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Horizontal Therapeutics

Off-the-Shelf
mitoTherapy

Precision Organelle Therapy for Mitochondrial Dysfunctions

Seed Round Platform Technology RMAT Eligible
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The Problem

Mitochondrial Dysfunction is a Root Cause of Disease

Mitochondria are essential for ATP energy generation and metabolic regulation. Damage or genetic defects lead to energy failure, contributing to devastating conditions.

Primary Mitochondrial Diseases

Affect ~1 in 4,500 people globally. No FDA-approved gene or cell therapies exist for most conditions. Care is largely supportive.

  • Muscle weakness & progressive neurodegeneration
  • Organ failures from ATP deficits in high-energy tissues
  • Multisystem, progressive illness affecting children & adults
  • Current treatments only manage symptoms
1:4,500
Global Prevalence
0
Approved Therapies for MELAS
$60B
Neurodegeneration Market
100%
Unmet Medical Need

Our Solution

Off-the-Shelf Mitochondrial Transplantation

Replace dysfunctional mitochondria with healthy donor organelles—like a cellular "battery swap"

🔬

01 — Isolate

Harvest respiration-competent mitochondria from healthy donor cells

🧬

02 — Multiply

Expand donor cells to produce unlimited mitochondria from a single source

❄️

03 — Cryopreserve

Store using proprietary formulation for off-the-shelf availability (>80% activity retained)

💉

04 — Deliver

Infusion or local injection to target tissues—direct intervention

⚡

05 — Restore

Cells uptake via endocytosis/TNTs → ATP production restored, oxidative stress reduced

Non-Invasive

No gene editing required

Scalable

One donor → many patients

Universal

Mutation-agnostic platform

Immediate

No patient-specific prep

Technology Platform

Three Integrated Pillars

A comprehensive platform combining diagnostics, therapeutics, and manufacturing innovation

Genetic IVD

Patient Stratification

  • Advanced NGS/TGS sequencing
  • Mitonuclear mismatch scoring
  • Companion diagnostic for patient selection
  • Optimal donor-recipient matching
Early Revenue 2027-28

Compatibility Algorithm

Mitonuclear Matching

  • Donor selection by mtDNA haplotype
  • Recipient compatibility optimization
  • Rigorous functional assays
  • Safety & efficacy validation
Proprietary Technology

Mito Cell Bank

Off-the-Shelf Supply

  • Cryopreserved inventory
  • 1 donor → 1000s of patients
  • Standardized lot characterization
  • Global cold-chain logistics
>80% Post-Thaw Viability

Validation

Proven Science, Clear Mechanism

Preclinical Proof-of-Concept

Mitochondrial transplantation improves organ function in animal models. Pilot study in pediatric cardiac patients showed:

80%
Survival (mito therapy)
29%
Survival (control)

No Safety Red Flags

No pro-inflammatory immune reactions observed in preclinical models. Transplanted mitochondria are immunologically inert when properly matched— no MHC-I on surface.

Mechanistic Evidence

Cellular Uptake

Mitochondria integrate via tunneling nanotubes (TNTs) and endocytosis

Functional Integration

Remain respiration-competent in host tissue, begin ATP production

Energy Restoration

Restored ATP levels, reduced cell death, decreased oxidative stress

Genetic Complementation

Donor mtDNA can complement defective patient mtDNA without nuclear editing

Market Position

Competitive Landscape

First-mover advantage in off-the-shelf allogeneic mitochondrial therapy

Company
Stage / Funding
Approach
Limitation
Minovia Therapeutics
Phase I/II • $16M
Allogeneic, IV delivery
Cannot cross BBB for neuro; limited to systemic
Cellvie Bio
Preclinical • $5.5M
Autologous, cardiac focus
4-week patient-specific delay; not scalable
Mitrix Bio
Early Research
Bioreactor-grown, aging
Very early stage; no clinical data
Horizontal Therapeutics
Seed • $8M raise
Off-the-shelf, scalable, PMD focus
Platform Leader
🎯
Mutation-Agnostic

Works regardless of specific genetic defect

⚡
Immediate Availability

No patient-specific manufacturing delay

🔒
IP Moat

Proprietary matching + manufacturing know-how

Regulatory Strategy

FDA Regulatory Tailwinds

NEW: Plausible Mechanism Pathway

"The FDA's new 'plausible mechanism' pathway provides a transformative opportunity for genetic diseases like PMDs..."

