Cell and Gene Therapy Video Recap—December 11, 2025

This biweekly Cell and Gene Therapy video reacap highlights major regulatory milestones and breakthrough advances across next-generation therapies.

🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Two Weeks

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Week 27 November–3 December 2025
Week 4–10 December 2025

📚 See the full Cell and Gene Therapy archive on our research hub page.

Transcript

Welcome to the latest edition of Gene & Cell Therapy Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.

Imvax has announced results from its Phase 2b trial for the treatment of glioblastoma multiforme (GBM). The trial, involving 99 patients, tested IGV-001, a personalized cancer immunotherapy. While the primary endpoint of progression-free survival (PFS) was missed, the company reported a significant 6.3-month improvement in median overall survival (OS). This OS data is expected to play a critical role in future treatment decisions and reimbursement evaluations. Imvax is now seeking a meeting with the FDA to discuss the regulatory path forward.

Solid Biosciences has received FDA Rare Pediatric Disease and Fast Track designations for SGT-212, a dual-route gene therapy for Friedreich’s ataxia. SGT-212 is designed to restore frataxin levels, a protein deficient in patients with this rare genetic disorder. The company is currently screening participants for a Phase 1b trial. The designations offer an expedited development timeline, potentially allowing quicker access to the market for this treatment.

GenSight Biologics has received approval from France’s National Agency for the Safety of Medicines and Health Products (ANSM) to begin the Phase II REVISE dose-ranging study of GS010, also known as LUMEVOQ. The study, which targets ND4-LHON, a mitochondrial disease that causes progressive vision loss, will evaluate the primary endpoint of Best Corrected Visual Acuity (BCVA) change after 1.5 years. If successful, this study could support early access programs and further regulatory approvals, with the trial scheduled to begin in January 2026.

The FDA has approved Breyanzi® (lisocabtagene maraleucel) as the first and only CAR T-cell therapy for adult patients with relapsed or refractory Marginal Zone Lymphoma (MZL). The approval follows impressive results from the TRANSCEND FL study, which showed a 95.5% overall response rate and a 62.1% complete response rate. This approval offers a much-needed treatment option for patients with limited alternatives in this challenging lymphoma subtype.

In another significant development, the FDA has approved Omisirge (omidubicel) as the first cell therapy for Severe Aplastic Anemia (SAA). Omisirge demonstrated rapid hematopoietic recovery and transfusion independence in clinical trials, providing a promising option for patients with SAA who may struggle with limited donor availability.

The FDA has also approved Waskyra, the first gene therapy for Wiskott-Aldrich Syndrome, a rare immune disorder. Waskyra utilizes autologous stem cells to correct the WAS gene mutation. In clinical trials, the therapy showed a 93% reduction in infections and a 60% reduction in bleeding events within the first year. This represents a significant advancement in gene therapy for rare immune disorders.

Vertex Pharmaceuticals has presented new data on CASGEVY®, a gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in children. The data demonstrates that CASGEVY® offers durable, life-changing benefits, with 100% of SCD patients remaining free from vaso-occlusive crises for over 12 months. This therapy has the potential to revolutionize the treatment of these pediatric conditions.

Lastly, UniQure faces regulatory challenges with its Huntington’s disease gene therapy, AMT-130. Feedback from the FDA suggests that the Phase I/II data may not be sufficient to support a Biologics License Application (BLA). The FDA’s concerns could lead to a delayed path to market, with additional trials or data potentially needed. This setback could impact both investors and patients, delaying access to a treatment for this devastating disease.

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Why It Matters

FDA Approvals and Designations: Rapid approvals and designations such as Rare Pediatric Disease and Fast Track can significantly shorten the time for therapies to reach patients.
Gene Therapy Advancements: The increasing success of gene therapies for rare and chronic diseases highlights their transformative potential.
New Treatment Options: With the approval of therapies like Breyanzi for MZL and Omisirge for SAA, patients now have additional options for conditions with limited treatment landscapes.
Regulatory Hurdles: Challenges faced by companies like UniQure highlight the regulatory complexities and the length of time often needed to bring cutting-edge therapies to market.

🗓️ Explore weekly details and sources

Week 27 November–3 December 2025
Week 4–10 December 2025

📚 See the full Cell and Gene Therapy archive on our research hub page.

FAQ

What is Omisirge and how does it work?

Omisirge (omidubicel) is a cell therapy for severe aplastic anemia, aimed at enhancing hematopoietic recovery. The FDA approved it based on strong clinical results demonstrating transfusion independence and rapid immune recovery.

What is Waskyra, and what does its approval mean for Wiskott-Aldrich syndrome patients?

Waskyra is the first gene therapy approved for Wiskott-Aldrich syndrome. It uses a patient’s stem cells to correct the mutation in the WAS gene, significantly improving immune function and reducing bleeding and infections.

What new data did Vertex present for CASGEVY® in children?

Vertex’s data showed that CASGEVY® gene therapy resulted in 100% of children with SCD being free from vaso-occlusive crises for at least 12 months, offering significant clinical benefits for pediatric patients.

What challenges is UniQure facing with its Huntington’s gene therapy, AMT-130?

UniQure is facing regulatory challenges as the FDA suggested that current Phase I/II data may not be sufficient for a biologics license application (BLA) submission, meaning a longer and more complex path to market.

Entities / Keywords

Imvax; IGV-001; glioblastoma; Solid Biosciences; SGT-212; Friedreich’s ataxia; GenSight Biologics; GS010; LUMEVOQ; ND4-LHON; Breyanzi; lisocabtagene maraleucel; r/r MZL; Omisirge; omidubicel; Severe Aplastic Anemia; Waskyra; Wiskott-Aldrich syndrome; CASGEVY; Sickle Cell Disease; Beta Thalassemia; UniQure; AMT-130; Huntington’s Disease.