DelveInsight’s “Frontotemporal Dementia (FTD) – Pipeline Insight, 2025” delivers an in-depth analysis of the FTD drug development landscape, spotlighting innovative assets, key clinical trial progress, and the emerging shift toward precision neurology. FTD encompasses a spectrum of disorders, including behavioral variant FTD (bvFTD) and primary progressive aphasia (PPA), with significant genetic overlap with ALS and other neurodegenerative diseases—fueling cross-indication drug development.
Key late-stage programs include Alector’s AL001 (INFRONT-3 trial), a monoclonal antibody designed to restore progranulin levels in GRN mutation carriers. Denali Therapeutics, Passage Bio, and Sangamo Therapeutics are also pursuing gene therapy and enzyme replacement strategies aimed at halting or reversing disease progression.
The pipeline is increasingly supported by biomarker-driven trial designs, leveraging tools like neurofilament light chain (NfL), tau PET imaging, and fluid biomarkers to measure drug response and enable earlier diagnosis. Regulatory bodies are also showing flexibility through orphan drug designations and accelerated pathways for genetically defined subgroups.
As 2025 unfolds, the FTD pipeline signals a new era of genetically targeted and pathophysiology-specific treatments. With increased investment, deeper molecular understanding, and strong collaboration among academia, industry, and advocacy groups, the future of Frontotemporal Dementia drug development is shifting from symptom management to disease modification.
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Key Takeaways from the Frontotemporal Dementia Pipeline Report
• DelveInsight’s frontotemporal dementia pipeline analysis depicts a strong space with 15+ active players working to develop 20+ pipeline drugs for frontotemporal dementia treatment.
• The leading frontotemporal dementia companies include Transposon Therapeutics, Alector, Vesper Bio, AviadoBio, Passage Bio, Eli Lilly and Company, Coya Therapeutics, Neurimmune Therapeutics, Asceneuron SA, and others are evaluating their lead assets to improve the frontotemporal dementia treatment landscape.
• Key frontotemporal dementia pipeline therapies in various stages of development include TPN-101, AL001, AVB-101, VES001, PBFT02, GRN gene therapy, COYA 302, NI008, Research Program M1 PAM, and others.
• In June 2025, AviadoBio initiated patient dosing in multiple countries for AVB‑101’s Phase I/II ASPIRE‑FTD trial targeting genetic FTD; results are anticipated by 2026.
• In May 2025, Vesper Bio announced that it had achieved a significant enrollment milestone in the ongoing Phase Ib/IIa SORT-IN-2 study of VES001 for GRN-associated Frontotemporal Dementia.
• In May 2025, Passage Bio showcased process development data for a high-productivity, suspension-based manufacturing method for PBFT02 at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).
• In December 2024, CervoMed announced that its Phase IIb RewinD‑LB trial for dementia with Lewy bodies had failed to meet primary endpoints, but the drug remained well tolerated; discussions are underway to advance neflamapimod into FTD-focused trials in mid‑2025.
• In November 2024, the FDA granted Orphan Drug Designation to CervoMed’s neflamapimod, an oral p38 MAP kinase inhibitor, as a potential treatment for general forms of FTD.
Frontotemporal Dementia Overview
Frontotemporal Dementia (FTD) is a group of neurodegenerative disorders characterized by progressive damage to the frontal and/or temporal lobes of the brain, leading to changes in behavior, personality, language, and movement. Unlike Alzheimer’s disease, memory is often preserved in the early stages, but social conduct, emotional control, and language skills are significantly affected.
FTD typically occurs between the ages of 45 and 65, making it one of the most common causes of early-onset dementia. The condition can be sporadic or inherited, with genetic mutations such as MAPT, GRN, and C9orf72 commonly involved. There is currently no cure for FTD, and treatment focuses on managing symptoms through behavioral therapy, medications for mood and behavior, and supportive care. Research is ongoing into disease-modifying therapies targeting the underlying molecular causes.
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Frontotemporal Dementia Treatment Analysis: Drug Profile
AL001: Alector/GSK
AL001 is a recombinant human IgG1 monoclonal antibody developed by Alector, in collaboration with GSK, targeting sortilin (SORT1) to treat Frontotemporal Dementia (FTD). It works by inhibiting a key pathway responsible for progranulin degradation, thereby raising progranulin levels in patients. AL001 has received Orphan Drug, Fast Track, and Breakthrough Therapy designations from the FDA for FTD treatment. In July 2021, GSK partnered with Alector for the global development and commercialization of AL001 and AL101, both aimed at increasing progranulin levels. AL001 is currently in Phase III trials for FTD patients with GRN or C9orf72 mutations.
TPN-101: Transposon Therapeutics
TPN-101 is an investigational therapy that targets LINE-1 reverse transcriptase, an enzyme responsible for the replication of LINE-1 retrotransposons in the human genome. Dysregulation of LINE-1 activity can trigger innate immune responses, contributing to neurodegenerative and autoimmune diseases. By inhibiting LINE-1 reverse transcriptase, TPN-101 aims to address underlying disease mechanisms in Frontotemporal Dementia. The drug is currently in Phase II clinical development for FTD.
AVB-101: AviadoBio
AVB-101 is a gene therapy candidate designed to restore progranulin levels by delivering a functional GRN gene directly into the brain. It is administered via a minimally invasive stereotactic procedure targeting the thalamus, a brain region that connects to the frontal and temporal lobes—areas significantly affected in FTD. This targeted delivery is intended to cross the blood-brain barrier effectively, ensure localized treatment, and reduce systemic exposure. In November 2023, AVB-101 received Fast Track designation from the FDA and is currently in Phase I/II trials for the treatment of Frontotemporal Dementia.
Learn more about the novel and emerging frontotemporal dementia pipeline therapies.
Frontotemporal Dementia Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.
By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
By Route of Administration
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
By Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
Scope of the Frontotemporal Dementia Pipeline Report
• Coverage: Global
• Key Frontotemporal Dementia Companies: Transposon Therapeutics, Alector, Vesper Bio, AviadoBio, Passage Bio, Eli Lilly and Company, Coya Therapeutics, Neurimmune Therapeutics, Asceneuron SA, and others.
• Key Frontotemporal Dementia Pipeline Therapies: TPN-101, AL001, AVB-101, VES001, PBFT02, GRN gene therapy, COYA 302, NI008, Research Program M1 PAM, and others.
Explore detailed insights on drugs used in the treatment of frontotemporal dementia here.
Table of Contents
1. Introduction
2. Executive Summary
3. Frontotemporal Dementia Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Frontotemporal Dementia Pipeline Therapeutics
6. Frontotemporal Dementia Pipeline: Late-Stage Products (Phase III)
7. Frontotemporal Dementia Pipeline: Mid-Stage Products (Phase II)
8. Frontotemporal Dementia Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix
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