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Investing in Next-Generation Precision Therapeutics

Our pipeline reflects Dawn Therapeutics’ strategic progression from discovery to translational readiness. All programmes are currently in preclinical stages and will advance according to milestone-driven decision gates and regulatory planning.

Lead Programme — DTX-101 (MPS-I H, Hurler Syndrome)

Recent Milestones

Recent Milestones

Indication:

Hurler syndrome (MPS-I H), targeting both neurological and skeletal manifestations

Modality / Vector:

In vivo “stealth” lentiviral vector delivering functional IDUA gene

Status:

Preclinical optimization, toxicology, biodistribution, CMC development

Next Milestone:

IND-enabling studies in progress; regulatory consultations planned

Platform & Modality Programmes

While DAWN-01 is our lead translational candidate, we simultaneously develop platform technologies and modular payload approaches to enable broader applications. These are under early-stage validation.

Platform Cards — WP Perfect
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CNS Delivery Platform

Focus & Purpose

Enable targeted gene/RNA delivery to the brain across the blood–brain barrier.

Stage & Objective

Proof-of-concept in Alzheimer’s and other neurodegeneration models.

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Cartilage & Bone Delivery Platform

Focus & Purpose

Target genetic and degenerative disease of skeletal tissues.

Stage & Objective

Delivery optimization of payloads like IDUA mRNA/gene.

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LNP–mRNA Payloads

Focus & Purpose

Express therapeutic proteins or enzymes in localized tissues.

Stage & Objective

Biodistribution, expression durability, safety profiling.

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RNAi / ASO Payloads

Focus & Purpose

Suppress pathogenic genes in neurological or musculoskeletal disease.

Stage & Objective

Feasibility studies, dose–response evaluation.

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AAV Vector Designs

Focus & Purpose

Durable gene transfer in selected tissues.

Stage & Objective

Vector engineering, expression control, safety evaluation.

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Translational Tools & Assays

Focus & Purpose

Assay development to evaluate therapeutic performance across models.

Stage & Objective

Pharmacology, biodistribution, and tissue readouts.

Exploratory Discovery Projects

In parallel with our lead and platform programmes, we maintain a discovery-stage portfolio to identify new disease targets and payload combinations suited to our delivery systems. These are in earliest concept or pre-validation stages and are prioritized based on scientific rationale, unmet need, and translational feasibility.

Key Principles & Milestones

01

Go / No-Go gating

Each programme advances through staged decision points based on efficacy, safety, and manufacturability.

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02

Biomarker-guided decisions

Preclinical studies incorporate biomarker endpoints, functional readouts, and pharmacokinetics to guide dose and safety margins.

03

Translational planning

Payload design, delivery engineering, and manufacturing work are undertaken early to minimize gaps toward clinical readiness.

04

Regulatory alignment

We engage external experts and regulatory consultants to ensure the pipeline is structured for future regulatory submission.

05

Collaborative flexibility

Certain programmes may be licensed, co-developed, or spun out depending on strategic alignment.

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DISCLAIMER: All pipeline programmes are investigational and currently in preclinical development. Their inclusion here does not imply guaranteed progression to clinical trials or regulatory approval.
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