What Ensoma does

Ensoma is a Boston-based, clinical-stage genomic medicine company founded in 2019/2020 that develops in vivo hematopoietic stem cell (HSC) engineering therapies. Its proprietary Engenious Platform combines off-the-shelf, high-capacity virus-like particles (VLPs) — capable of carrying up to 35 kilobases of genetic cargo — with proprietary CRISPR-Cas and base editing tools to engineer HSCs directly within the patient. This in vivo architecture eliminates the ex vivo cell harvesting, manufacturing, and myeloablative conditioning required by conventional gene and cell therapies, with the goal of one-time, durable treatments potentially administered on an outpatient basis.

The lead clinical program, EN-374, is in a multicenter Phase 1/2 trial (NCT06876363) for X-linked chronic granulomatous disease (X-CGD), a rare immunodeficiency affecting 1 in 100,000–200,000 live births. EN-374 became the first in vivo HSC-directed gene insertion therapy to enter clinical evaluation when the first patient was dosed in December 2025, and has received FDA Orphan Drug and Rare Pediatric Disease designations as well as UK MHRA Clinical Trial Authorization. The pipeline additionally includes a sickle cell disease program using in vivo base editing to reactivate fetal hemoglobin, and a solid tumor program generating multiplexed CAR-M, NK, and T cells from engineered HSCs for HER2+ tumors.

Ensoma operates as a pre-revenue biotech funded by venture and strategic capital — approximately $138 million disclosed across a January 2023 $85 million financing and September 2025 $53 million round, with a syndicate including Gilead, 5AM Ventures, the Bill & Melinda Gates Foundation, Qatar Investment Authority, Viking Global Investors, and others. The company acquired Danish CRISPR-Cas specialist Twelve Bio ApS in February 2023 to deepen its gene editing toolkit. Future revenue is expected to derive from commercialization of one-time gene therapies via specialty pharmacies and designated treatment centers, with potential price points comparable to other approved gene therapies (e.g., $3.5M for hemophilia B).

Name

Ensoma

Legal name

Ensoma, Inc.

Website

https://ensoma.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

11–50 employees

Short description

Ensoma is a Boston-based clinical-stage biotech developing in vivo HSC engineering therapies via its Engenious Platform, which combines high-capacity VLPs with CRISPR-Cas and base editing to deliver one-time gene therapies for X-CGD, sickle cell disease, and solid tumors.

Ownership category

akta.pro rank

Product category

In Vivo Gene Therapy

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

Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL)

akta.pro secondary industries

Stem Cell Therapies (MSC/iPSC/HSC-derived) (HLAAABAE), Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery) (HLAAACAL)

Keywords

• In vivo gene therapy
• Hematopoietic stem cell engineering
• CRISPR gene editing
• Virus-like particle delivery
• Genomic medicines

Headquarters

HQ city

Boston

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, Infrastructure, Marketing or Sales

Revenue model

1. Gene therapy development and commercialization: Ensoma is developing one-time, durable gene therapies for genetic diseases (X-CGD, sickle cell disease) and cancer (solid tumors). As a clinical-stage company, it operates pre-revenue, funded by private investors and strategic partnerships. Revenue would derive from commercialization of its pipeline therapies upon regulatory approval.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels6 records

Core offering

Ensoma develops one-time, in vivo hematopoietic stem cell (HSC) engineering gene therapies through its proprietary Engenious Platform, which combines high-capacity virus-like particles (VLPs) with proprietary CRISPR-Cas and base editing tools. Its lead clinical-stage product EN-374 delivers a CYBB transgene to HSCs in vivo for X-linked chronic granulomatous disease (X-CGD), and the pipeline extends to in vivo base editing for sickle cell disease and multiplexed in vivo CAR-M, NK, and T cell therapy for solid tumors.

Product overview

Ensoma is an in vivo hematopoietic stem cell (HSC) engineering company developing one-time, off-the-shelf therapies through its Engenious Platform. The platform combines high-capacity virus-like particles (VLPs) with proprietary CRISPR-Cas and base editing tools to engineer HSCs directly within the body. Lead clinical program EN-374 (for X-linked chronic granulomatous disease) is in Phase 1/2 trials. The pipeline includes programs for sickle cell disease (base editing to reactivate fetal hemoglobin) and solid tumors (multiplexed CAR-M, NK, T cell therapy).

Differentiator

Problem solved

Functional benefit

Brands

• Engenious: The company's proprietary technology platform combining virus-like particles (VLPs) with advanced gene engineering tools for in vivo HSC engineering.

