This week’s Cell & Gene Therapy update highlights two major FDA approvals in severe aplastic anemia and Wiskott–Aldrich syndrome, along with key policy shifts, emerging gene therapy data, and important clinical developments across the field.
In Today’s Newsletter
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In Today’s Newsletter
🧬 FDA Approves Omisirge for Severe Aplastic Anemia (SAA) [1] [US • 05 Dec 2025]
Context: Omisirge, a novel cell therapy, received FDA approval for SAA, offering promising early and sustained recovery outcomes for patients.
Key point: 86% of patients had sustained neutrophil recovery and transfusion independence after treatment.
Implication: This approval provides a transformative new option for patients with SAA, especially those without a matched donor.
🤝 Breyanzi Approved for Relapsed/Refractory Marginal Zone Lymphoma [2] [US • 04 Dec 2025]
Context: The FDA approved Breyanzi as the first CAR-T therapy for MZL, showing 95.5% overall response rate in the study.
Key point: This approval makes Breyanzi the first CAR-T therapy for five different cancer types.
Implication: Breyanzi’s approval expands access to CAR-T for patients with multiple cancer types, advancing the field of immunotherapy.
🧬 Abeona Announces First Patient Treatment with ZEVASKYN for RDEB [3] [US • 08 Dec 2025]
Context: ZEVASKYN is the first autologous gene therapy approved for RDEB, a rare genetic skin condition.
Key point: The first patient was treated at Lucile Packard Children’s Hospital.
Implication: This marks a significant milestone for the treatment of rare genetic diseases and offers new hope for RDEB patients.
🧪 FDA Shifts CAR-T Approval Strategy to Superiority Trials [4] [US • 09 Dec 2025]
Context: FDA’s new policy requires CAR-T therapies to show superiority in randomized controlled trials (RCTs) over existing treatments.
Key point: This shift sets a higher evidentiary standard for CAR-T developers, potentially impacting future approvals.
Implication: While this policy could enhance treatment standards, it may slow the approval process for new CAR-T therapies.
🧠 FDA Approves Waskyra for Wiskott-Aldrich Syndrome [5] [US • 10 Dec 2025]
Context: Waskyra, the first gene therapy for Wiskott-Aldrich Syndrome (WAS), received FDA approval, reducing severe infections and bleeding.
Key point: The therapy reduced severe infections by 93% and bleeding by 60%.
Implication: Waskyra offers a groundbreaking treatment option for WAS, a rare and life-threatening immune disorder.
🤔 uniQure Faces Regulatory Setback for Huntington’s Gene Therapy [6] [US • 04 Dec 2025]
Context: The FDA has raised concerns over the adequacy of data from uniQure’s Phase I/II trials of AMT-130 for Huntington’s disease.
Key point: UniQure now faces a longer and more challenging regulatory road ahead.
Implication: The setback puts AMT-130’s approval timeline in doubt, impacting Huntington’s disease gene therapy progress.
🩸 CSL’s Hemgenix Shows Durable Efficacy in Hemophilia B [7] [EU • 07 Dec 2025]
Context: CSL Behring presented long-term data at ASH showing Hemgenix’s sustained efficacy in hemophilia B patients, with a 90% reduction in bleeding.
Key point: The therapy continues to show durability, with factor IX activity sustained at 36% over five years.
Implication: Hemgenix offers long-term benefits for hemophilia B patients, reducing the need for continuous treatment.
💡 SCTbio and Fortrea Collaborate to Advance Gene Therapies [8] [EU • 04 Dec 2025]
Context: SCTbio and Fortrea announced a partnership to streamline the development of advanced cell and gene therapies.
Key point: This collaboration will enhance manufacturing and clinical trial logistics for gene therapies.
Implication: The partnership aims to reduce development timelines, improving access to cutting-edge therapies.
🧬 ETX101 Gene Therapy Shows Promise for Dravet Syndrome [9] [US • 05 Dec 2025]
Context: ETX101, a gene therapy for Dravet syndrome, showed a 78% median reduction in seizures and significant neurodevelopmental improvements in children.
Key point: The therapy demonstrated efficacy in reducing seizures and improving cognitive skills, especially in younger patients.
Implication: ETX101 offers potential as a disease-modifying therapy for Dravet syndrome, with further data expected in 2026.
💉 BE-CAR7 Achieves World-First Remission in T-cell Leukaemia [10] [EU • 10 Dec 2025]
Context: BE-CAR7, a base-edited CAR-T therapy, achieved remission in aggressive T-cell leukaemia, with 64% of patients remaining disease-free.
Key point: The therapy shows transformative potential for treating T-cell leukaemia, with two-thirds of patients now in remission.
Implication: BE-CAR7 could revolutionize treatment for previously incurable T-cell leukaemia, offering hope to patients who had no other options.
🩸 Vertex’s CASGEVY Shows Promise for Younger Sickle Cell Patients [11] [US • 06 Dec 2025]
Context: CASGEVY’s data in children aged 5-11 years with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia shows durable benefits.
Key point: The therapy achieved high rates of disease-free periods and transfusion independence in younger patients.
