityBio; gene therapy; MPS IIIA; MPS I; MPS II; Rett syndrome; Stargardt disease; high-grade glioma; CAR-NK; AAV; geographic atrophy; indolent lymphoma.