AVB-101 / AviadoBio - LARVOL DELTA

AviadoBio Announces Completion of Second Cohort in Phase 1/2 ASPIRE-FTD Clinical Trial Studying AVB-101 for FTD-GRN (Businesswire) - "AviadoBio...announced the completion of the second dose cohort in its Phase 1/2 ASPIRE-FTD clinical trial. ASPIRE-FTD is evaluating multiple doses of AviadoBio’s investigational gene therapy, AVB-101, in people living with frontotemporal dementia (FTD) with GRN gene mutations (FTD-GRN). The company intends to initiate dosing for a third cohort in Q3 2025 and expects to share early biomarker data in 2026. The announcement was made at the Association for Frontotemporal Degeneration (AFTD) annual caregiver conference in Denver."

P1/2 data • Trial status • Dementia

Direct image-guided convective perfusion of the bilateral thalami offers a consistent approach to CNS dosing: first-in-human experience with gene therapy for frontotemporal dementia. (ASGCT 2025) - P1/2 | "The current study exploits the properties of CED to deliver AVB-101 (recombinant adeno-associated virus serotype 9 (AAV9) encoding the human granulin ( GRN ) gene) to treat frontotemporal dementia (FTD) associated with progranulin deficiency... First-in-human experience with real-time MR-guided convective delivery provides new insights into the consistency of thalamic gene therapy perfusion properties. The isotropic tissue composition, low propensity for perivascular leakage and its broad interconnectedness makes the thalamus an ideal gene therapy target for a wide variety of neurologic disorders. Disease Focus of Abstract:Other Other: Frontotemporal Dementia"

Gene therapy • P1 data • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Gene Therapies • GRN

Development of an in vitro potency assay for AVB-101, a recombinant AAV9 gene therapy for the treatment of frontotemporal dementia with progranulin mutations (ESGCT 2024) - No abstract available

Gene therapy • Preclinical • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Gene Therapies

Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications (PRNewswire) - "AviadoBio Ltd...and Astellas Pharma Inc...today announced an exclusive option and license agreement for AVB-101...Under the terms of the agreement, Astellas will have the option to receive a worldwide exclusive license for the development and commercialization rights to AVB-101 in FTD-GRN and other potential indications. Astellas will make a $20 million equity investment and up to $30 million in upfront payments for the option to license AVB-101. AviadoBio is also eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties if Astellas exercises its option."

Licensing / partnership • CNS Disorders • Dementia

ASPIRE-FTD Phase 1/2 Clinical Trial Opens in the U.S. at The Ohio State University Wexner Medical Center (Businesswire) - "AviadoBio...today announced that its Phase 1/2 ASPIRE-FTD clinical trial is now open and recruiting patients in the United States at The Ohio State University. ASPIRE-FTD is evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). The study is also recruiting in Europe with study sites in the Netherlands, Poland, and Spain, with additional sites expected to open in multiple countries."

Enrollment open • CNS Disorders • Dementia

Pre-clinical development of AVB-101, an AAV gene therapy treatment for frontotemporal dementia with progranulin mutations (AAIC 2024) - "The preclinical data suggest that intrathalamic infusion of AVB-101 constitutes a novel and promising approach to supplement PGRN in the CNS, supporting the clinical development of AVB-101 for FTD-GRN."

Gene therapy • Preclinical • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Gene Therapies • GRN • TARDBP

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations (Businesswire) - "AviadoBio...today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States."

Trial status • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

ClearPoint Neuro congratulates partner AviadoBio on the first patient treated in its ASPIRE-FTD clinical trial evaluating AVB-101 in people with frontotemporal dementia with mutations in the GRN gene (GlobeNewswire) - "ClearPoint Neuro, Inc...congratulates its partner AviadoBio for treating its first patient in the Phase 1/2 ASPIRE-FTD clinical trial evaluating its investigational gene therapy AVB-101 in people with frontotemporal dementia (FTD) with mutations in the progranulin gene (GRN)."