NEJM Sounding Board: FDA's New Plausible Mechanism Pathway (Nov 2025)

Our Regulatory Advantages

  • Orphan Drug Designation — 7-year exclusivity, tax credits, expedited review
  • Rare Pediatric Disease — Priority review voucher eligibility
  • RMAT Designation — Rolling BLA, frequent FDA interactions
  • Small Trial Sizes — n=20 vs n=500 for traditional programs

Development Timeline

6-12 Months
Preclinical Efficacy

Cell models, ATP measurements, wound healing proof-of-concept

12-18 Months
Manufacturing & IND-Enabling

GMP scale-up, toxicology studies, dose formulation

18-24 Months
Phase 1/2 Clinical Trial

Open-label study in MELAS patients, safety + efficacy signals

Target
Accelerated Approval

Via plausible mechanism pathway with biomarker endpoints

Market Opportunity

Platform Expansion Strategy

Initial Target

MELAS

Severe mitochondrial encephalomyopathy with zero approved treatments. Patients suffer recurrent neurological episodes.

$500M+
Market Potential
Platform Expansion

Chronic Wounds

Diabetic foot ulcers and pressure sores. Impaired mitochondrial function hinders repair.

Homeostasis
Inflammation
Proliferation
Remodeling

Bioenergetic Deficit Gap

Long-Term Vision

Neurodegeneration

ALS, Parkinson's, Alzheimer's subsets with mitochondrial dysfunction. Massive unmet need and market opportunity.

$60B+
Total Market
Premium Pricing Justified

Comparable orphan drugs: Spinraza $750K/yr • Zolgensma $2.1M one-time

85-90%
Target Gross Margin

Future Vision

Transformative Platform Growth

A roadmap to global accessibility and expanded therapeutic scope

🏦

Mitochondrial Banks

Advanced cryostorage enables readily accessible "bio-banks," dramatically reducing costs and improving access for acute care.

🧬

Personalized Medicine

Autologous transplantation and precision-targeted delivery align with the global shift toward individualized therapies.

🌏

Global Expansion

Targeting emerging markets with rapidly aging populations (e.g., Asia-Pacific) to drive massive future demand.

🔗

Adjunct Therapies

Integrating mitoTherapy with immunotherapy or gene editing to synergistically expand the therapeutic scope.

Team & Advisors

Founding Team & Scientific Leadership

Combined expertise in translational medicine, mitochondrial biology, and clinical development

Baylor College of Medicine

Baylor College of Medicine Heritage

Founding team with deep clinical and translational medicine expertise, leveraging decades of experience in molecular diagnostics, mitochondrial biology, and genetic therapeutics from one of the world's leading medical research institutions.

👨‍⚕️
Brendan H.L. Lee, MD, PhD

Chair, Molecular & Human Genetics

President, American Society of Human Genetics

👩‍🔬
Huda Y. Zoghbi, M.D.

Director, Duncan Neurological Research

Fellow, National Academy of Sciences

👩‍⚕️
Sau Wai Cheung, Ph.D.

Professor, Molecular & Human Genetics

Fellow, American Board of Medical Genetics

👨‍🔬
Fernando Scaglia, M.D.

Director, Pediatric Mito Medicine Clinic

Clinical Director, BCM-CUHK Joint Center

Strategic Infrastructure

🏭
cGMP Manufacturing

Proprietary analytics partnership with top-tier CDMO

📋
Regulatory CRO

International pathway navigation & RMAT acceleration

🚚
Cold-Chain Logistics

Flawless product integrity from plant to point-of-care

Financials

Use of Proceeds & Execution Plan

$8M Seed/Pre-Series A to achieve IND filing in 24 months

24-Month Execution Roadmap

Period Key Objectives Deliverables
Q1-Q2 Foundation: CDMO partnership secured, Analytical panel, Preclinical efficacy models. CDMO Work Order, Draft Assay Protocols, In Vivo Designs
Q3-Q4 Development: In vivo efficacy data, Process dev (3 engineering batches), FDA Pre-IND meeting. Preclinical Reports, SOPs, FDA Meeting Minutes
Q5-Q6 Validation: GLP tox study, GMP-like batch, Potency assay validated. GLP Tox Report, Stability Data (3mo), Validation Report
Q7-Q8 Regulatory: GMP clinical batch released, IND submitted, Phase 1 sites selected. CoA for GMP Batch, IND Submission Confirmation

Budget Allocation

Process Dev & GMP 40% ($3.2M)
Preclinical R&D 30% ($2.4M)
Regulatory & Clinical 20% ($1.6M)
Operations & Contingency 10% ($0.8M)

*Buffer ensures runway extension post-IND filing

Series A Target: $20-30M

Triggered by Phase 1/2 data readout. Funds pivotal trials and commercial preparation.

Investment Opportunity

The Ask

$8M

Seed / Pre-Series A Round

18-24
Months Runway
IND
Filing Target
RMAT
Pathway Eligible
1st
Mover Advantage

Why Invest Now?

✓ Ground-Floor Entry

Pre-data valuation with significant upside

✓ Regulatory Tailwinds

New FDA pathways favor our approach

✓ Platform Optionality

PMD success unlocks $60B+ neuro market

✓ World-Class Advisors

BCM's top mitochondrial experts

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"Pioneering a new class of organelle therapy to transform how we treat energy-deficit diseases."