Products and services

• EN-374 First-in-class in vivo hematopoietic stem cell (HSC)-directed gene insertion therapy for X-linked chronic granulomatous disease (X-CGD). Uses virus-like particles (VLPs) to deliver the CYBB transgene to HSCs to restore NADPH oxidase function in neutrophils; currently in a multicenter Phase 1/2 trial (NCT06876363) in the U.S. and U.K. for adult and pediatric X-CGD patients.
• Sickle Cell Disease Program Preclinical in vivo HSC engineering program using base editing to reactivate fetal hemoglobin (HbF) for treatment of sickle cell disease, designed to simplify the patient treatment journey compared with complex ex vivo approaches that require blood transfusions, stem cell mobilization, myeloablative conditioning, and extended hospital stays.
• Solid Tumor CAR Program Preclinical in vivo HSC engineering program generating multiplexed CAR-M, NK, and T cells for solid tumor treatment through lineage-restricted expression of CARs, with preclinical proof-of-concept demonstrated in HER2+ tumor models showing durable tumor control and prolonged survival compared to controls.

Quantifiable outcome

• EN-374 Phase 1/2 trial demonstrated tolerability in first participant with all adverse events being low-grade; no serious AEs or dose-limiting toxicities observed
• +3 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

Feature6 records

Partnerships

Two partnerships are on record, tiered core and major.

• Profluent BiocoreStrategic or Co-development Partner · 9 December 2025Profluent Bio and Ensoma entered a strategic collaboration to develop AI-designed base editors for in vivo hematopoietic stem cell therapies. The companies aim to leverage Profluent's AI technology to create advanced gene editing tools that could improve treatments for hematologic and immune diseases. Located in Emeryville, California.
• Twelve Bio ApSmajorOthers · 9 February 2023Ensoma completed the acquisition of Twelve Bio ApS, a Danish gene editing company specializing in next-generation CRISPR-Cas medicines, following Danish regulatory approval. Key personnel from Twelve Bio joined Ensoma's leadership and advisory teams as part of the integration. The acquisition enhanced Ensoma's CRISPR technology capabilities as part of the $85M financing.

Scale indicators4 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Locanabio: RNA-targeting genetic medicines company developing treatments for neuromuscular and neurodegenerative diseases. Comparable as Ensoma's CEO Jim Burns previously led Locanabio, and both companies operate in the in vivo genetic medicines space with platform-based approaches.
• bluebird bio: Pioneer in ex vivo lentiviral gene therapy with approved products in sickle cell disease (Lyfgenia) and beta-thalassemia. Directly comparable as a competing gene therapy approach for the same hemoglobinopathy indication Ensoma targets with in vivo base editing, and Drew Dietz (VP Clinical) led late-stage trials at bluebird.
• CRISPR Therapeutics: Co-developer of Casgevy (exa-cel), the first FDA-approved CRISPR/Cas9 gene therapy, partnered with Vertex for sickle cell disease and beta-thalassemia. Most direct competitor to Ensoma's in vivo SCD program using ex vivo CRISPR editing of patient HSCs.
• Beam Therapeutics: Clinical-stage base editing company developing in vivo and ex vivo base editing therapies across hemoglobinopathies, oncology, and rare diseases. Directly comparable as Ensoma's base editing-based sickle cell program uses similar underlying editing chemistry against Beam's pipeline.
• Intellia Therapeutics: Leader in in vivo CRISPR/Cas9 and Cas9-based gene editing with clinical-stage programs in transthyretin amyloidosis and hereditary angioedema. Comparable as a clinical-stage in vivo gene editing company pursuing systemic delivery of editing payloads, though Intellia uses LNP delivery rather than Ensoma's VLP approach.
• Verve Therapeutics: Clinical-stage in vivo base editing company targeting cardiovascular diseases with a one-time treatment approach. Highly comparable as a fellow in vivo gene editing platform company that has validated the systemic in vivo editing approach clinically, addressing a similar regulatory and capital markets landscape.
• Editas Medicine: Clinical-stage CRISPR gene editing company developing ex vivo and in vivo gene editing therapies for hemoglobinopathies and ophthalmologic indications. Comparable as a clinical-stage editing company with overlapping hemoglobinopathy focus and similar platform-based approach.

Emerging players

• Shape Therapeutics: Preclinical-stage RNA editing and gene therapy company with platform technologies for in vivo gene correction. Comparable as an emerging player in the in vivo gene editing space; Ensoma's VP Clinical Drew Dietz previously led clinical development at Shape.
• Chroma Medicine: Preclinical-stage epigenetic editing company developing gene silencing and activation therapies. Comparable as an emerging platform-based gene regulation company with related leadership pedigree (Ensoma's SVP Legal previously SVP at Chroma).
• Prime Medicine: Clinical-stage gene editing company developing prime editing technology for precise genetic corrections across multiple diseases. Comparable as an emerging gene editing platform pursuing broad indication coverage, though using a distinct editing chemistry (prime editing) versus Ensoma's base editing.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/EnsomaBio
• LinkedInlinkedin.com/company/ensomabio

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Funding overview

Funding rounds4 records

Investors22 records

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

M&A1 record

Investments

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

Frequently asked questions about Ensoma