This Cell and Gene Therapy video recap highlights major regulatory milestones, late-stage gene and cell therapy progress, and first-in-class treatment approvals across rare and severe diseases. Key developments include early access programs, pivotal trial advances, and landmark regulatory decisions shaping patient access.
🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Four Weeks
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- Week 11–17 December 2025
- Weeks 18 December–7 January 2025
📚 See the full Cell and Gene Therapy archive on our research hub page.
Top Stories Covered in This Video
Chapters
0:00 Introduction
0:08 GS010/LUMEVOQ® Israel: GenSight receives regulatory authorization for early access in ND4-LHON patients
0:38 GS010/LUMEVOQ® France: Compassionate use program approved by ANSM for ND4-LHON
0:58 ITVISMA SMA: First global administration of onasemnogene abeparvovec in Abu Dhabi for SMA patients ≥2 years 1:23 Zugo-cel CAR-T: CRISPR reports promising results in autoimmune and B-cell malignancies
1:58 ST-920 Fabry: Sangamo initiates rolling BLA submission for Fabry disease gene therapy
2:19 DTX401 GSDIa: Ultragenyx completes BLA submission for Glycogen Storage Disease Type Ia
2:40 HB-adMSCs Parkinson’s: Hope Biosciences Phase II trial shows motor improvement and safety
3:03 Yartemlea TA-TMA: FDA approves first MASP-2 inhibitor for hematopoietic stem cell transplant-associated TMA 3:31 IKAR-003 AMD: Ikarovec partners with VectorBuilder for office-based intravitreal gene therapy delivery
3:50 TSHA-102 Rett Syndrome: Taysha progresses pivotal trial and aligns with FDA on BLA submission
4:17 How to reach us
Transcript
Welcome to the latest edition of Gene and Cell Therapy Updates, covering recent breakthroughs in the field. Brought to you by LucidQuest.
GenSight has received regulatory authorization in Israel for the early access of GS010/LUMEVOQ® in ND4-LHON patients. This approval allows for individual patient access with physician initiation, ethics committee approval, and Ministry authorization. Bilateral injections are expected in Q1 2026, offering urgent treatment to those with debilitating vision loss.
In France, GS010/LUMEVOQ® has been granted compassionate use authorization by the ANSM for ND4-LHON patients, expanding early access options for those with serious and rare diseases ahead of pivotal trial data.
In Abu Dhabi, the first global administration of ITVISMA, an onasemnogene abeparvovec gene therapy, was delivered to SMA patients aged two and older. ITVISMA, which replaces the defective SMN1 gene, was granted accelerated approval in the UAE, highlighting the growing adoption of gene therapies for older SMA patients.
CRISPR Therapeutics shared promising updates on Zugo-cel, a CRISPR-edited CAR-T therapy, in autoimmune diseases and relapsed/refractory large B-cell lymphoma. In autoimmune diseases, a patient with SLE achieved drug-free remission by Month 6. In B-cell malignancies, Zugo-cel showed a 90% overall response rate and a 70% complete response rate at a 600 million cell dose. These results support further development and combination studies.
Sangamo Therapeutics has initiated a rolling BLA submission for ST-920, a gene therapy for Fabry disease. The therapy showed positive results in the STAAR study, with a significant improvement in eGFR at 52 weeks, making it a promising one-time treatment for Fabry patients.
Ultragenyx has completed its BLA submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa). The Phase 3 GlucoGene study showed significant improvements in euglycemia and reduced cornstarch intake in 52 patients, with up to 6 years of follow-up data.
Hope Biosciences has reported positive results from its Phase II trial of HB-adMSCs in Parkinson’s disease. The therapy improved motor function with six intravenous infusions over 32 weeks. It was safe and well-tolerated, supporting progression to Phase III trials.
The FDA has approved Yartemlea, the first MASP-2 inhibitor, for the treatment of thrombotic microangiopathy (TMA) in stem cell transplant patients. With a complete response rate of 61–68% and a 100-day survival rate of 73–74%, this approval provides a life-saving option for TMA patients.
IKAR-003, an AAV gene therapy for AMD, will soon be delivered intravitreally in doctors’ offices through a partnership with VectorBuilder. This office-based approach could expand accessibility to gene therapy, transforming treatment for AMD.
Taysha Gene Therapies has made progress with TSHA-102, a gene therapy for Rett syndrome. The first patient has been dosed in the REVEAL trial, and the FDA has agreed to include ASPIRE study data in the BLA submission. Completion of both trials is expected by Q2 2026, supporting an accelerated regulatory pathway for Rett syndrome gene therapy.
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Why It Matters
- Global expansion of gene therapy programs demonstrates broader patient access and regulatory flexibility.
- Advancements in autoimmune, neurodegenerative, and ophthalmic gene therapies show potential to transform care for rare and debilitating diseases.
- Collaborations and early access programs accelerate treatment availability ahead of pivotal data.
🗓️ Explore details and sources
- Week 11–17 December 2025
- Weeks 18 December–7 January 2025
📚 See the full Cell and Gene Therapy archive on our research hub page.
FAQ
What does early access of GS010/LUMEVOQ® mean for patients? [1,2]
It allows patients with ND4-LHON in Israel and France to receive a potentially vision-saving treatment before it is officially approved.
Why is ITVISMA’s global administration significant? [3]
It is the first time this treatment is being used in patients aged two years and older, expanding treatment beyond infants.
What makes Zugo-cel notable? [4]
It shows strong results in autoimmune diseases and relapsed B-cell lymphoma, supporting wider use of CAR-T therapies.
How could ST-920 and DTX401 change treatment? [5,6]
They could become one-time treatments for Fabry disease and GSDIa, with long-lasting, disease-changing effects.
What is the importance of HB-adMSCs in Parkinson’s? [7]
They show that stem cell therapy can be safe and effective, with the potential to slow disease progression.
Why is Yartemlea approval impactful? [8]
It is the first approved treatment for TA-TMA, a serious and life-threatening condition after transplant.
How could IKAR-003 delivery change ophthalmology? [9]
It allows gene therapy to be given in outpatient settings, reducing the need for hospital-based care.
What progress has TSHA-102 made for Rett syndrome? [10]
The first patient has been treated in a key trial, and upcoming regulatory submissions will include safety data.
Entities / Keywords
GS010/LUMEVOQ; ND4-LHON; ITVISMA; SMA; Zugo-cel; CRISPR; CAR-T; ST-920; Fabry disease; DTX401; GSDIa; HB-adMSCs; Parkinson’s disease; Yartemlea; TA-TMA; IKAR-003; AMD; TSHA-102; Rett syndrome; Taysha Gene Therapies; FDA; ANSM.