Australia & New Zealand LRA Watchdog Update—December 2025

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Tongted Das, PhD
Monash Health
Australia

Jessica Sue, BSc, MHSM
Junior Associate Editor, ISCT Telegraft
Sydney Cord Blood Bank
Sydney Children's Hospital Network
Australia

ISCT Representation in Recent Regulatory Work (Australia)

The ISCT committee participated in the following recent public consultations:

Proposed TGA Annotations to ICH E6(R3) Guideline for Good Clinical Practice (GCP): Principles and Annex I and 12-month Transition Period. The ISCT’s comments were submitted to the TGA on October 8th, 2025.
TGA Proposed Reforms to Implement a Biological Groups Order. Comments were submitted on November 20th, 2025.

AOTEAROA – NEW ZEALAND

Medsafe

Medsafe Regulatory has no updates on cell & gene therapy in the 4th quarter of 2025. The Medicines Amendment Bill and its verification pathway (under Medsafe) had been introduced to the House of Representatives since 31 Mar 2025. Medsafe may accelerate the approval of treatments that are already approved by two recognised overseas regulatory authorities¹.

The New Zealand government is also updating the Gene Technology Bill, to establish a dedicated regulator and enable safe use of gene technology (such as genetic engineering or genetic modification). The Bill is now going through the parliamentary process² (updated 12 Nov 2025).

Ministry of Health (New Zealand) official’s briefing³ (released 15 Sep 2025) indicated decision to extend the current regulation of xenotransplantation procedures via Order in Council under the Medicines Act 1981 Part 7A to 30 Sep 2030. While the Bill is a regenerative-medicine area and not strictly gene therapy, it is indicating regulatory movement around advanced therapies.

Australia

Therapeutic Goods Administration (TGA)

The Therapeutic Goods Administration (TGA) is addressing the increase in unlawful advertising on social media and has updated its Advertising therapeutic goods on social media guidance⁴ to help advertisers to understand their responsibilities when promoting therapeutic goods via these platforms, which must comply with regulatory requirements under the Therapeutic Goods Act 1989 (the Act).

TGA consultation (14 Oct 2025 - 11 Nov 2025) sought stakeholder feedback on proposed legislative amendments clarifying submission and fee processes of the Technical Master File (TMF) and Type II Plasma Master File (PMF) for blood/plasma manufacturers⁵. These changes may affect manufacturing documentation and regulatory processes relevant to biological and some cell therapy supply chains.

AusBiotech and Medicines Australia

On 1 Oct 2025, AusBiotech and Medicines Australia launched an Advanced Therapies Advisory Group to provide strategic input on the advanced-therapies ecosystem (policy, reimbursement, manufacturing) to the industry sector⁶. While this group is not a regulator, it will influence the stakeholder/regulatory boundary and guidance significances.

Office of the Gene Technology Regulator

The Office of the Gene Technology Regulator (OGTR) has published its Annual Report (1 Jul 2024 – 30 Jun 2025) on 30 Sep 2025, highlighting licensing activities, inspections and regulatory developments relevant to gene-modification, with some notable efforts were the consultation on draft amendments to the Gene Technology Act 2000, hosting the 10th National Institutional Biosafety Committee Forum (IBC Forum), and the collaboration initiatives between Australia and New Zealand regulatory frameworks for genetically modified organisms (GMOs)⁷.

International Regulatory Updates Relevant to Australia & New Zealand

United States Food & Drug Administration (FDA)

On October 15th, 2025, the biopharmaceutical company Tr1X announced that the FDA cleared their IND application for TRX319 for the treatment of progressive multiple sclerosis. TRZ319 is an allogeneic, off-the-shelf CAR-Tr1 Treg cell therapy designed to pair targeted B-cell control with active anti-inflammatory signaling and T-cell regulation, in order to restore immune balance. A multi-center open-label, dose-escalation Phase 1/2a study will evaluate safety and tolerability⁸.

