VTx-002 / VectorY - LARVOL DELTA

A scalable and quality driven insect cell–baculovirus manufacturing platform enables 1000L production of VTx-002, an AAV based vectorized antibody for ALS (ESGCT 2026) - No abstract available

From Single-Cell Analysis to Clinical Dose: Setting the Therapeutic Threshold for VTx-002 in ALS Motor Neurons (ESGCT 2026) - No abstract available

Clinical

Leveraging Analytical Characterization to Drive Scalable GMP Manufacturing and Regulatory Advancement of VTx-002, an AAV-based vectorized antibody for ALS (ESGCT 2026) - No abstract available

No DRG toxicity in AAV5-vectorized antibody therapy after intracisternal magna administration in nonhuman primates (ASGCT 2026) - "Transient mild increases in CSF NfL after AAV5 infusion rapidly declined to levels comparable to vehicle within 6 months and remained lower than levels seen with AAV9-based therapies, indicating a favorable safety profile. Conclusion These GLP-compliant data support that VTx-002, an AAV5-vectorized antibody therapy, efficiently and selectively transduces motor neurons within the CNS of NHPs and exhibits a favorable safety profile, demonstrating no significant adverse findings, hepatotoxicity, or DRG pathology, thereby supporting VTx-002 advancement to clinical development."

CNS Disorders • Gene Therapies • CSF NfL • NEFL • TARDBP

From Single-Cell Analysis to Clinical Dose: Setting the Therapeutic Threshold for VTx-002 in ALS Motor Neurons (ASGCT 2026) - "This supports the rationale for selecting safe and effective dosing regimens for future clinical trials. Overall, this integrative approach offers a rational and systematic framework for translating gene therapy doses to clinical applications in ALS and other neurodegenerative disorders."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • Respiratory Diseases • CHAT • TARDBP

Early CMC investment enables scalable and commercially viable manufacturing of VTx-002, a first-in-class AAV-based vectorized antibody for treatment of ALS (ASGCT 2026) - "By enabling large-scale, high-yield AAV production with consistent quality, this manufacturing strategy expands the scope of gene therapy beyond rare diseases to indications to those with larger patient populations and higher dosing requirements. Such an approach supports the broader application of AAV-based therapies to neurodegenerative diseases and other conditions where both clinical impact and sustainable manufacturing at scale are essential."

Amyotrophic Lateral Sclerosis • CNS Disorders • Gene Therapies • Infectious Disease • Rare Diseases • TARDBP

Integrated vector and process design enables scalable manufacturing of VTx-002, a first-in-class AAV-based vectorized antibody targeting TDP-43 in ALS (ASGCT 2026) - "Ultimately, we demonstrated robust scale-up performance and strong batch comparability from 2 L research-scale production through 1000 L GMP clinical manufacturing. Conclusion In conclusion, VectorY developed a manufacturing platform that secures excellent quality, consistency and scale-up which support clinical development of its pipeline products."

CNS Disorders • Infectious Disease • TARDBP

VectorY Therapeutics Receives U.K. MHRA Authorization and EMA Approval to Initiate Phase 1/2 PIONEER-ALS Clinical Trial of VTx-002 in People Living with Amyotrophic Lateral Sclerosis (ALS) (Businesswire) - "PIONEER-ALS is a multicenter, open label, dose-escalation Phase 1/2 clinical study that evaluates two dose levels of VTx-002 and is expected to enroll 12 adults with ALS across sites in the U.S., Europe and the U.K. The primary objective of this study is to evaluate VTx-002 safety, tolerability, pharmacokinetics, and exploratory efficacy in people living with ALS."

First-in-human • Trial status • Amyotrophic Lateral Sclerosis

VTx-002-01-001: Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Vector Y Therapeutics

First-in-human • Trial initiation date • Amyotrophic Lateral Sclerosis • CNS Disorders • FUS • SOD1

VTx-002-01-001: A Phase 1/2 Study of the Safety and Tolerability of ICM VTx-002 in participants with ALS (clinicaltrialsregister.eu) - P1/2 | N=2 | Not yet recruiting | Sponsor: VectorY Therapeutics B.V.

