This biweekly Cell and Gene Therapy video recap summarizes key developments, including regulatory actions, clinical progress, data releases, and strategic partnerships. The focus is on advancements that could shape the future of therapies and patient care.
🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Two Weeks
🗓️ Explore details and sources
- Week 22–28 January 2026
- Weeks 29 January–4 February 2025
📚 See the full Cell and Gene Therapy archive on our research hub page.
Top Stories Covered in This Video
Chapters
0:00 Introduction
0:09 FDA Grants Wugen’s WU-CART-007 Breakthrough Therapy Designation for relapsed T-ALL/T-LBL
0:41 ImmunityBio Reports positive Phase 2 data for ANKTIVA® plus CAR-NK in glioblastoma
1:12 Intellia FDA lifts clinical hold on CRISPR trial after patient death
1:38 Ultragenyx Announces long-term data for UX111 in Sanfilippo syndrome
2:05 REGENXBIO Faces FDA clinical hold for RGX-111 and RGX-121
2:33 Taysha Doses first patient in pivotal Rett syndrome gene therapy trial
3:04 AstraZeneca Vows $15B investment in China for cell therapy and radioconjugates
3:32 ImmunityBio Launches Phase 2 chemotherapy-free CAR-NK trial in indolent lymphomas
3:56 How to reach us
Transcript
Welcome to the latest edition of Gene and Cell Therapy Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest. In today’s update, we cover several key developments in gene and cell therapy across the US, EU, and Asia.
First up, the FDA has granted Wugen’s investigational CAR-T therapy, WU-CART-007, Breakthrough Therapy Designation for treating relapsed T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL). This designation, granted on January 21, 2026, will accelerate the development and review of the therapy. This marks a significant step forward for the potential treatment of these aggressive T-cell malignancies.
Next, ImmunityBio has reported promising Phase 2 data for ANKTIVA® plus CAR-NK in the treatment of recurrent glioblastoma. The ongoing trial, as of January 23, 2026, shows encouraging results, with the combination therapy demonstrating positive immune competence and survival benefits. This could offer a new standard in the treatment of glioblastoma, with potential for improved patient outcomes.
In a major development, Intellia has resumed its CRISPR trial after the FDA lifted the clinical hold placed following a patient death. The Magnitude and Magnitude-2 studies, which are investigating Intellia’s CRISPR therapy for transthyretin amyloidosis, are set to move forward. This resumption could pave the way for a breakthrough treatment in this rare, fatal disease.
Ultragenyx has announced long-term data from its Phase 1/2 trial of UX111 gene therapy in children with Sanfilippo syndrome (MPS IIIA). As of February 3, 2026, the therapy has shown sustained biomarker improvements and clinical benefits across multiple developmental domains, providing hope for long-term treatment options in this neurodegenerative disease.
However, REGENXBIO faces a regulatory challenge, as the FDA has placed a hold on its RGX-111 and RGX-121 gene therapies following a case of neoplasm in the RGX-111 trial. While no similar events have been reported in other participants, the investigation continues, potentially delaying progress in rare genetic diseases.
On a more positive note, Taysha Gene Therapies has dosed the first patient in its pivotal trial for TSHA-102, a gene therapy for Rett syndrome. The REVEAL trial, which was initiated on February 2, 2026, is a critical step toward developing a treatment for this rare neurodevelopmental disorder. Taysha is also working closely with the FDA to align on BLA submission plans.
In China, AstraZeneca has made a bold move by announcing a $15 billion investment in cell therapy and radioconjugates. The investment, planned to support R&D and manufacturing through 2030, underscores China’s growing influence in biopharma and international collaborations. This will enhance AstraZeneca’s capabilities in the rapidly evolving field of cell therapy.
Lastly, ImmunityBio has launched a Phase 2 trial to evaluate its chemotherapy-free CAR-NK cell therapy in indolent non-Hodgkin lymphoma (iNHL). This trial, which began on February 2, 2026, is exploring a regimen combining CAR-NK and ANKTIVA®, potentially offering a less toxic treatment option for lymphoma patients.
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Why It Matters
- Regulatory Milestones: The FDA’s Breakthrough Therapy Designations and clinical trial advances continue to play a pivotal role in accelerating the approval of groundbreaking treatments. These milestones not only expedite access to therapies but also demonstrate how regulatory bodies are embracing new technologies in gene and cell therapies. For instance, Wugen’s CAR-T therapy for T-ALL/T-LBL and Intellia’s CRISPR-based therapy are now on fast tracks to potentially changing the standard of care for previously untreated or poorly treated diseases.
- Strategic Investment and Global Expansion: AstraZeneca’s $15B investment into China underscores the growing importance of global partnerships and local innovation hubs in accelerating the development of cell and gene therapies. This move also aligns with China’s expanding capabilities in the biotech space, making it an attractive site for clinical trials, manufacturing, and innovation.
- Therapeutic Innovation in Rare Diseases: The long-term data from Ultragenyx and the advancements in Taysha’s Rett syndrome gene therapy provide hope for patients with rare, often neglected diseases. These therapies are addressing significant unmet medical needs and could pave the way for novel treatments that transform the management of such conditions, offering patients a chance for improved quality of life or even disease stabilization.
- Efforts in Non-Toxic Therapies: ImmunityBio’s chemotherapy-free CAR-NK trials and other advancements in off-the-shelf cell therapies represent a shift toward less toxic, more accessible treatments. By eliminating the need for harsh chemotherapy regimens, such approaches make life-saving therapies more accessible to a broader population while reducing the side effects typically associated with cancer treatments.
🗓️ Explore details and sources
- Week 22–28 January 2026
- Weeks 29 January–4 February 2025
📚 See the full Cell and Gene Therapy archive on our research hub page.
FAQ
What does the FDA’s Breakthrough Therapy Designation mean for Wugen’s WU-CART-007?
This designation will expedite the development of Wugen’s CAR-T therapy, potentially bringing it to market faster for treating T-ALL/T-LBL.
How will Ultragenyx’s long-term data for UX111 impact patients with Sanfilippo syndrome?
A: The data suggests UX111 could provide a long-term solution to slow or halt the progression of this devastating neurodegenerative disorder in children.
What’s the significance of Taysha’s Rett syndrome gene therapy trial?
The initiation of the pivotal trial for TSHA-102 offers new hope for the treatment of Rett syndrome, with potential regulatory approval in sight.
Why is AstraZeneca focusing on China with its $15B investment?
A: The investment aims to enhance AstraZeneca’s capabilities in cell therapies and radioconjugates, while capitalizing on China’s growing scientific prowess in biopharma.
Entities / Keywords
FDA; Wugen; WU-CART-007; ImmunityBio; ANKTIVA®; CAR-NK; Intellia; CRISPR; Ultragenyx; UX111; REGENXBIO; RGX-111; RGX-121; Taysha; Rett syndrome; AstraZeneca; cell therapy; lymphoma.
References
[4] https://www.biospace.com/press-releases/ultragenyx-announces-positive-longer-term-data-demonstrating