What Repair Biotechnologies does

Repair Biotechnologies, Inc. is a private clinical-stage biotechnology company founded in 2018 and headquartered in Syracuse, New York (Delaware-incorporated). The company is developing the Cholesterol Degrading Platform (CDP), a first-in-class therapeutic modality that uses lipid nanoparticle (LNP)-delivered mRNA encoding an enzyme to selectively degrade toxic excess free cholesterol inside cells into non-toxic, readily excreted metabolites. The platform addresses the previously undruggable target of intracellular cholesterol accumulation and is positioned to achieve actual regression of existing atherosclerotic plaque, rather than only slowing disease progression as statins and PCSK9 inhibitors do. The company's lead product candidate, REP-0003, targets atherosclerotic plaque regression and is initially being developed for homozygous familial hypercholesterolemia (HoFH), with planned expansion into broader atherosclerosis, metabolic dysfunction-associated steatohepatitis (MASH), and other cholesterol-driven conditions affecting the heart, arteries, liver, lungs, and brain.

REP-0003 has received FDA Orphan Drug Designation (May 2025) for HoFH, providing seven years of post-approval market exclusivity, tax credits on clinical trial costs, and waived FDA user fees, as well as Rare Disease Evidence Principles (RDEP) eligibility (May 2026), which enables a streamlined approval pathway based on a single adequate and well-controlled clinical study. Repair Biotechnologies' revenue model is the traditional pharmaceutical product-sale model, with revenue currently at zero and future revenue contingent on FDA approval and commercial launch; distribution is expected to flow through specialty pharmacies and hospital networks for rare disease indications such as HoFH. The go-to-market motion is currently scientific and regulatory rather than commercial, encompassing Scientific Advisory Board engagement, academic conference presentations (American Heart Association Scientific Sessions 2025), and a manufacturing partnership with AGC Biologics.

The company is led by CEO and co-founder Reason (a longevity industry advocate and founder of Fight Aging!), CSO Mourad Topors, PhD (former Harvard Medical School principal investigator and Pfizer drug development program architect), and CMO Bobby Khan, MD, PhD (cardiologist and Professor of Medicine at the University of Central Florida). Its Scientific Advisory Board was strengthened in 2025 with the appointments of Dr. Deepak L. Bhatt (Director of Mount Sinai Fuster Heart Hospital) and Prof. Frederick J. Raal (a leading familial hypercholesterolemia specialist). Repair Biotechnologies has raised approximately $30 million cumulatively across multiple funding rounds since 2018, backed by longevity-focused investors including Healthspan Capital, Emerging Longevity Ventures, SENS Research Foundation, Methuselah Foundation, LongevityTech.Fund, Thynk Capital, and Bioverge, and has filed a Form D for an exempt securities offering of up to $50 million.

Name

Repair Biotechnologies

Legal name

Repair Biotechnologies, Inc.

Website

https://repairbiotechnologies.com

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

1–10 employees

Short description

Repair Biotechnologies is a pre-revenue clinical-stage biotechnology company developing the Cholesterol Degrading Platform (CDP), an mRNA-LNP therapy that selectively degrades toxic intracellular cholesterol. Its lead candidate REP-0003 targets atherosclerotic plaque regression, initially for homozygous familial hypercholesterolemia (HoFH) patients.

Ownership category

akta.pro rank

Product category

Cardiovascular Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Cardiometabolic & Other Systemic Gene Therapies (HLAAACAI)

akta.pro secondary industries

Enzyme Replacement & Metabolic Disorder Therapies (HLAIAIAB), Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization) (HLAAAIAC)

Keywords

• MRNA therapeutics
• Cholesterol degradation
• Atherosclerosis treatment
• Rare disease therapy
• Lipid nanoparticle delivery

Headquarters

HQ city

Syracuse

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Others

Revenue model

1. Therapeutic Product Revenue: Pre-revenue clinical-stage biotech company. Revenue expected from future sales of REP-0003 and other CDP-based therapies upon FDA approval. Target markets include homozygous familial hypercholesterolemia (HoFH), atherosclerosis, and metabolic dysfunction-associated steatohepatitis (MASH). Orphan Drug Designation provides 7 years of market exclusivity.

Go-to-market motion1 record

Marketing channels4 records

Core offering

Repair Biotechnologies develops the Cholesterol Degrading Platform (CDP), a first-in-class mRNA therapy platform that encodes an enzyme to selectively and safely degrade toxic excess intracellular free cholesterol into non-toxic, readily excreted metabolites. Its lead clinical-stage candidate, REP-0003, is an LNP-delivered mRNA therapy targeting atherosclerotic plaque regression, with an initial indication of homozygous familial hypercholesterolemia (HoFH) and broader applicability to atherosclerosis, MASH, and other cholesterol-driven conditions.

