This Cell & Gene Therapy update highlights momentum across cell and gene therapy, spanning regulatory approvals, late-stage submissions, clinical trial milestones, and new treatment launches. Advances range from rare diseases to oncology and neurology, reflecting accelerating translation from development to real-world care.
In Today’s Newsletter
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🧬 Hope Biosciences Reports Positive Phase II Parkinson’s Trial Results [1] [US • 22 Dec 2025]
Context: Hope Biosciences’ allogeneic adipose-derived mesenchymal stem cell therapy (HB-adMSCs) achieved statistically significant motor function improvements in early-to-moderate Parkinson’s patients.
Key point: End-of-study MDS-UPDRS Part III scores improved −9.82 points vs −0.50 in placebo; effect grew over successive infusions.
Implication: Supports repeated allogeneic stem cell therapy as a promising avenue for motor function restoration in Parkinson’s disease.
🌱 EUDA Launches Comprehensive Stem Cell Therapy Platform and Shenzhen Longevity Clinic [2] [SG/CN • 23 Dec 2025]
Context: EUDA expanded its regenerative medicine strategy, upgrading a cGMP facility and establishing its first clinic in Shenzhen.
Key point: Integrates iPSC and T-cell therapies for immune, skin, and healthy aging interventions.
Implication: Positions EUDA to lead Asia’s longevity sector with an end-to-end regenerative ecosystem.
🧬Qihan Biotech Initiates Phase I/IIa Trial for CAR-T Therapy in Refractory SLE [3] [CN/US • 18 Dec 2025]
Context: QT-019B, an off-the-shelf allogeneic CAR-T therapy targeting CD19/BCMA, is being tested in refractory systemic lupus erythematosus.
Key point: Trial evaluates safety, tolerability, and efficacy in an open-label, multicenter design with dose-escalation and expansion stages.
Implication: Could provide a more accessible CAR-T treatment for severe autoimmune disease patients.
💉 CARsgen Submits Dual INDs for Allogeneic BCMA CAR-T CT0596 [4] [CN • 28 Dec 2025]
Context: INDs submitted for Phase Ib/II trials in relapsed/refractory multiple myeloma and plasma cell leukemia.
Key point: CT0596 incorporates gene edits to reduce GvHD and host rejection; preliminary IIT data shows safety and early efficacy.
Implication: Initiates registration-oriented clinical development for novel BCMA-targeted CAR-T therapy.
🩸 Omeros’ Yartemlea Approved by FDA for TA-TMA [5] [US • 26 Dec 2025]
Context: First approved therapy for hematopoietic stem cell transplant-associated thrombotic microangiopathy.
Key point: Demonstrated 61–68% complete response rates; survival at 100 days ~73–74%.
Implication: Provides a life-saving therapy for a high-mortality post-transplant complication.
🌏 Korea Clears Regenerative Medicine Trials for Rare Diseases [6] [KR • 19 Dec 2025]
https://www.koreabiomed.com/news/articleView.html?idxno=30049
Context: Government approvals granted for Sjögren’s syndrome, knee osteoarthritis, and glioblastoma cell therapy studies.
Key point: Trials leverage autologous or progenitor cells to restore tissue function and assess efficacy in patients.
Implication: Advances clinical translation of regenerative medicine for rare and aggressive diseases in Korea.
🧬 T-MAXIMUM Pharma Receives FDA IND Clearance for MT027 CAR-T in Glioblastoma [7] [US • 21 Dec 2025]
Context: MT027 is an allogeneic B7-H3-targeted CAR-T therapy for recurrent glioblastoma.
Key point: IND clearance enables Phase II evaluation; therapy uses non-viral gene-editing platform.
Implication: Paves the way for allogeneic CAR-T in difficult-to-treat solid tumors.
👁️ Ikarovec & VectorBuilder Partner to Deliver Office-Based Eye Gene Therapy [8] [UK • 06 Jan 2026]
Context: IKAR-003, preclinical gene therapy for intermediate AMD, can now be administered via intravitreal injection in-office.
