MPAN-C19orf12 Therapeutics Master
Novel approach to treating lysosomal storage diseases through targeted mRNA delivery systems. This project focuses on developing lipid nanoparticle formulations optimized for enzyme replacement therapy.
Duration
18 months
Team Size
12 members
Progress
67% Complete
Research Objectives
mRNA Design
Optimize codon usage and 5'/3' UTR sequences for enhanced protein expression
Formulation Development
Engineer lipid nanoparticles for targeted delivery to affected tissues
In Vitro Testing
Validate enzyme activity and cellular uptake in patient-derived cell lines
Preclinical Studies
Assess safety, biodistribution, and efficacy in animal models
Project Timeline
Discovery
Preclinical
IND Preparation
Clinical Readiness
Discovery
| Project | Name | Subitems | Project Manager | Project Timeline | Milestone Status | Readiness Level | Risk Level | Public Visibility | Investor Visibility | Public summary | Investor Summary |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NBIA-MPAN mRNA Therapy | - | - | Planned | In Vitro | - | Yes | No | ||||
| NBIA-MPAN Therapuetic Program | - | - | Planned | Human Evidence | - | No | Yes | ||||
| Target Validation | - | - | Completed | Human Evidence | Low | Yes | Yes | Discovery Milestone Completed and Program Advanced to next stage | Target Validation completed with supportive in Vitro evidence. Next step is mechanism confirmation and advancement toward lead candidate slelection. | ||
| Mechanism of Action | - | - | Completed | Human Evidence | Medium | No | Yes | ||||
| In Vitro Efficacy | - | - | Planned | In Vitro | High | No | Yes | ||||
| BBB Penetration | - | - | Planned | In Vitro | Low | No | Yes |
Preclinical
| Project | Name | Subitems | Project Manager | Project Timeline | Milestone Status | Readiness Level | Risk Level | Public Visibility | Investor Visibility | Public summary | Investor Summary |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Lead Candidate Selection | - | - | In Progress | Preclinical | Medium | - | No | ||||
| Preclinical Toxicology Initiated | - | - | In Progress | Preclinical | - | - | No | ||||
| Biodistribution Study | - | - | Planned | Preclinical | Medium | - | No |
IND Preparation
| Project | Name | Subitems | Project Manager | Project Timeline | Milestone Status | Readiness Level | Risk Level | Public Visibility | Investor Visibility | Public summary | Investor Summary |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Pre-IND Meeting Preparation | - | - | Blocked | Preclinical | Medium | - | Yes | ||||
| IND Enabling Studies Intiated | - | - | In Progress | Clinical | Medium | - | Yes |
Clinical Readiness
| Project | Name | Subitems | Project Manager | Project Timeline | Milestone Status | Readiness Level | Risk Level | Public Visibility | Investor Visibility | Public summary | Investor Summary |
|---|---|---|---|---|---|---|---|---|---|---|---|
| No data available | |||||||||||