EX VIVO & IN VIVO GENE CORRECTION

Personalized Gene Editing Two Distinct Gene-Correction Strategies for LGMD2B/R2

ACGENETIC Therapeutics is developing two distinct, variant-directed approaches to address the underlying genetic causes of LGMD2B/R2. Our ex vivo strategy generates gene-corrected muscle satellite cells from iPSCs for intramuscular (IM) administration, while our in vivo strategy delivers gene-editing components intravenously (IV) to directly correct endogenous muscle satellite cells. These approaches may provide a foundation for future expansion into other inherited disorders.

EXPLORE OUR STRATEGIES

Explore Our Two Gene-Correction Strategies

About · Company

Beginning with patients. Advancing through science.

We investigate variant-directed approaches and functional restoration for inherited muscle disorders.

Two complementary approaches

PILLAR 01 · EX VIVO

iPSC-based cell therapy approach

We investigate correction of patient-derived cells and differentiation toward Pax7+ muscle progenitor and satellite-cell states.

PILLAR 02 · IN VIVO

LNP-based direct correction approach

We are developing a non-viral platform for transient delivery of mRNA and engineered guide RNA.

Our Research Began with a Patient’s Determination

We remain committed to advancing research toward new therapeutic approaches for inherited muscle disorders

Founded by a Patient Driven by Science

ACGENETIC was founded by a patient with LGMD2B/R2 to pursue new therapeutic approaches for inherited muscle disorders. His family’s shared experience with the disease further strengthens the company’s long-term dedication to this research.

Science · Overview

Uniting precision correction with targeted delivery

Our strategy considers editing, delivery, cell state, and genomic stability together.

Pipeline · Overview

From correction
to functional restoration.

Starting with DYSF correction in LGMD 2B/R2, we are building two complementary programs.

PROGRAM 01 · EX VIVO

iPSC-based cell therapy approach

DYSF-corrected iPSC → Pax7+ cells → local transplantation

Local delivery · IM
Stage 1 · iPSC gene correction Completed
Stage 2 · Pax7+ cell differentiation In progress
PROGRAM 02 · IN VIVO

LNP-based direct correction approach

Formulation → delivery research → in vivo assessment

Systemic delivery · IV
Stage 1 · LNP–mRNA formulation and in vitro evaluation In progress
Stage 2 · Preclinical in vivo delivery and efficacy Planned
Stages describe internal research progress and do not indicate clinical-stage status or regulatory approval.

Advancing Gene-Corrected iPSC-Derived Muscle Satellite Cells for Intramuscular Delivery

Our ex vivo strategy focuses on differentiating gene-corrected iPSCs into muscle satellite cells for intramuscular (IM) administration

Strategy 01
Ex Vivo Correction

We evaluate whether muscle satellite cells derived from gene-corrected iPSCs can engraft and contribute to the formation of dysferlin-expressing myofibers

Ex Vivo Research Workflow

  1. DYSF variant correction in iPSCs
  2. Quality assessment of gene-corrected iPSCs
  3. Differentiation into muscle satellite cells
  4. Preclinical intramuscular (IM) delivery and engraftment evaluation
  5. Assessment of dysferlin-expressing myofiber formation
EXPLORE OUR OTHER STRATEGY

In Vivo Platform

Advancing Transient In Vivo Gene Correction through Intravenous LNP Delivery

Our in vivo strategy delivers LNP-encapsulated gene-editing components intravenously (IV), aiming to achieve transient gene-editor expression and directly edit endogenous muscle satellite cells

Strategy 02
In Vivo Correction

We evaluate transient gene-editor expression following intravenous LNP delivery, along with biodistribution, immunogenicity, redosing potential, and genomic integrity

In Vivo Research Workflow

  1. LNP encapsulation of gene-editing components
  2. LNP formulation and surface engineering for muscle delivery
  3. Intravenous (IV) delivery and biodistribution assessment
  4. Assessment of transient gene-editor expression and editing in endogenous muscle satellite cells
  5. Assessment of immunogenicity, redosing potential, and genomic integrity
EXPLORE OUR OTHER STRATEGY

Ex Vivo Platform

Patients & Mission

We are Patients.
We are Scientists.

Patients remain central from the first research question through the path toward the clinic.

Focused on outcomes that matter to patients

We envision an ecosystem where patient organizations and clinical centers can engage from the preclinical stage.

Platform Expansion

A research platform designed to adapt by variant

We are exploring a master-protocol concept combining a shared delivery foundation with variant-specific guide design.

Variant-Adaptable
Master Protocol Concept

01

Shared delivery foundation

02

Variant-specific guide RNA

03

Disease and variant expansion research

This is an early concept and does not represent a finalized trial design or regulatory agreement.

Together, We Can Advance Research for Inherited Muscle Disorders