Carmine Therapeutics
Carmine Therapeutics is a Cambridge, MA-based biotech developing non-viral gene therapies through its proprietary REGENT platform, which uses red blood cell extracellular vesicles to deliver large nucleic acid payloads for rare genetic diseases including cystic fibrosis, retinal, and pulmonary disorders.
- Company typePrivate
- Founded2019
- HeadquartersCambridge, United States
- Headcount11–50
- GTM typeB2B
- OfferingServices
What Carmine Therapeutics does
Carmine Therapeutics is a privately-held, clinical-stage biotechnology company developing non-viral gene therapies based on red blood cell extracellular vesicles (RBCEVs). Founded in 2019 by EVX Ventures (formerly Esco Ventures X) alongside scientific co-founders Prof. Harvey Lodish of MIT/Whitehead Institute, Prof. Minh Le, and Prof. Jiahai Shi, the company is headquartered in Cambridge, Massachusetts, with research operations in Singapore. The proprietary REGENT® (Red Cell EV Gene Therapy) platform leverages RBCEVs — physiologically produced, biocompatible vesicles that lack genetic material — as non-immunogenic delivery vehicles capable of carrying DNA or RNA payloads ranging from 20bp to over 30kb, supporting potential repeat dosing, modular surface functionalization for tissue-specific targeting, and simplified manufacturing relative to viral-based systems. Pipeline programs target rare genetic diseases including cystic fibrosis, retinal disorders, pulmonary conditions, and CNS disorders.
The company operates as a B2B biotech with an enterprise-partnership go-to-market model rather than direct product sales. In June 2020, Carmine entered a research collaboration with Takeda Pharmaceutical covering two rare disease targets; under the agreement Takeda receives an option to license programs following pre-clinical proof of concept and assumes responsibility for clinical development and commercialization, while Carmine is eligible for over $900M in total milestone payments plus tiered royalties. The company has raised more than $9.4M in seed financing led by Esco Ventures X and a Series A first close in October 2022 with participation from Huagai Capital, the Cystic Fibrosis Foundation, EVX Ventures, and Simcere Pharmaceuticals. Leadership includes CEO Don Haut, PhD (formerly Chief Business Officer at AskBio during its US$4B Bayer acquisition) and a Scientific Advisory Board chaired by Prof. Mark Kay of Stanford, with the original RBCEV co-inventors continuing to support the platform's scientific development.
Carmine Therapeutics firmographics
Firmographics- Name
- Carmine Therapeutics
- Legal name
- Carmine Therapeutics, Inc.
- Website
- https://carminetherapeutics.com
- Company type
- Private
- Founded year
- 2019
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Carmine Therapeutics is a Cambridge, MA-based biotech developing non-viral gene therapies through its proprietary REGENT platform, which uses red blood cell extracellular vesicles to deliver large nucleic acid payloads for rare genetic diseases including cystic fibrosis, retinal, and pulmonary disorders.
- Ownership category
- akta.pro rank
Carmine Therapeutics industry classification
Industry- Product category
- Non-viral Gene Therapy Platform
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Non-viral Gene Delivery & Gene Therapy (LNP, polymers, electroporation, physical methods) (HLAAACAC)
- akta.pro secondary industries
- Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Neurology/CNS Gene Therapies (HLAAACAH)
Keywords
Where Carmine Therapeutics is headquartered
LocationHeadquarters
- HQ city
- Cambridge
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
Carmine Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Technology or R&D, Personnel, Operations, Infrastructure
Revenue model
- Partnership Milestone Payments: Carmine receives upfront payments, research funding support, and is eligible for milestone payments upon achieving development and commercial milestones. From the Takeda partnership, Carmine is eligible for over $900M USD in total milestone payments plus tiered royalties.
- Royalties: Carmine receives tiered royalties on net sales of products developed through partnerships such as the Takeda collaboration, following successful development and commercialization.
- Series A Financing: Equity financing round to fund R&D operations, with participation from existing investors Huagai Capital, Cystic Fibrosis Foundation, and others. First close announced in October 2022.
