Pipeline: From Research Tools to Life-Saving Therapies

Phase I: Research protein products

Establishing revenue & scientific credibility (Years 1–2) · Status: Active development

Focus: Premium B2B research reagents for biopharma and academic labs.

Product 1: Homogeneously phosphorylated tau proteins

The innovation: First commercial source of site-specifically phosphorylated tau proteins with homogeneous phosphorylation at all 85 potential sites; single-site and multi-site variants; validated by mass spectrometry; 95% purity.

Applications — Alzheimer’s research: Mechanistic studies; drug screening for aggregation inhibitors; antibody development. Diagnostic development: Biomarker calibration; immunoassay standards; clinical test validation.

Target customers: Academic neuroscience labs; pharmaceutical R&D; diagnostic companies; biotech developing tau therapeutics. Market position: Unique offering; $500–2,000 per 100 µg; growing Alzheimer’s research reagents market ($8B+ annually).

Product line: Year 1: 5–10 key phosphorylation sites (Ser199, Ser202, Thr231, Ser396, etc.); Year 2: 20–30 variants including combinations; custom phosphorylation patterns on request.

Product 2: Clickable antibodies

The innovation: Site-specific incorporation of click chemistry handles—defined DAR; superior homogeneity vs. traditional methods; retained antigen binding; multiple click options (azide, alkyne, tetrazine).

Applications: ADC development; diagnostics & imaging; research tools. Service offerings: Standard IgG with clickable ncAAs; custom sequences; full conjugation services; platform licensing.

Pricing: Research-grade antibodies $1,000–5,000 per mg; custom conjugation $10K–50K; platform licensing negotiated.

Product 3: Custom engineered proteins

The service: Contract protein engineering incorporating client-specified ncAAs—50+ validated ncAAs; proteins up to 100 kDa; multiple incorporation sites; GMP-compatible processes. Process: Feasibility ($5K–15K); optimization & production ($25K–100K); characterization; commercial licensing.

Phase II: Antibody–drug conjugates

Precision oncology therapeutics (Years 3–5) · Status: Preclinical development

Focus: Next-generation ADCs leveraging the clickable antibody platform.

Our advantage: Superior DAR control; enhanced therapeutic index; reproducible manufacturing; established platform from Phase I products.

Lead candidate 1 — HER2-targeted ADC: Indication HER2+ breast and gastric cancer; trastuzumab-based or next-generation anti-HER2; MMAE payload; DAR 4; cleavable or non-cleavable linker.

Lead candidate 2 — Solid tumor ADC: EGFR+, TROP-2+, or Nectin-4+ tumors; novel payloads (PBD dimers, topoisomerase inhibitors); DAR 2–4.

Lead candidate 3 — Hematological ADC: CD19+, CD22+, or BCMA+ blood cancers; high-potency payloads; DAR 2; rapid cellular uptake.

Phase III: Protein editing therapies

Revolutionary treatment for age-related disease (Years 5+) · Status: Platform development

Focus: In vivo protein editing for neurodegenerative diseases and aging.

The paradigm shift: From symptom management to disease prevention—modify disease-causing proteins in vivo; prevent toxic aggregation; resist oxidative damage; maintain proteostasis.

Safety profile: Transient mRNA delivery (no DNA changes); reversible and dose-controllable; tissue-specific targeting; repeat dosing as needed.

Program 1: Alzheimer’s disease — tau editing

Indication: Early to moderate Alzheimer’s disease. Target population: ~6.7M patients in US; biomarker-positive (elevated CSF p-Tau, amyloid PET+); MMSE 20–26.

Therapeutic strategy: Oxidation-resistant tau; aggregation-blocking tau; stabilized tau (proline analogs in microtubule-binding repeats). Delivery: CNS-targeted LNPs; intrathecal or IV; monthly or quarterly dosing.

Program 2: Parkinson’s disease — alpha-synuclein editing

Indication: Early Parkinson’s disease

Target population:

• 1M patients in US
• Hoehn & Yahr Stage 1-2
• Dopaminergic deficit confirmed by DaTscan

Therapeutic strategy:

Oxidation-proof α-synuclein:

• Prevent ROS-induced aggregation triggers

NAC domain modification:

• Block hydrophobic core formation
• Prevent Lewy body assembly

Tetrameric stabilization:

• Cross-linking ncAAs to maintain soluble tetramer
• Prevent pathogenic monomer accumulation

Delivery:

• Substantia nigra-targeted VLPs
• Stereotactic injection or systemic with CNS penetration
• Quarterly dosing protocol

Program 3: ALS — SOD1 & TDP-43 editing

Indication: ALS (SOD1 mutations, sporadic with TDP-43 pathology)

Target population:

• 30K patients in US
• Familial SOD1 mutations (~20% of cases)
• Sporadic with TDP-43 aggregation (~45% of cases)

Therapeutic strategy:

SOD1 stabilization:

• Metal-chelating ncAAs at active site
• Enhanced Cu/Zn binding
• Prevention of misfolding and aggregation

TDP-43 cytoplasmic exclusion:

• Modified RNA-binding domains
• Prevent aberrant phase separation
• Maintain nuclear localization

Delivery:

• Intrathecal LNP-mRNA
• Direct CNS access to motor neurons
• Monthly dosing

Program 4: Age-related macular degeneration

Indication: Dry AMD (geographic atrophy)

Target population:

• 11M patients in US (dry AMD)
• No approved therapies
• Progressive vision loss

Therapeutic strategy:

RPE65 photo-stabilization:

• Photo-stable tyrosine variants
• Resist retinal photo-oxidation
• Maintain visual cycle function

ABCA4 oxidation protection:

• Selenocysteine analogs at vulnerable cysteines
• Prevent lipofuscin accumulation

Complement factor modulation:

• Reduce drusen-associated inflammation

Program 5: Aging & proteostasis (long-term)

Concept: General enhancement of cellular protein quality control

Approach:

• Enhanced chaperones (Hsp70, Hsp90)
• Optimized proteasome function
• Improved antioxidant defenses

Target applications:

• Age-related frailty
• Sarcopenia
• Cognitive decline in healthy aging

Status: Research stage, 5-10 year horizon

Learn more

Investors: Investors · Patients: Patients · Science: Science & technology.

← Back to home