Product overview

Repair Biotechnologies develops first-in-class therapies based on the Cholesterol Degrading Platform (CDP), a proprietary technology that enables targeted cells to safely break down excess cholesterol inside cells. The lead product is REP-0003, an mRNA therapy encoding an enzyme to degrade toxic intracellular free cholesterol for treating atherosclerotic plaque in homozygous familial hypercholesterolemia (HoFH) and general atherosclerosis. The CDP platform is modular and can be applied to multiple indications including MASH and other undisclosed liver conditions.

Differentiator

Problem solved

Functional benefit

Products and services

• REP-0003 Lead clinical-stage mRNA therapy based on the Cholesterol Degrading Platform; encodes an enzyme delivered via lipid nanoparticles that selectively degrades toxic excess intracellular free cholesterol to regress atherosclerotic plaque. Initial indication is homozygous familial hypercholesterolemia (HoFH), with planned expansion to broader atherosclerosis.
• Cholesterol Degrading Platform (CDP) Proprietary modular mRNA therapy platform that enables targeted cells to safely break down unwanted excess cholesterol by encoding a cholesterol-degrading enzyme. Tailorable across multiple indications (atherosclerosis, MASH, other liver and age-related cholesterol-driven conditions) and delivery mechanisms.

Quantifiable outcome

• 48% reduction in atherosclerotic plaque lipids in mice after single AAV-based CDP treatment
• +2 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

One partnership is on record.

• AGC BiologicscoreStrategic or Co-development Partner · 19 November 2025AGC Biologics, a leading global CDMO, partnered with Repair Biotechnologies to develop and manufacture a novel mRNA therapeutic targeting atherosclerotic plaques. The collaboration focuses on developing and manufacturing the therapy to rapidly stabilize and reduce the size of atherosclerotic plaques in major blood vessels. AGC's Heidelberg facility provides comprehensive mRNA manufacturing from plasmid DNA to final drug product encapsulated in lipid nanoparticles, compliant with EMA and FDA GMP standards.

Scale indicators4 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Verve Therapeutics: Develops in vivo gene-editing therapies (LNP-delivered base editors) that durably lower LDL-C by silencing PCSK9, ANGPTL3, and Lp(a) for cardiovascular disease. Most direct mechanistic peer to Repair's LNP-mRNA enzyme approach for intracellular cholesterol modulation.
• Intellia Therapeutics: Clinical-stage company using CRISPR/Cas9 delivered via lipid nanoparticles to edit genes in vivo, including transthyretin amyloidosis and hereditary angioedema programs. Closely comparable LNP-delivered nucleic-acid platform and cardiovascular-adjacent therapeutic strategy.
• Alnylam Pharmaceuticals: Pioneer in RNA interference therapeutics, including Leqvio (inclisiran), an approved siRNA targeting PCSK9 for hypercholesterolemia. Competes in the same lipid-lowering indication space with an approved, commercially launched RNA-based therapy.
• Arrowhead Pharmaceuticals: Develops RNAi-based therapies using targeted delivery platforms for cardiovascular and metabolic diseases, including programs against PCSK9 and ANGPTL3. Highly comparable RNA-based therapeutic approach for lipid management.
• Tenaya Therapeutics: Clinical-stage genetic medicines company focused on heart disease, using both gene therapy and gene editing approaches. Targets cardiovascular indications with a therapeutic platform strategy analogous to Repair's CDP.
• Lexeo Therapeutics: Clinical-stage company developing genetic medicines for cardiovascular diseases, including AAV-based therapies for familial cardiomyopathies and dyslipidemias. Direct competitor in cardiovascular genetic medicine with overlapping indication focus.

Emerging players

• Beam Therapeutics: Clinical-stage base editing company with a cardiovascular program targeting LP(a) using in vivo LNP delivery. Closely related nucleic-acid platform and lipid-targeting therapeutic strategy, though at an earlier stage of clinical development in cardiovascular indications.
• Korro Bio: Clinical-stage RNA editing company leveraging ADAR-based oligonucleotides for cardiometabolic and other diseases, including programs targeting lipid pathways. Comparable nucleic-acid approach to modulating disease-driving transcripts in cardiovascular indications.
• ProQR Therapeutics: Clinical-stage RNA therapeutics company developing Axiomer RNA-editing technology and other oligonucleotide platforms for rare and metabolic diseases. Adjacent RNA-based therapeutic modality with overlapping rare disease development approach.

Broad incumbents

• Moderna: Industry-leading mRNA therapeutics company whose LNP-mRNA platform underpins the COVID vaccine franchise and a broad pipeline in rare diseases and cardiometabolic indications. Broad incumbent with the same underlying mRNA-LNP delivery modality as Repair's CDP.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/repairbiotechnologies

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds9 records

Investors7 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Repair Biotechnologies