Trial status • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

AviadoBio Announces FDA IND Clearance and Fast Track Designation for AVB-101 for the Treatment of Frontotemporal Dementia with Progranulin (GRN) Mutations (Businesswire) - "AviadoBio...today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AVB-101 to be studied in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene. In addition, the FDA has granted Fast Track designation to AVB-101 for the treatment of FTD-GRN to slow disease progression."

Fast track designation • IND • CNS Disorders • Dementia

AviadoBio Announces Presentation of Promising Preclinical Safety and Biodistribution Data of its Gene Therapy Candidate AVB-101 for FTD-GRN at the Annual European Society of Gene and Cell Therapy Congress (BioSpace) - "AviadoBio...announced today the presentation of preclinical safety and biodistribution data on AVB-101 at the 30th annual European Society of Gene and Cell Therapy (ESGCT) Congress in Brussels....AVB-101 delivered by intrathalamic infusion constitutes a novel and promising approach to address unmet medical needs in FTD-GRN, with the Phase 1/2 clinical trial (ASPIRE-FTD) currently open in Europe. Intrathalamic delivery of AVB-101 was well tolerated at all doses tested, with no mortality or observable clinical adverse events seen. Results showed that human progranulin (hPGRN) was most abundant in the thalamus, a key hub for biodistribution in the brain, but also detected throughout the brain. AVB-101 increased hPGRN levels in the temporal and frontal lobes, the cortical regions most severely affected by FTD-GRN."

Preclinical • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

AviadoBio Announces Initiation of Phase 1/2 Clinical Trial, ASPIRE-FTD, Evaluating AVB-101 in People with Frontotemporal Dementia with GRN Mutations (Businesswire) - "AviadoBio...today announced that it has initiated the Phase 1/2 ASPIRE-FTD clinical study of its investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with mutations in the progranulin (GRN) gene. ASPIRE-FTD is an open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, AVB-101 will be delivered as a one-time treatment into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States....Preclinical safety and biodistribution data following intrathalamic delivery to non-human primates demonstrate good tolerability and widespread progranulin expression in brain tissues. These data will be presented at the 30th Annual European Society of Gene & Cell Therapy (ESGCT) meeting in Brussels, Belgium Oct. 24-27, 2023."

Preclinical • Trial status • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration

ASPIRE-FTD: A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN) (clinicaltrials.gov) - P1/2 | N=9 | Recruiting | Sponsor: AviadoBio Ltd

Gene therapy • New P1/2 trial • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Gene Therapies • GRN

Intra-thalamic delivery of AVB.PGRN rescues pathology in Grn null mice and achieves widespread cortical expression in a large animal model (ESGCT 2022) - No abstract available

Preclinical

Intrathalamic Delivery of AVB.PGRN Rescues Pathology in Grn Null Mice and Achieves Widespread Cortical Expression in a Large Animal Model without Expression in the Liver (ASGCT 2022) - "Intra-thalamic delivery of AVB.PGRN rescues pathology observed in the Grn-/- mouse model and achieves widespread PGRN expression in the cortex of a large mammal with no PGRN expression outside the CNS. Taken together, the data suggest that AVB.PGRN delivered by ITM injection constitutes a novel and promising approach to address areas of current medical need in FTD."

Preclinical • Alzheimer's Disease • CNS Disorders • Dementia • Frontotemporal Lobar Degeneration • Immunology • TARDBP

AviadoBio’s Gene Therapy Candidate Receives Orphan Designation from Food and Drug Administration and European Commission in Frontotemporal Dementia (GlobeNewswire) - “AviadoBio…announced today that both the U.S. Food and Drug Administration (FDA) and the European Commission (EC) have granted orphan designation to AVB-101, a one-time gene therapy for the treatment of frontotemporal dementia (FTD)….‘We look forward to initiating clinical trials for AVB-101 later in 2022…’”

European regulatory • New trial • Orphan drug • CNS Disorders • Frontotemporal Lobar Degeneration