Go-to-market motion1 record
Distribution channels1 record
Marketing channels4 records
Carmine Therapeutics product offering
Product offeringCore offering
Carmine Therapeutics develops a proprietary Red Cell EV Gene Therapy (REGENT®) platform that uses red blood cell extracellular vesicles (RBCEVs) as a non-viral delivery vehicle for nucleic acid therapeutics. The platform underpins a pipeline of disease-modifying gene therapies for rare diseases including cystic fibrosis, retinal, and pulmonary disorders, licensed and co-developed with global pharmaceutical partners such as Takeda.
Product overview
Carmine Therapeutics is pioneering a new class of genetic medicines based on red blood cell extracellular vesicles (RBCEVs). The company's core offering is the REGENT® (Red Cell EV Gene Therapy) platform, which generates a pipeline of next-generation disease-modifying gene therapies. The platform overcomes limitations of viral-based gene therapies through non-immunogenic, re-dosable vesicles with large payload capacity (>30kb) and modular surface functionalization for targeted tissue delivery. The company is developing programs in retinal diseases, pulmonary disorders, and cystic fibrosis.
Differentiator
Problem solved
Functional benefit
Brands
- REGENT: Red Cell EV Gene Therapy (REGENT) platform - proprietary technology for generating next-generation disease-modifying gene therapies using red blood cell extracellular vesicles (RBCEVs).
Products and services
- REGENT® Platform (Red Cell EV Gene Therapy) Proprietary gene therapy delivery platform built on red blood cell extracellular vesicles, designed for license and co-development with pharmaceutical partners across rare disease indications.
- Non-viral Gene Therapy Pipeline (retinal, pulmonary, and cystic fibrosis programs) Pipeline of disease-modifying gene therapy candidates targeting retinal diseases, pulmonary disorders, and cystic fibrosis, developed through research collaborations and option-to-license structures with pharmaceutical partners.
Quantifiable outcome
- Eligible for over $900M USD in milestone payments plus tiered royalties from Takeda partnership
- +2 more outcomes
Companies that use Carmine Therapeutics
Customer profileSegments2 records
Ideal customer profiles1 record
Carmine Therapeutics technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Carmine Therapeutics partnerships and signals
Strategic signalPartnerships
Two partnerships are on record, tiered core and minor.
- Takeda Pharmaceutical Company LimitedcoreResearch collaboration to discover, develop and commercialize transformative non-viral gene therapies for two rare disease targets using Carmine's REGENT technology. Carmine receives upfront payment, research funding, and is eligible for over $900M USD in total milestone payments plus tiered royalties. Takeda has option to license programs following pre-clinical proof of concept and is responsible for clinical development and commercialization.
- LabCentralminorWon Bristol-Myers Squibb's 2019 Golden Ticket for LabCentral, which provides subsidized laboratory space in LabCentral's Kendall Square facility in Cambridge, MA.
Scale indicators5 records
Recent moves6 records
Expansion highlights5 records
Carmine Therapeutics competitors and assessment
Company assessmentDirect peers
- Evox Therapeutics: UK-based biotech engineering extracellular vesicles (exosomes) for therapeutic delivery of nucleic acids and proteins, including gene therapy applications. Directly comparable to Carmine as a fellow extracellular-vesicle-based delivery platform targeting similar rare disease and CNS indications.
- Generation Bio: Non-viral gene therapy company using lipid nanoparticle (LNP) delivery of DNA and mRNA payloads to the liver and other tissues. Comparable to Carmine as another non-viral gene therapy platform pursuing redosable, large-payload delivery, with overlapping rare disease and CNS ambitions.
- Rubius Therapeutics: Developed red blood cell-based cell therapies (including for gene therapy and enzyme replacement). Highly comparable to Carmine given the shared red-blood-cell foundation and Prof. Harvey Lodish as a scientific co-founder of both companies; relevant benchmark for the modality's commercial trajectory.
