This biweekly Cell and Gene Therapy video recap covers the latest regulatory actions, clinical trial updates, data releases, and strategic partnerships. Key developments from the past two weeks highlight shifts in treatment landscapes and new therapeutic approaches.
🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Two Weeks
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- Week 5–11 February 2026
- Week 12–18 February 2026
📚 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 Regenxbio: FDA rejected RGX-121 for Hunter syndrome due to concerns over trial design and surrogate endpoints
0:54 Eli Lilly: Acquired Orna Therapeutics for up to $2.4B to advance in vivo CAR-T cell therapies
1:29 Ultragenyx: Layoffs after gene therapy struggles, still awaiting FDA decision for UX111 in Sanfilippo A
2:01 ExCellThera: Zemcelpro® (dorocubicel) receives NUB Status 1 in Germany for blood cancer treatment
2:34 CareDx: AlloHeme™ shows promise for relapse monitoring in AML and MDS patients post-cell therapy
3:10 Gyala Therapeutics: Initiates Phase I/IIa clinical trial of GYA01 for relapsed AML and T-ALL
3:47 Kincell Bio: Partners with RegCell to advance Treg cell therapies for autoimmune diseases
4:17 MEDIPOST: Submits IND amendment to FDA for Phase III trial of stem cell therapy in knee osteoarthritis
4:44 How to reach us
Transcript
Welcome to the latest edition of Gene & Cell Therapy Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.
First, Regenxbio’s RGX-121 for Hunter syndrome has been rejected by the FDA. The rejection was primarily due to concerns over the trial population definition, the use of a natural-history control group, and the reliance on heparan sulfate as a surrogate endpoint for accelerated approval. While the decision came after a delayed PDUFA date and clinical holds, including a separate RGX-111 trial in which a patient developed a brain tumor, the brain tumor issue was not cited as a cause for the rejection. This rejection may impact prescriber decisions and payer reviews as more data becomes available.
In a significant acquisition, Eli Lilly has entered into an agreement to acquire Orna Therapeutics for up to $2.4 billion. This deal focuses on advancing in vivo CAR-T cell therapies, particularly circular RNA and lipid nanoparticle-based therapies, such as ORN-252 for autoimmune diseases. The acquisition aims to simplify CAR-T production by reducing complexity, cost, and logistical challenges, thus broadening Lilly’s gene therapy pipeline.
Ultragenyx has announced layoffs affecting 10% of its workforce after facing setbacks in gene therapy trials, including disappointing results from brittle bone disease studies and slow progress with the FDA regarding UX111 for Sanfilippo A. Despite these struggles, Ultragenyx continues to focus on high-impact opportunities as it navigates this restructuring process, which may involve shifts in clinical focus and resource allocation.
ExCellThera’s Zemcelpro® (dorocubicel) has received NUB Status 1 in Germany, which grants supplementary reimbursement while broader reimbursement is still under evaluation. This designation supports Zemcelpro® in treating hematological malignancies in patients who do not have suitable donor cells. The NUB Status 1 designation is expected to facilitate adoption in Germany and may provide momentum as the company seeks to expand into other markets.
CareDx’s AlloHeme™, an AI-powered NGS-based solution, has shown promising clinical validation in the detection of relapse in AML and MDS patients following hematopoietic cell therapy. The ACROBAT study demonstrated 85% sensitivity, with a median 41-day lead time before clinical relapse detection. This development expands CareDx’s precision medicine portfolio, providing a blood-based tool for earlier relapse monitoring in these patients.
Gyala Therapeutics has initiated a Phase I/IIa clinical trial of GYA01, a CD84-targeting CAR-T cell therapy, in patients with relapsed or refractory AML and T-ALL. The trial, funded with €3.7 million from Spain’s Ministry of Science, will include dose escalation and expansion phases. This marks a significant step in expanding CAR-T cell therapies for hematologic malignancies, offering hope for patients with limited treatment options.
Kincell Bio has partnered with RegCell to advance Treg cell therapies for autoimmune diseases. Kincell will provide CMC and GMP manufacturing support for RegCell’s platform, which reprograms T cells into antigen-matched Tregs without gene editing. This collaboration could offer a new approach to immune tolerance in autoimmune disorders and supports the development of next-generation cell therapies.
Finally, MEDIPOST has submitted an IND amendment to the FDA for a Phase III trial of its mesenchymal stem cell therapy in knee osteoarthritis. The trial is aimed at addressing symptomatic cartilage defects in patients suffering from this degenerative joint disease. This move advances regenerative medicine for osteoarthritis, addressing an unmet need in joint disease treatments.
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Why It Matters
- FDA’s CRL for RGX-121 emphasizes growing regulatory caution around surrogate endpoints and natural-history control arms, especially in gene therapies targeting neurodegenerative conditions.
- Lilly’s Acquisition of Orna Therapeutics highlights a shift toward simplifying in vivo cell therapies, potentially increasing accessibility and affordability of treatments for autoimmune diseases.
- ExCellThera’s NUB Status provides early reimbursement pathways for Zemcelpro®, signaling a promising move toward widespread adoption in Europe.
- CareDx’s AlloHeme expands beyond solid organ transplants, making a significant stride in cell therapy relapse detection.
- New CAR-T and Treg Cell Therapy Trials by Gyala and Kincell Bio demonstrate the expanding potential of cell therapies to target blood cancers and autoimmune diseases.
🗓️ Explore details and sources
- Week 5–11 February 2026
- Week 12–18 February 2026
📚 See the full Cell and Gene Therapy archive on our research hub page.
FAQ
What did the FDA cite in rejecting Regenxbio’s RGX-121?
The FDA cited concerns about trial design, including population definition, use of a natural-history control, and reliance on heparan sulfate as a surrogate endpoint in the accelerated approval filing [1].
What exactly is Lilly acquiring from Orna?
Lilly is acquiring a circular RNA + LNP platform for in vivo cell engineering, which includes ORN-252, a CD19-targeting CAR-T for autoimmune diseases; the deal is valued up to $2.4B, contingent on milestones [2].
Why did Ultragenyx lay off employees?
Ultragenyx is restructuring after the failure of clinical trials and slow FDA progress with UX111, its gene therapy for Sanfilippo A. The company is focusing resources on high-value opportunities moving forward [3].
What is Zemcelpro® and why is it important?
Zemcelpro® (dorocubicel) is a stem cell therapy for blood cancer patients who lack suitable donor cells, and it has received NUB Status 1 in Germany for reimbursement support [4].
What is AlloHeme™ used for?
AlloHeme™ is an AI-powered, blood-based test for early relapse detection in AML and MDS patients following hematopoietic cell transplantation. It shows strong clinical performance and sensitivity [5].
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
Ultragenyx; UX111; Sanfilippo A; Bayley-III; CSF HS
ExCellThera; Zemcelpro; dorocubicel; blood cancers; NUB Status 1
CareDx; AlloHeme; relapse detection; AML; MDS
Gyala Therapeutics; GYA01; CD84; AML; T-ALL
Kincell Bio; RegCell; Tregs; autoimmune diseases
MEDIPOST; stem cell therapy; knee osteoarthritis
