What Capricor Therapeutics does

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company developing cell and exosome-based therapeutics, with its lead asset Deramiocel (CAP-1002), an allogeneic cardiosphere-derived cell (CDC) therapy in late-stage Phase 3 development for Duchenne muscular dystrophy (DMD). The company originated from Eduardo Marbán's research and was incorporated in 2005, gaining its public listing via a 2013 reverse merger with Nile Therapeutics. The HOPE-3 Phase 3 trial reported positive topline data in December 2025, meeting its primary upper-limb function endpoint (PUL v2.0, p=0.029) and demonstrating significant cardiac benefit (LVEF improvement versus placebo). The FDA is currently reviewing the BLA with a PDUFA target action date of August 22, 2026, following the lifting of a July 2025 Complete Response Letter. Deramiocel has received RMAT, Orphan Drug, and Rare Pediatric Disease designations. A second platform, StealthX, is a proprietary exosome-based delivery technology for oligonucleotides, proteins, and small molecules, currently in preclinical and early development.

The company is pre-revenue with no product sales as of Q1 2026 and reported a full-year 2025 net loss of $105M. Revenue to date has been limited to ratable recognition of upfront payments and government grants. Capitalization is supported by approximately $318M in cash as of December 31, 2025, a $150M December 2025 public offering, prior $75M (October 2024) and $23M (September 2023) raises, and a market capitalization of approximately $1.23B-$1.26B. Commercialization is structured around an exclusive partnership with Nippon Shinyaku (via its US subsidiary NS Pharma) covering the United States, Japan, and (as of September 2024) Europe, with up to $715M in potential milestones plus revenue sharing. The company is also building internal commercial capabilities and has filed a breach-of-contract lawsuit against NS Pharma/Nippon Shinyaku in May 2026 alleging inadequate launch preparation. The addressable DMD therapeutics market is projected to grow from $6.80B in 2025 to $26.88B by 2035, providing a substantial tailwind if Deramiocel secures approval.

Name

Capricor Therapeutics

Legal name

Capricor Therapeutics, Inc.

Website

https://capricor.com

Company type

Public

Founded year

2005

Operating status

Operating

Headcount range

251–500 employees

Short description

Capricor Therapeutics is a clinical-stage biotechnology company developing Deramiocel, an allogeneic cardiac-derived cell therapy for Duchenne muscular dystrophy, alongside a proprietary StealthX exosome delivery platform, targeting rare disease patients globally through its Nippon Shinyaku partnership.

Ownership category

akta.pro rank

Product category

Biotechnology — Cell and Exosome Therapeutics for Rare Diseases

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

Cell Therapy Developers (Allogeneic/Off-the-shelf) (HLAAABAB)

Keywords

• Cell therapy biotechnology
• Exosome therapeutics
• Duchenne muscular dystrophy
• Regenerative medicine
• Biologics manufacturing

Headquarters

HQ city

San Diego

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Product Revenue (Projected): Capricor is pre-revenue with no product sales. Upon FDA approval of Deramiocel, the company expects to generate product revenue from sales in the United States through its partnership with Nippon Shinyaku and potentially through direct sales capabilities.
2. Milestone Payments from Nippon Shinyaku: Capricor receives milestone payments tied to development, regulatory, and commercial achievements under its agreement with Nippon Shinyaku. Potential milestones up to $715 million, plus revenue sharing from product sales in licensed territories.
3. Ratable Recognition of Upfront Payment: Revenue recognized ratably from the $30 million upfront payment received from Nippon Shinyaku in Q1 2022 under the US Exclusive Commercialization and Distribution Agreement. Q3 2023 revenue was $6.2 million from this recognition.
4. Government Grants: Capricor has received funding from the California Institute for Regenerative Medicine (CIRM), classified as a loan/liability pending project completion outcomes.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

Capricor Therapeutics develops allogeneic cardiosphere-derived cell therapies, with its lead product candidate Deramiocel (CAP-1002) in Phase 3 development for Duchenne muscular dystrophy, and a proprietary StealthX™ exosome platform for delivering nucleic acids, proteins, and small molecules to treat or prevent various diseases. The company is pre-commercial with no approved products and no product revenue as of 2026.

Product overview

Capricor Therapeutics is a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases. The company's product portfolio consists of two core platforms: (1) Deramiocel (CAP-1002), an allogeneic cardiac-derived cell therapy in late-stage Phase 3 development for Duchenne muscular dystrophy, representing the lead commercial candidate; and (2) the StealthX™ exosome platform, a proprietary technology for engineering exosomes to deliver therapeutic cargo including oligonucleotides, proteins, and small molecules. These platforms address distinct therapeutic approaches - cell therapy for direct treatment of muscular and cardiac degeneration, and exosome-based delivery for broader applications in vaccinology and targeted therapeutics.

Differentiator

Problem solved

Functional benefit

Brands

• StealthX™: Proprietary exosome platform technology for delivering nucleic acids, proteins, and small molecules to treat or prevent a variety of diseases

Products and services

• Deramiocel (CAP-1002) An allogeneic cardiosphere-derived cell therapy in Phase 3 HOPE-3 clinical development for Duchenne muscular dystrophy (DMD). Demonstrates immunomodulatory, antifibrotic, and regenerative effects for dystrophinopathies and heart disease. Has received FDA RMAT, Orphan Drug, and Rare Pediatric Disease designations. Developed and manufactured by Capricor for distribution via its exclusive partner Nippon Shinyaku/NS Pharma in the US and Japan.
• StealthX Exosome Platform Proprietary exosome technology platform for developing next-generation therapeutics. Enables scalable production and loading of therapeutic oligonucleotides (siRNA, PMO), proteins, and small molecules into exosomes for targeted drug delivery. Used in preclinical development for vaccinology applications including an NIH-collaborated SARS-CoV-2 vaccine program.