Implication: CASGEVY could significantly improve outcomes for younger patients with SCD and thalassemia, with regulatory filings planned for 2026.
Why It Matters
- Regulatory shifts such as the FDA’s new CAR-T approval policies will increase rigor in clinical trial designs, potentially slowing the approval process for new cancer therapies.
- Advancements in gene therapies for conditions like Dravet syndrome and hemophilia B are providing long-term, life-changing solutions, shifting the treatment landscape for rare diseases.
- Strategic collaborations in cell and gene therapy development are essential for accelerating the availability of transformative therapies for rare diseases and cancers.
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FAQ
What is the significance of Omisirge’s approval? [1]
Omisirge offers a novel cell therapy option for SAA, providing rapid recovery and transfusion independence, especially for patients without a matched donor.
How does Breyanzi’s approval impact MZL patients? [2]
Breyanzi provides the first CAR-T therapy for MZL, with exceptional response rates, offering new hope for patients with this rare cancer.
What are the potential benefits of ZEVASKYN for RDEB patients? [3]
ZEVASKYN offers a groundbreaking autologous gene therapy for RDEB, treating a debilitating skin condition with potential for long-lasting results.
What does the FDA’s CAR-T policy change mean for developers? [4]
The FDA’s policy change will require developers to conduct randomized superiority trials, raising the bar for future CAR-T approvals.
What does the approval of Waskyra mean for WAS patients? [5]
Waskyra offers a new gene therapy for Wiskott-Aldrich syndrome, significantly reducing severe infections and bleeding.
What challenges does uniQure face with AMT-130? [6]
UniQure is facing a challenging regulatory path after the FDA questioned the adequacy of data from its Phase I/II trials for Huntington’s disease.
How does CSL’s Hemgenix benefit hemophilia B patients? [7]
Hemgenix provides long-term efficacy in hemophilia B, reducing bleeding and eliminating the need for continuous prophylactic treatment.
What is the impact of the SCTbio-Fortrea collaboration? [8]
This collaboration aims to streamline the development and delivery of cell and gene therapies, accelerating the availability of novel treatments.
What are the early results of ETX101 in Dravet syndrome? [9]
ETX101 shows promise as a one-time, disease-modifying therapy, significantly reducing seizures and improving cognitive skills in young children.
How has BE-CAR7 advanced leukaemia treatment? [10]
BE-CAR7 offers a new hope for T-cell leukaemia patients, achieving remission in most treated patients, including those previously considered incurable.
What new data did Vertex present on CASGEVY? [11]
CASGEVY shows durable clinical benefits in younger patients with SCD and thalassemia, offering a transformative treatment option with regulatory filings planned for 2026.
Entities / Keywords
Omisirge; SAA; CAR-T therapy; Breyanzi; MZL; ZEVASKYN; RDEB; Waskyra; WAS; AMT-130; Huntington’s disease; Hemgenix; CSL Behring; SCTbio; Fortrea; ETX101; Dravet syndrome; BE-CAR7; T-cell leukaemia; CASGEVY; Vertex Pharmaceuticals.
References
- https://www.accessnewswire.com/newsroom/en/biotechnology/fda-approves-omisirger-as-first-approved-cell-therapy-to-treat-severe-aplastic-anemia-1115210
- https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibbs-Breyanzi-Approved-by-the-U-S–FDA-as-the-First-and-Only-CAR-T-Cell-Therapy-for-Adults-with-Relapsed-or-Refractory-Marginal-Zone-Lymphoma-MZL/default.aspx
- https://fox59.com/business/press-releases/globenewswire/9598015/abeona-therapeutics-announces-first-patient-treatment-with-zevaskyn-gene-therapy/
- https://www.fiercebiotech.com/biotech/fda-steers-cancer-car-t-developers-toward-randomized-superiority-trials-policy-shift
- https://www.reuters.com/business/healthcare-pharmaceuticals/fda-approves-first-gene-therapy-rare-immune-disorder-2025-12-09/
- https://www.biospace.com/fda/fda-minutes-confirm-challenging-path-ahead-for-uniqures-huntingtons-gene-therapy
- https://www.fiercepharma.com/pharma/ash-csl-proves-hemophilia-b-gene-therapy-hemgenix-holds-after-5-years-post-dosing
- https://www.globenewswire.com/news-release/2025/12/04/3199647/0/en/SCTbio-and-Fortrea-Forge-Strategic-Collaboration-to-Accelerate-Cell-and-Gene-Therapy-Development-and-Delivery.html
- https://www.businesswire.com/news/home/20251205992024/en/Encoded-Therapeutics-Presents-Positive-Interim-Efficacy-Data-from-Initial-Dose-Levels-of-Phase-12-Trials-Evaluating-ETX101-Gene-Therapy-in-Dravet-Syndrome
- https://www.openaccessgovernment.org/gene-therapy-achieves-world-first-remission-of-incurable-leukaemia/202283/
- https://investors.vrtx.com/news-releases/news-release-details/vertex-presents-new-data-casgevyr-including-first-ever-data