- Codiak BioSciences: Developed engineered exosome (engEx) therapeutics for nucleic acid and protein delivery. Closely comparable to Carmine in modality — exosome/EV-based drug delivery — though Codiak has largely wound down or restructured, illustrating the execution risk in this emerging class.
Emerging players
- Capricor Therapeutics: Clinical-stage biotech developing extracellular vesicle-based therapies (CAP-1002, exosome-based delivery). Comparable in its use of vesicles as a therapeutic or delivery modality, though Capricor is more clinically advanced in cardiac and Duchenne indications.
- Verve Therapeutics: Clinical-stage biotech developing in vivo gene editing therapies for cardiovascular disease using LNP delivery. Comparable as a non-viral, redosable genetic medicine platform pursuing indications beyond classical rare-disease gene therapy.
- Haima Therapeutics: Early-stage biotech developing platelet-inspired and cell-derived delivery systems for gene therapy and regenerative medicine. Comparable as a niche emerging player pursuing cell-derived (non-viral) delivery of therapeutic cargo, with similar scientific roots in blood-cell biology.
Broad incumbents
- BioMarin Pharmaceutical: Commercial-stage gene therapy company with an approved AAV product (Roctavian) and pipeline across hemophilia and rare diseases. Relevant broad incumbent comparable to Carmine given overlapping rare disease focus and AAV-vs-non-viral competitive dynamics.
- uniQure: Established gene therapy company with an approved AAV-based product (Hemgenix) and a pipeline across rare diseases including CNS and metabolic indications. Comparable as a broad incumbent competitor in the gene therapy space, especially where Carmine targets CNS and rare diseases.
- AskBio: AAV gene therapy platform acquired by Bayer for ~$4 billion, with clinical programs across neuromuscular and CNS indications. Comparable as an incumbent AAV gene therapy competitor; Carmine's CEO Don Haut previously led business development at AskBio before the Bayer transaction.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat3 records
Key risks7 records
Key highlights7 records
Customer concentration
Carmine Therapeutics social profiles
Digital presenceCarmine Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Carmine Therapeutics leadership team
Management profileNumber of profiles
Profiles9 records
Carmine Therapeutics funding detail
Funding detailFunding overview
Funding rounds2 records
Investors5 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Carmine Therapeutics M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Carmine Therapeutics
What does Carmine Therapeutics do?
Carmine Therapeutics develops a proprietary Red Cell EV Gene Therapy (REGENT®) platform that uses red blood cell extracellular vesicles (RBCEVs) as a non-viral delivery vehicle for nucleic acid therapeutics. The platform underpins a pipeline of disease-modifying gene therapies for rare diseases including cystic fibrosis, retinal, and pulmonary disorders, licensed and co-developed with global pharmaceutical partners such as Takeda.
Is Carmine Therapeutics a public or private company?
Carmine Therapeutics is a private company. It is classified as venture growth investor backed and is currently operating.
When was Carmine Therapeutics founded?
Carmine Therapeutics was founded in 2019. It employs 11 to 50 people.
Where is Carmine Therapeutics based?
Carmine Therapeutics is headquartered in Cambridge, United States, in the North America region.
How does Carmine Therapeutics make money?
Three revenue lines are on record. Partnership Milestone Payments are the primary driver. The others are royalties and series A Financing.
Who are Carmine Therapeutics's main competitors?
Direct peers on record are Evox Therapeutics, Generation Bio, Rubius Therapeutics and Codiak BioSciences. Emerging players are Capricor Therapeutics, Verve Therapeutics and Haima Therapeutics. Broad incumbents are BioMarin Pharmaceutical, uniQure and AskBio.
Does Carmine Therapeutics have an API?
No public API is recorded for Carmine Therapeutics.
What industry is Carmine Therapeutics in?
Carmine Therapeutics's product category is Non-viral Gene Therapy Platform. Its primary akta.pro industry code is HLAAACAC, Non-viral Gene Delivery & Gene Therapy (LNP, polymers, electroporation, physical methods), with a secondary code of HLAAACAE, Gene Therapy for Rare/Monogenic Diseases. Its NAICS code is 541714 and its SIC code is 2836.