New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders • FUS

VectorY Therapeutics Announces First Participant Dosed in Phase 1/2 PIONEER-ALS Clinical Trial of VTx-002 in People with Amyotrophic Lateral Sclerosis (ALS) (Businesswire) - "The participant was treated at the lead clinical trial site, the Sean M. Healey & AMG Center for ALS at Mass General Brigham, a premier treatment center for neurodegenerative diseases and pioneering research....PIONEER-ALS is a multicenter, open label, dose-escalation Phase 1/2 clinical study that will evaluate two dose levels of VTx-002 and is expected to enroll 12 adults with ALS across sites in the U.S., Europe and the U.K....VectorY Therapeutics expects to continue enrolling participants in the PIONEER-ALS trial across multiple clinical sites."

Trial status • Amyotrophic Lateral Sclerosis

VTx-002-01-001: Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS (clinicaltrials.gov) - P1/2 | N=12 | Recruiting | Sponsor: Vector Y Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • First-in-human • Amyotrophic Lateral Sclerosis • CNS Disorders • FUS • SOD1

VectorY Therapeutics Receives FDA Fast Track Designation for VTx-002, a First-in-Class Vectorized Antibody Targeting Underlying Disease Biology in ALS (Businesswire) - "In December 2025, the Company announced that the FDA cleared its Investigational New Drug (IND) application for VTx-002 for the treatment of ALS and concurrently announced plans to initiate PIONEER-ALS, a Phase 1/2 clinical study designed to evaluate the safety, tolerability, and pharmacodynamic activity of VTx-002 in people living with ALS. VectorY intends to enroll its first patient in the PIONEER-ALS study in early 2026."

Fast track • IND • Trial status • Amyotrophic Lateral Sclerosis

Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS (clinicaltrials.gov) - P1/2 | N=12 | Not yet recruiting | Sponsor: Vector Y Therapeutics

New P1/2 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

VectorY Therapeutics Receives FDA Clearance of IND to Proceed with the PIONEER-ALS Phase 1/2 Trial of VTx-002, a First-in-Class Vectorized Antibody Targeting TDP-43 Pathology in ALS (Businesswire) - "PIONEER-ALS, an open label, dose-escalation Phase 1/2 clinical study, will evaluate two dose levels of VTx-002 and is expected to enroll 12 adults with ALS. The primary objective of this study is to evaluate VTx-002 safety and tolerability."

IND • New P1/2 trial • Amyotrophic Lateral Sclerosis

Non-clinical development and phase 1/2 clinical trial design of VTx-002, an AAV5.2 vectorized intrabody targeting TDP-43 for amyotrophic lateral sclerosis (ALS-MND 2025) - "Collectively, our non-clinical findings suggest that AAV-mediated intrabody delivery targeting TDP-43 is a viable therapeutic strategy for ALS paving the way for clinical development in ALS and other TDP-43-related disorders. phase 1/2 clinical trial is planned to be an open-label, dose-escalating study in early-stage ALS patients. The primary objective of the study is safety and tolerability."

Clinical • P1/2 data • Amyotrophic Lateral Sclerosis • Anesthesia • CNS Disorders • FUS • SOD1 • TARDBP

UMC Utrecht and VectorY Therapeutics collaborate in ALS Biomarker Study (Businesswire) - "UMC Utrecht has enrolled its first patient for a new study to identify biomarkers of Amyotrophic Lateral Sclerosis (ALS). The observational investigator-initiated study is led by Professor Dr. Leonard van den Berg and supported by VectorY Therapeutics....The new longitudinal study announced today aims to identify blood or cerebrospinal fluid (CSF) biomarkers of ALS patients. Finding these biomarkers is expected to help diagnose ALS and monitor disease progression and improve the design of future clinical studies. It is also anticipated that biomarkers will provide insights into ALS pathogenesis, supporting the development of new therapeutics....The study aims to enroll 70 ALS patients in the Netherlands."

Enrollment status • Licensing / partnership • New trial • Amyotrophic Lateral Sclerosis • CNS Disorders

VectorY Raises €129 Million ($138 Million) Series A Financing to Advance Vectorized Antibody Programs in Neurodegenerative Diseases (Yahoo Finance) - "VectorY Therapeutics...today announced the close of a €129 million ($138 million) Series A financing to advance its vectorized antibody programs in neurodegenerative diseases. The round was co-led by EQT Life Sciences and the Forbion Growth Opportunities Fund....VectorY will use the proceeds to support the clinical development of VTx-002, its lead vectorized antibody program targeting TDP-43 for the treatment of ALS. The Company will also accelerate the development of its vectorized antibody platform and additional pipeline programs targeting proteinopathies causing other neurodegenerative diseases."

Financing • Amyotrophic Lateral Sclerosis • CNS Disorders