Quantifiable outcome

• 54% slowing of skeletal muscle disease progression in DMD patients treated with Deramiocel versus placebo
• +4 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered core and minor.

• Nippon Shinyaku Co., Ltd.coreStrategic or Co-development Partner · 17 September 2024Capricor signed a binding term sheet with Nippon Shinyaku to expand commercialization of Deramiocel for Duchenne muscular dystrophy in Europe. Capricor receives a $20 million upfront payment, a $15 million equity investment, and potential milestone payments up to $715 million. Capricor develops and manufactures the drug for approved regions while Nippon Shinyaku handles sales and distribution. This agreement builds upon the existing US and Japan distribution agreement.
• Nippon Shinyaku Co., Ltd. (NS Pharma US Subsidiary)coreChannel Partner/ Reseller/ Distributor · 17 September 2024Exclusive commercialization and distribution agreement for Deramiocel (CAP-1002) for DMD in the United States and Japan. The agreement includes a $15 million equity investment, $20 million upfront payment, and potential milestones up to $715 million. Nippon Shinyaku's US subsidiary NS Pharma handles US commercialization, while Nippon Shinyaku handles Japan distribution.
• Nippon Shinyaku Co., Ltd.coreStrategic or Co-development Partner · 17 September 2024European expansion of existing partnership for Deramiocel commercialization. The deal includes revenue sharing from product sales in European markets, extending Capricor's cash runway into 2026. Capricor will develop and manufacture the therapy while Nippon Shinyaku handles commercialization in Europe, subject to regulatory approval.
• Parent Project Muscular Dystrophy (PPMD)minorGTM or Marketing Partner · 13 July 2023Capricor partnered with Parent Project Muscular Dystrophy to host a webinar providing community updates on Phase 3 HOPE-3 trial results. PPMD is a patient advocacy organization that supports families and drives research for Duchenne muscular dystrophy.
• National Institutes of Health (NIH)minorStrategic or Co-development Partner · 24 January 2023Collaboration with NIH for clinical trial of novel exosome-based multivalent vaccine for SARS-CoV-2. Capricor's StealthX™ exosome platform technology is being utilized for this vaccine development program.

Scale indicators8 records

Recent moves7 records

Expansion highlights4 records

Broad incumbents

• Pfizer: Pfizer is a large biopharma that has historically run DMD gene therapy programs (e.g., fordadistrogene movaparvovec). Comparable as a deep-pocketed incumbent competing in the same DMD space, but with a broader portfolio and AAV-based rather than cell-based modality.
• bluebird bio: bluebird is a commercial-stage gene and cell therapy company with approved products (Lyfgenia, Zynteglo, Skysona). Comparable as a rare-disease cell/gene therapy developer, though operating with lentiviral gene modification rather than cardiosphere-derived cells.
• Vertex Pharmaceuticals: Vertex is a leader in cell and gene therapy following the Casgevy approval for sickle cell disease and beta-thalassemia, with a broad pipeline across CF and rare diseases. Comparable as a commercial-stage cell/gene therapy company, though not focused on DMD.

Direct peers

• PTC Therapeutics: PTC commercializes Translarna (ataluren) for nonsense-mutation DMD and has an active DMD pipeline (including vatiquinone). Comparable to Capricor as a DMD-focused commercial/clinical-stage rare disease biotech, though with a small-molecule rather than cell therapy approach.
• Solid Biosciences: Solid Biosciences is a clinical-stage gene therapy company developing SGT-003 for DMD using an AAV-SLB101 capsid. Directly comparable to Capricor as a DMD-focused clinical-stage company, though pursuing gene therapy rather than cell therapy.
• Regenxbio: Regenxbio is developing RGX-202, an AAV gene therapy for DMD, leveraging its NAV technology platform. Directly comparable as a clinical-stage DMD gene therapy developer using a differentiated delivery platform.
• Mesoblast: Mesoblast develops allogeneic cell therapies (remestemcel-L, rexlemestrocel-L) for inflammatory and cardiac conditions. Directly comparable as an allogeneic, off-the-shelf cell therapy developer using proprietary cell platforms — the closest pure cell-therapy analog to Deramiocel.
• Sarepta Therapeutics: Sarepta is the leading commercial-stage DMD therapeutics company, with the approved gene therapy Elevidys and exon-skipping drugs (Exondys 51, Vyondys 53, Amondys 45). It is the most direct competitive comparable to Capricor given overlapping patient population, though it operates via gene therapy and antisense mechanisms rather than cell therapy.

Emerging players

• Athersys: Athersys (now operating as Helux) is a clinical-stage regenerative medicine company developing MultiStem, an off-the-shelf allogeneic cell therapy. Comparable as an early-stage allogeneic cell therapy developer, though with broader indications and a less advanced pipeline.
• Helixmith: Helixmith (formerly ViroMed, formerly VM BioPharma) is a clinical-stage gene therapy company developing Engensis (donaperminotide pegol) for DMD and other indications. Comparable as a DMD-focused clinical-stage developer, though pursuing a plasmid DNA rather than cell therapy approach.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xtwitter.com/Capricor
• Facebookfacebook.com/capricortherapeutics
• LinkedInlinkedin.com/company/capricor-inc-
• Instagraminstagram.com/capricortherapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Subsidiaries1 record

Funding overview

Funding rounds14 records

Investors9 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 Capricor Therapeutics