Tauopathies are neurodegenerative diseases characterized by pathological tau protein accumulation. Though therapies involving monoclonal antibodies and small-molecule inhibitors have progressed, they have so far failed in multiple clinical trials, underscoring the need for innovative molecular approaches. RNA-based therapies offer an alternative disease-modifying approach by being able to target tau at its molecular origin. Diverse modalities, such as mRNA, ASO, RNAi, and SSO, offer distinct promises. Though their challenges are equally diverse, they also share common problems. This review examines the nascent field of RNA therapeutics for tauopathies, outlining emerging modalities, translational barriers, molecular targets, clinical trials, and patent trends.

We are pleased to share that we will be presenting a poster at Tau Global Conference. This work showcases our progress on developing an AI-native target probe engine, starting with some of the most challenging CNS targets. It's an exciting step toward our platform built for the targets that matter most. We look forward to discussing the work with the community and welcome questions during the poster session.

Nanil Therapeutics is invited to the  Goodwin + KPMG 7th Annual Symposium at the 2026 JP Morgan Healthcare Conference in San Francisco. We are honored to be invited to the Goodwin Symposium at the J.P. Morgan Healthcare Conference in San Francisco in January 2026. This premier event brings together the life sciences and healthcare innovation community to explore emerging trends, investment opportunities, and transformative technologies shaping the future of health. We look forward to engaging with industry leaders, investors, and partners at this influential gathering.

Quantum computing is offering a new window into the molecular forces behind dementia. We are the first to apply quantum computing to a disease-relevant tau peptide fragment, directly motivated by tauopathy. By benchmarking quantum electronic energies against classical molecular dynamics and Hartree-Fock calculations, we reveal energetic signatures that traditional simulations miss. Our controlled, fragment-based workflow sets the first benchmark framework for integrating quantum electronic structure with classical simulation in peptide biophysics. These results show that quantum computing provides complementary insight, not redundancy, when studying protein energetics. Together, they mark a foundational step toward quantum-enhanced molecular understanding in proteinopathy research.

Emerging generative models for biology focus on DNA, non-coding RNA, or proteins, ignoring information hidden in mRNA. Additionally, in protein engineering and mRNA therapeutics the design of mRNA sequences is still a challenge, lacking a clear framework. Here, we introduce and rigorously evaluate two novel methods: a foundational model for mRNA and a reinforcement learning mRNA design framework built on such a model. codonGPT is the first generative foundational language model trained directly on coding mRNA sequences. To solve the problem of synonymous constraints that are only unique to mRNA, we introduce a novel method of inference-time masking, along with house-keeping genes evaluation. For the first time, we also rigorously demonstrate, that for precise mRNA therapeutics design, reinforcement learning on such a model provides a clear framework for biological optimization. Our study introduces a novel foundational model for mRNA and a new reinforcement learning based paradigm for mRNA sequence design.

Our abstract on a next-generation neurotherapeutic platform integrating predictive modeling with translational neuroscience has been accepted for presentation at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference 2025 in San Diego. This work highlights our progress in developing tools that bridge molecular discovery with therapeutic design for Alzheimer’s and related disorders. Our team will be attending to present these advances and engage with global leaders in neurodegeneration research.

We are excited to share that Nanil will be attending the Alzheimer’s Association International Conference (AAIC) 2025 in Toronto this July. As the world’s largest forum for dementia research, AAIC is an incredible opportunity to connect with leading scientists, clinicians, and innovators who share our passion for advancing dementia research and care. We are especially looking forward to engaging with new discoveries in biomarker science and therapeutic development, areas close to our mission at Nanil. It’s always inspiring to be part of a community pushing the boundaries of what's possible in neurodegeneration, and we can’t wait to be there in person this year. See you in Toronto!

Emerging generative models for biology focus on DNA, non-coding RNA, or proteins, ignoring information hidden in mRNA. Additionally, in protein engineering and mRNA therapeutics the design of mRNA sequences is still a challenge, lacking a clear framework. Here, we introduce and rigorously evaluate two novel methods: a foundational model for mRNA and a reinforcement learning mRNA design framework built on such a model. codonGPT is the first generative foundational language model trained directly on coding mRNA sequences. To solve the problem of synonymous constraints that are only unique to mRNA, we introduce a novel method of inference-time masking, along with house-keeping genes evaluation. For the first time, we also rigorously demonstrate, that for precise mRNA therapeutics design, reinforcement learning on such a model provides a clear framework for biological optimization. Our study introduces a novel foundational model for mRNA and a new reinforcement learning based paradigm for mRNA sequence design.

• Problem: CNS delivery of biologics is hindered by the BBB. Only a few peptides (e.g., Angiopep-2, TAT) are known to mediate transport, and they suffer from issues like off-target effects, instability, and poor tunability.
  
  

• Gap: Current BBB models focus on small molecules. No model exists for systematically discovering or screening BBB-penetrant peptides/domains in proteins or biologics.
  
  

• Contribution: We present the first transformer-based BBB embedding and scoring model, capable of discovering novel shuttle domains and guiding CNS biologic design.

• 1. The model offers a first-in-class in silico screening tool for BBB shuttle peptides and protein domains

• 2. Uncovers both known and novel motifs associated with transcytosis

• 3. Interpretability reveals biophysically meaningful features (e.g., basic patching, structural motifs)

• 4. Bridges the gap between sequence-level design and CNS-targeted biologics

• 5. Extensible to multi-objective optimization (e.g., stability, immunogenicity)

Nanil Therapeutics was invited to the  Goodwin + KPMG 6th Annual Symposium at the 2025 JP Morgan Healthcare Conference in San Francisco. We had a fantastic time attending the event and learning from our more established peers. The event brought together an inspiring mix of healthcare leaders, innovators, and investors, and offered valuable insights into the future of biotech, medtech, and digital health. From thought-provoking panels to meaningful conversations during the networking sessions, the Symposium was a highlight of the week. We're grateful to Goodwin and KPMG for hosting such a thoughtful and energizing gathering, and we look forward to staying connected with the incredible community that came together in San Francisco.