CorrectSequence Therapeutics

What CorrectSequence Therapeutics does

CorrectSequence Therapeutics is a clinical-stage biotechnology company founded in 2020 and headquartered in Shanghai, China, that develops next-generation gene editing therapies using a proprietary base editing platform. The company's core technology is the transformer Base Editor (tBE), a single-base editing system that uses a cleavable lock mechanism activating only at on-target sites, eliminating both gRNA-dependent and gRNA-independent off-target mutations without requiring DNA double-strand breaks. CorrectSequence holds a global exclusive perpetual license for the underlying base editing patents from ShanghaiTech University, which also houses several of its scientific co-founders. The company has built a portfolio of three clinical-stage programs: CS-101, an ex vivo gene editing therapy for transfusion-dependent β-thalassemia that received China's first NMPA IND approval for a base editing therapy in April 2024; CS-206, an ex vivo base editing therapy for sickle cell disease; and CS-121, an in vivo LNP-delivered gene editing therapy targeting APOC3 for hypertriglyceridemia, representing the company's expansion into metabolic disease and in vivo delivery.

The company operates a capital-efficient clinical model, having raised approximately USD 60 million across three disclosed funding rounds between 2020 and 2024 from investors including Sequoia China, Zoo Capital, New Alliance Capital, and TF Capital. It has built vertically integrated operations spanning an R&D center at the Shanghai Advanced Research Institute (Chinese Academy of Sciences, established June 2021), a CMC and cGMP manufacturing center at Zhangjiang Cell Industry Park (established May 2022), and a Regulatory & Clinical Center in Beijing (established May 2022). As of available data, CorrectSequence has treated more than 10 transfusion-dependent β-thalassemia patients from China, Laos, Malaysia, and Pakistan, and reported a 15+ month VOC-free outcome in its first sickle cell disease patient, with clinical results published in Nature in April 2026. The company is pre-revenue and generates no product sales; future revenue is expected to come from commercialization of approved gene editing therapies, with pricing and reimbursement models not yet disclosed.

Name

CorrectSequence Therapeutics

Legal name

CorrectSequence Therapeutics Co., Ltd.

Website

https://correctsequence.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

11–50 employees

Short description

CorrectSequence Therapeutics is a Shanghai-based, clinical-stage biotech developing tBE base editing therapies for genetic diseases, including CS-101 for β-thalassemia, CS-206 for sickle cell disease, and CS-121 for hypertriglyceridemia, serving patients globally.

Ownership category

akta.pro rank

Product category

Gene Therapy / Base Editing Biotechnology

NAICS

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

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro industry

PRIMARYHLAAACAN

Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream)

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAAACAL

Gene Therapy Delivery Devices & Administration Systems (catheters, intrathecal, ocular delivery)

Healthcare & Life Sciences/Biotechnology & Biologics/Gene Therapy (AAV/lentiviral/other vectors)

SECONDARYHLAAAIAC

Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization)

Healthcare & Life Sciences/Biotechnology & Biologics/Biologic Drug Discovery Platforms (screening, antibody engineering, synthetic biology)

Keywords

• Gene editing therapies
• Base editing technology
• Cell therapy development
• Biopharmaceutical research
• Genetic disease treatments

Headquarters

HQ city

Shanghai

HQ country

China

HQ region

Asia

Offices5 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

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

Revenue model

1. Gene Therapy Development: Pre-revenue clinical-stage company developing base editing therapies. Revenue will be generated through commercialization of therapeutic products after regulatory approval.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

CorrectSequence Therapeutics is a clinical-stage biotechnology company developing gene-editing therapeutics for severe genetic and metabolic diseases. Its pipeline is built on the proprietary transformer Base Editor (tBE) platform and includes lead assets CS-101 for β-thalassemia, CS-206 for sickle cell disease, and CS-121 for hypertriglyceridemia, with both ex vivo and in vivo delivery approaches enabled by LNP and AAV vectors.

Product overview

CorrectSequence Therapeutics is a clinical-stage biotechnology company pioneering transformer Base Editor (tBE) technology for developing gene editing therapies. The company's portfolio includes three main therapeutic products: CS-101 (base editing therapy for β-thalassemia, NMPA-approved), CS-206 (base editing therapy for sickle cell disease), and CS-121 (in vivo LNP-delivered therapy targeting APOC3 for hypertriglyceridemia). All products utilize the proprietary tBE platform which offers higher editing efficiency and better safety profiles compared to traditional CRISPR/Cas gene editing by eliminating off-target mutations through a unique lock-key mechanism.

Differentiator

Problem solved

Functional benefit

Products and services

• CS-101 Ex vivo gene-editing therapy for β-thalassemia using CorrectSequence's tBE base-editing platform, designed as a one-time curative treatment for patients with transfusion-dependent β-thalassemia and administered through hospital-based clinical investigators.
• CS-206 Ex vivo gene-editing therapy for sickle cell disease built on the tBE platform, intended as a one-time curative treatment for patients with severe sickle cell disease and administered through clinical trial sites.
• CS-121 In vivo gene-editing therapy for hypertriglyceridemia delivered using the tBE platform via LNP or AAV vectors, intended to provide a one-time treatment for patients with severe hypertriglyceridemia.
• tBE Base Editing Platform Proprietary transformer Base Editor (tBE) platform that performs precise base conversions without DNA double-strand breaks; offered to biopharmaceutical partners for out-licensing, as evidenced by the tBE licensing agreement with EdiGene.