Key point: Partnership potentially worth $1B; technology enables wider clinical adoption.
Implication: Could significantly improve access and early intervention in age-related macular degeneration.
🧬 Taysha Gene Therapies Updates TSHA-102 Rett Syndrome Program [9] [US • 06 Jan 2026]
Context: REVEAL pivotal trial and ASPIRE safety trial progressing; FDA alignment achieved for broad label submission.
Key point: First patient dosed in REVEAL; completion expected Q2 2026.
Implication: Positions TSHA-102 to transform Rett syndrome therapy, potentially benefiting 15,000–20,000 patients in US/EU/UK.
🤝 ENCell and Japan’s CRC Sign Strategic Partnership for Cell and Gene Therapy Expansion [10] [KR/JP • 24 Dec 2025]
https://www.koreabiomed.com/news/articleView.html?idxno=30094
Context: ENCell signed a strategic partnership with Cell Resources Corporation (CRC) to expand cell and gene therapy business in Japan.
Key point: Partnership enables licensing, CDMO services, and technology transfer of ENCell’s MSC-based therapies, including EN001 for CMT, DMD, and sarcopenia.
Implication: Accelerates Japanese market entry and global technology transfer of next-generation regenerative therapies.
🧬 NeuExcell Reports Clinical Results with NXL-004 for Malignant Glioma [11] [SG • 18 Dec 2025]
Context: NXL-004, the first in situ conversion gene therapy, reprograms glioma cells into non-dividing neurons or triggers apoptosis.
Key point: Median OS >12 months in recurrent malignant glioma patients; one patient achieved complete response after tumor resection plus NXL-004 injection.
Implication: Demonstrates feasibility and clinical potential of in situ cell conversion therapy for aggressive brain tumors.
🇮🇱 GenSight Biologics Receives Early Access Authorization for GS010/LUMEVOQ® in Israel [12] [IL • 23 Dec 2025]
Context: Israel’s Ministry of Health authorizes individual patient early access to GS010/LUMEVOQ® for ND4-LHON.
Key point: Bilateral injection expected Q1 2026; for patients with life-threatening or seriously debilitating vision loss.
Implication: Expands early patient access to GS010/LUMEVOQ® while pivotal Phase III RECOVER study is underway.
🇫🇷 GenSight Biologics Granted Compassionate Use Authorization for GS010/LUMEVOQ® in France [13] [FR • 22 Dec 2025]
Context: French ANSM grants named patient early access program (AAC) for GS010/LUMEVOQ® in ND4-LHON.
Key point: Access provided when no other approved therapy exists; based on favorable benefit-risk ratio.
Implication: Provides life-altering therapy for rare mitochondrial optic neuropathy patients ahead of full market authorization.
🧬 Modalis Publishes Human Gene Therapy Study Demonstrating LAMA1 Activation for LAMA2-CMD [14] [JP/US • 25 Dec 2025]
Context: CRISPR-GNDM® epigenome editing platform safely activates LAMA1 as a therapeutic approach for LAMA2-CMD.
Key point: Preclinical data show improved survival, muscle function, and favorable safety in NHPs.
Implication: Supports progression to clinical trials of MDL-101 as a one-time, durable therapy for severe pediatric muscular dystrophy.
🤝 Minaris and Cell and Gene Therapy Catapult Announce Collaboration [15] [UK/US • 18 Dec 2025]
Context: Collaboration develops lentiviral (LV) and AAV delivery technologies to improve manufacturing, quality, and reduce costs.
Key point: Shared lab resources in London; advances in LV/AAV vector production.
Implication: Enhances global CGT manufacturing capabilities, facilitating faster access to advanced therapies.
🌍 Abu Dhabi First to Deliver ITVISMA Gene Therapy for SMA [16] [AE • 29 Dec 2025]
Context: ITVISMA (onasemnogene abeparvovec) administered at Sheikh Khalifa Medical City for SMA patients ≥2 years old.
