Horizontal Therapeutics
Precision Organelle Therapy for Mitochondrial Dysfunctions
The Problem
Mitochondria are essential for ATP energy generation and metabolic regulation. Damage or genetic defects lead to energy failure, contributing to devastating conditions.
Affect ~1 in 4,500 people globally. No FDA-approved gene or cell therapies exist for most conditions. Care is largely supportive.
Our Solution
Replace dysfunctional mitochondria with healthy donor organelles—like a cellular "battery swap"
Harvest respiration-competent mitochondria from healthy donor cells
Expand donor cells to produce unlimited mitochondria from a single source
Store using proprietary formulation for off-the-shelf availability (>80% activity retained)
Infusion or local injection to target tissues—direct intervention
Cells uptake via endocytosis/TNTs → ATP production restored, oxidative stress reduced
No gene editing required
One donor → many patients
Mutation-agnostic platform
No patient-specific prep
Technology Platform
A comprehensive platform combining diagnostics, therapeutics, and manufacturing innovation
Validation
Mitochondrial transplantation improves organ function in animal models. Pilot study in pediatric cardiac patients showed:
No pro-inflammatory immune reactions observed in preclinical models. Transplanted mitochondria are immunologically inert when properly matched— no MHC-I on surface.
Mitochondria integrate via tunneling nanotubes (TNTs) and endocytosis
Remain respiration-competent in host tissue, begin ATP production
Restored ATP levels, reduced cell death, decreased oxidative stress
Donor mtDNA can complement defective patient mtDNA without nuclear editing
Market Position
First-mover advantage in off-the-shelf allogeneic mitochondrial therapy
Works regardless of specific genetic defect
No patient-specific manufacturing delay
Proprietary matching + manufacturing know-how
Regulatory Strategy
Published November 2025 — FDA's transformative pathway for genetic diseases allows approval based on strong biological rationale and early clinical improvements in small patient cohorts.
Cell models, ATP measurements, wound healing proof-of-concept
GMP scale-up, toxicology studies, dose formulation
Open-label study in MELAS patients, safety + efficacy signals
Via plausible mechanism pathway with biomarker endpoints
Market Opportunity
Severe mitochondrial encephalomyopathy with zero approved treatments. Patients suffer recurrent neurological episodes.
Diabetic foot ulcers and pressure sores. Impaired mitochondrial function hinders repair. Rapid proof-of-concept readout.
ALS, Parkinson's, Alzheimer's subsets with mitochondrial dysfunction. Massive unmet need and market opportunity.
Comparable orphan drugs: Spinraza $750K/yr • Zolgensma $2.1M one-time
Team & Advisors
Experts in mitochondrial biology, genetics, and clinical development from Baylor College of Medicine
Chair, Molecular & Human Genetics
President, American Society of Human Genetics
Fellow, National Academy of Medicine
Director, Duncan Neurological Research
Fellow, National Academy of Sciences
Fellow, National Academy of Medicine
Professor, Molecular & Human Genetics
Fellow, American Board of Medical Genetics
Director, Pediatric Mito Medicine Clinic
Clinical Director, BCM-CUHK Joint Center
Specialized expertise
CDMO partnership
International pathway
Global distribution
Financials
18-24 months runway to achieve key value-inflection milestones
| Preclinical R&D | 40% | $2.0M |
| Process Dev & GMP | 25% | $1.25M |
| Regulatory & Clinical Prep | 15% | $0.75M |
| Team & Operations | 15% | $0.75M |
| IP & Legal | 5% | $0.25M |
Upon Phase 1/2 data readout to fund pivotal trials and commercial preparation
Investment Opportunity
Seed / Pre-Series A Round
Pre-data valuation with significant upside
New FDA pathways favor our approach
PMD success unlocks $60B+ neuro market
BCM's top mitochondrial experts
"Pioneering a new class of organelle therapy to transform how we treat energy-deficit diseases."