Quantifiable outcome

• 15+ months vaso-occlusive crisis-free survival in sickle cell disease patient treated with CS-206
• +4 more outcomes

Named customers4 records

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Five partnerships are on record, tiered minor and core.

• Shanghai Clinical Research and Trial CenterminorImplementation/ SI/ Consulting Partner · 1 January 2024Acknowledged partner in clinical trial support and regulatory coordination for base editing therapies.
• Fudan University (Children's Hospital of Fudan University)coreStrategic or Co-development Partner · 1 November 2023Research collaboration with Prof. Li Yang from Center for Molecular Medicine, Children's Hospital of Fudan University. Joint publication in Cell Stem Cell on tBE therapy for beta-hemoglobinopathies. Clinical treatment of thalassemia patients.
• Wuhan UniversitycoreStrategic or Co-development Partner · 1 November 2023Research collaboration on base editing therapy development. Prof. Hao Yin is a professor at Wuhan University Medical Research Institute.
• First Affiliated Hospital of Guangxi Medical UniversitycoreStrategic or Co-development Partner · 1 October 2023Clinical trial collaboration for CS-101 and CS-206 therapies. Investigator-initiated trial (IIT) conducted for beta-thalassemia and sickle cell disease treatments. First clinical application site for company's base editing therapies.
• ShanghaiTech UniversitycoreTechnology or Integration · 1 August 2020Global exclusive perpetual license for base editing patents including transformer Base Editor (tBE) and enhanced Base Editor (eBE). Scientific co-founders (Jia Chen, Li Yang, Hao Yin, Bei Yang) are professors at ShanghaiTech University. Collaboration on research and development of base editing technologies.

Scale indicators8 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Beam Therapeutics: Beam Therapeutics is a US clinical-stage biotech pioneering base editing technology, the same gene editing modality as CorrectSequence's tBE platform. Both companies develop base editors targeting point mutations in hemoglobinopathies, metabolic disease, and oncology, making Beam the closest direct global peer.
• EdiGene: EdiGene is a Chinese clinical-stage gene editing company developing ex vivo and in vivo gene editing therapies for hemoglobinopathies and other genetic diseases. Both are China-based clinical-stage gene editing peers targeting overlapping indications including β-thalassemia and sickle cell disease.
• Verve Therapeutics: Verve Therapeutics develops in vivo base editing and gene editing therapies for cardiovascular disease, including programs targeting PCSK9, ANGPTL3, and LPA. CorrectSequence's CS-121 (APOC3 editing for hypertriglyceridemia) directly overlaps Verve's cardiovascular in vivo gene editing focus.
• Editas Medicine: Editas Medicine is a clinical-stage gene editing company developing CRISPR-based therapies including for sickle cell disease and other genetic disorders. Operates in the same gene editing therapeutic category as CorrectSequence, though with CRISPR/Cas technology rather than base editing.

Broad incumbents

• CRISPR Therapeutics: CRISPR Therapeutics is a leading clinical-stage gene editing company with the first FDA-approved CRISPR therapy (Casgevy for sickle cell disease). Competes directly in the sickle cell disease space where CorrectSequence's CS-206 is also being developed, and serves as the incumbent benchmark for the field.
• Intellia Therapeutics: Intellia Therapeutics is a clinical-stage gene editing company developing both in vivo and ex vivo CRISPR/Cas9 therapies for genetic diseases including transthyretin amyloidosis and hereditary angioedema. Operates in the same gene editing therapy category with broader modality coverage than CorrectSequence.
• Sangamo Therapeutics: Sangamo Therapeutics is a clinical-stage genomic medicine company developing zinc finger nuclease-based gene editing and gene regulation therapies for hemoglobinopathies and metabolic diseases. Pioneer in the gene editing field and broadly comparable as a precision gene medicine developer.

Emerging players

• Prime Medicine: Prime Medicine is developing prime editing, a precision gene editing approach that like base editing avoids double-strand breaks and offers high accuracy. Operates in adjacent precision gene editing modality and targets similar monogenic diseases, making it a thematic peer.
• Caribou Biosciences: Caribou Biosciences develops precision genome editing therapies using CRISPR base editing and other modalities for hematologic diseases and oncology. Operates in adjacent precision gene editing space with similar disease area focus.

Regional players

• Bioray Laboratories: Bioray Laboratories is a Chinese clinical-stage gene and cell therapy company with programs in hemoglobinopathies including β-thalassemia and sickle cell disease. Competes with CorrectSequence in the China domestic gene therapy market for similar indications.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/correctsequence-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Funding overview

Funding rounds1 record

Investors4 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 CorrectSequence Therapeutics