Key point: UAE approved therapy on 25 Nov 2025; first global administration outside US.
Implication: Highlights Abu Dhabi as a leading hub for advanced neuromuscular care and precision medicine access.
🧬 CRISPR Therapeutics Updates Zugocaptagene Geleucel (Zugo-cel) Development [17] [CH/US • 22 Dec 2025]
Context: Zugo-cel, an allogeneic CD19 CAR-T therapy, evaluated in autoimmune and hematologic malignancies.
Key point: 90% ORR and 70% CRR in LBCL at 600M dose; SLE patient achieved DORIS remission through Month 6.
Implication: Demonstrates broad therapeutic potential across autoimmune diseases and hematologic cancers.
🩸 Sangamo Initiates Rolling Submission of BLA for ST-920 in Fabry Disease [18] [US • 18 Dec 2025]
Context: ST-920 (isaralgagene civaparvovec) gene therapy targets underlying Fabry disease pathology.
Key point: Positive STAAR study eGFR slope at 52 weeks; BLA under accelerated approval pathway.
Implication: Potential one-time durable treatment for adults with Fabry disease.
🧬 Ultragenyx Completes Rolling Submission of BLA for DTX401 in GSDIa [19] [US • 30 Dec 2025]
Context: DTX401 (pariglasgene brecaparvovec) is AAV gene therapy for Glycogen Storage Disease Type Ia.
Key point: Based on 52-patient program with 6-year follow-up; showed meaningful improvements in hypoglycemia control, euglycemia, and QoL.
Implication: Could become first therapy targeting the underlying cause of GSDIa, significantly improving patient outcomes.
Why It Matters
- Regulatory evolution is reshaping gene and cell therapy development: FDA’s new CAR-T policy requiring randomized superiority trials [4] and rolling BLA submissions (Sangamo [18], Ultragenyx [19]) are raising evidentiary standards, potentially slowing approval timelines but increasing confidence in safety and efficacy.
- Breakthrough therapies for rare and severe diseases: Approvals and early access programs for SAA (Omisirge [1]), Wiskott-Aldrich syndrome (Waskyra [5]), RDEB (ZEVASKYN [3]), ND4-LHON (GS010/LUMEVOQ [12,13]), hemophilia B (Hemgenix [7]), SMA (ITVISMA [16]), Fabry disease (ST-920 [18]), and GSDIa (DTX401 [19]) demonstrate transformative potential in previously underserved patient populations.
- Innovative gene therapy modalities expanding the therapeutic toolkit: In situ cell conversion (NXL-004 [11]), base editing (BE-CAR7 [10]), epigenome editing (MDL-101 [14]), and allogeneic CAR-T (Zugo-cel [17]) are pioneering next-generation approaches that may provide one-time, durable, or disease-modifying solutions.
- Strategic collaborations accelerate development and global access: Partnerships such as ENCell & CRC [10], SCTbio & Fortrea [8], Minaris & CGT Catapult [15], and CRISPR Therapeutics & Lilly [17] enhance manufacturing, clinical, and regulatory capabilities, enabling faster delivery of advanced therapies to patients worldwide.
- Focus on pediatric and early intervention: Several therapies target young patients or early-stage disease (CASGEVY [11], ETX101 [9], ITVISMA [16], DTX401 [19]), emphasizing the potential to prevent irreversible damage and improve long-term outcomes.
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FAQ
What is the significance of Omisirge’s approval? [1]
Omisirge provides a novel cell therapy for SAA, achieving 86% sustained neutrophil recovery and transfusion independence, especially important for patients without a matched donor.
How does Breyanzi impact patients with MZL? [2]
Breyanzi is the first CAR-T therapy approved for relapsed/refractory MZL, showing a 95.5% overall response rate, expanding CAR-T treatment access across five cancer types.