On November 14th, 2025, the FDA announced approval of new labeling, submitted by Sarepta Therapeutics, to include a Boxed Warning for Elevidys (delandistrogene moxeparvovec-rokl). Used to treat patients with Duchenne muscular dystrophy (DMD), the decision follows cases of fatal acute liver failure in non-ambulatory patients. The indication will be limited to ambulatory patients four years or older with confirmed mutation in the DMD gene⁹.

Orca Bio announced on October 6th, 2025 that the FDA has accepted their BLA for Orca-T for Priority Review. Orca-T is an investigational T-cell immunotherapy for the treatment of various hematological malignancies including Acute Myelogenous Leukemia, Acute Lympoblastic Leukemia and Myelodysplastic Syndrome, composed of highly purified regulatory T-cells and hematopoietic stem cells from peripheral blood from matched donors, and aims to improve the graft-versus-leukemia affect whilst minimizing graft-versus-host disease. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation¹⁰.

Umoja Biopharma announced on September 30th, 2025 that the FDA had granted Fast Track designation to UB-VV111, an investigational off-the-shelf product that generates CD19-directed CAR-T cells in-vivo, in patients with relapsed/refractory large B-cell lymphoma and CLL. UB-VV111 was the first in vivo CAR-T cell therapy to receive IND clearance by the FDA back in 2024¹¹.

The FDA placed two gene therapy clinical trials on hold: the phase-3 MAGNITUDE and MAGNITUDE-2 trials, which are evaluating nex-z, an investigational CRISPR based therapy for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN). The hold on these trials, conducted by Intellia Therapeutics, follows hospitalization of a patient with grade 4 liver transaminase elevations¹².

European Medicines Agency (EMA)

Following a clinical development program encompassing two clinical trials and an expanded access program, the EMA has recommended granting marketing authorization for Waskyra (etuvetidigene autotemcel), to treat patients older than 6 months of age with Wiskott-Aldrich syndrome (WAS) who have a mutation in the WAS gene. The applicant was Fondazione Telethon ETS. The treatment is for patients for which a stem cell transplant is appropriate but cannot find a match. Waskyra is developed by collecting the patients CD34+ cells which are then genetically modified to be able to produce a functional WAS protein and is given via intravenous infusion. The process was via the EMA pilot scheme by which non-profit and academic developers of ATMPs addressing unmet medical needs are given enhanced support by the EMA in navigating regulatory requirements¹³.

Foundation for the Accreditation of Cellular Therapy (FACT)

On November 10th, 2025, FACT published the first edition of the Standards for Immune Effector Cells in the Community Clinical Setting. These standards are designed to assist community oncology programs delivering cell therapies to patients near where they live and work and respond to the demand for clear and consistent information around the use of cell therapies¹⁴.

Also in November 2025, FACT published the 9th edition of the Hematopoietic Cellular Therapy (HCT) Standards and the 3rd edition of the Immune Effector Cell (IEC) Standards. A key feature of the updated standards is harmonization of language across all standards. In the HCT standards, there is now a single collection section covering different collection types¹⁵.

World Cord Blood Day 2025

World Cord Blood Day was held on November 17th, 2025. This year, the combination of early newborn screening for Krabbe disease combined with cord blood transplantation soon after birth and the potential use of cord blood for cerebral palsy was a focus¹⁶.

BioPhorum

Following their survey on biosafety in GMP environments, which showed variation in how it is managed across different organizations, BioPhorum have published a paper outlining a structured, risk-based approach around incorporation of biosafety in facilities manufacturing ATMPs¹⁷.

ISCT Representation in Recent Regulatory Work (International)

ISCT provided comments on the following consultation papers:

FDA recommendations to develop guidance on leveraging prior knowledge and experience to facilitate product development and application review. Comments were submitted on October 14th, 2025.
ICH (E20) guideline on Adaptive Designs for Clinical Trials.
ISCT was also invited to the WHO 81st Expert Committee meeting on Biological Standardization (ECBS) in Tunis on October 13th, 2025.