What are the benefits of ZEVASKYN for RDEB patients? [3]
ZEVASKYN offers an autologous gene therapy for this debilitating skin disease, providing potential for long-term improvement in wound healing and quality of life.
How will FDA’s CAR-T policy change affect developers? [4]
Future CAR-T therapies must demonstrate superiority in randomized trials, increasing evidence standards but potentially extending development timelines.
What does Waskyra approval mean for WAS patients? [5]
Waskyra reduces severe infections by 93% and bleeding by 60%, offering a transformative treatment option for a rare, life-threatening immune disorder.
What challenges does uniQure face with AMT-130? [6]
FDA feedback indicates inadequate Phase I/II data, suggesting a longer, more challenging regulatory path for Huntington’s disease therapy.
How does Hemgenix benefit hemophilia B patients? [7]
Hemgenix maintains factor IX activity at 36% over 5 years, dramatically reducing bleeding episodes and the need for ongoing prophylactic treatment.
What is the impact of the SCTbio-Fortrea collaboration? [8]
Streamlines clinical development and manufacturing for cell and gene therapies, accelerating access to advanced therapies.
What are early ETX101 results for Dravet syndrome? [9]
ETX101 reduces seizures by 78% and improves cognitive outcomes in young children, showing promise as a disease-modifying therapy.
How has BE-CAR7 advanced leukemia treatment? [10]
Achieves 64% disease-free remission in aggressive T-cell leukemia, offering hope for previously incurable patients.
What is the potential of CASGEVY in younger sickle cell patients? [11]
Provides high rates of transfusion independence and disease-free periods, representing a major advancement in pediatric therapy.
How does ENCell’s partnership with CRC accelerate market access? [10]
Enables technology transfer, licensing, and CDMO expansion, paving the way for commercialization of MSC and CAR-T therapies in Japan.
What is the significance of NXL-004 in glioma? [11]
First in situ conversion therapy showing complete tumor regression in early human studies, offering a novel approach to aggressive brain tumors.
How does early access to GS010/LUMEVOQ impact patients? [12,13]
Provides life-changing treatment for ND4-LHON before full market approval, critical for patients with rapid vision loss.
How does MDL-101 work for LAMA2-CMD? [14]
Uses CRISPR-GNDM® epigenome editing to activate compensatory LAMA1 gene, improving muscle function and survival in preclinical models.
How does the Minaris-CGT Catapult collaboration help developers? [15]
Improves LV/AAV delivery platforms, reducing costs and enhancing viral vector quality for advanced gene therapies.
What milestone does ITVISMA administration in Abu Dhabi represent? [16]
First global administration outside the US for SMA, demonstrating UAE leadership in precision medicine.
What are key results from Zugo-cel studies? [17]
Shows deep, sustained B-cell depletion in autoimmune disease and high ORR/CRR in hematologic malignancies, supporting broad clinical utility.
What is the potential of ST-920 and DTX401? [18,19]
ST-920 offers one-time therapy for Fabry disease; DTX401 addresses the underlying cause of GSDIa, both representing durable, transformative treatments.
Entities / Keywords
Omisirge; SAA; Breyanzi; CAR-T therapy; MZL; ZEVASKYN; RDEB; FDA; CAR-T superiority trials; Waskyra; WAS; uniQure; AMT-130; Huntington’s disease; Hemgenix; CSL Behring; SCTbio; Fortrea; ETX101; Dravet syndrome; BE-CAR7; T-cell leukemia; CASGEVY; Vertex; ENCell; CRC; EN001; NXL-004; Malignant glioma; GS010/LUMEVOQ; ND4-LHON; Modalis; MDL-101; LAMA1; LAMA2-CMD; Minaris; CGT Catapult; ITVISMA; SMA; Zugo-cel; CRISPR Therapeutics; ST-920; Fabry disease; DTX401; GSDIa.
References
https://www.koreabiomed.com/news/articleView.html?idxno=30094